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Displaying 1 - 137 of 137

Family Nurture Intervention in the NICU

Condition: Pediatrics
Investigator: Martha Welch, MD
Status: Closed
The purpose of this study is to compare neurodevelopment and activity in infants born very preterm (26 to 33 6/7 weeks gestational age (GA)) receiving Standard Care (SC) or Family Nurture Intervention (FNI) in the neonatal intensive care unit (NICU). This study investigator hypothesizes that FNI will improve: i) neonatal electroencephalographic activity ii…
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Study Evaluating KTE-C19 in Subjects With Acute Lymphoblastic Leukemia or B-Cell Non-Hodgkin Lymphoma (ZUMA-4)

Condition: Pediatrics / Cancer
Investigator: Prakash Satwani, MD
Status: Closed
The main purpose of this research study is to determine if the experimental product, KTE-C19, when administered after you receive 3 days of chemotherapy, is safe and effective in treating your leukemia or non-Hodgkin lymphoma.
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Study Evaluating KTE-C19 in Pediatric Subjects With Acute Lymphoblastic Leukemia or B-Cell Non-Hodgkin Lymphoma (ZUMA-4)

Condition: Pediatrics
Investigator: Prakash Satwani, MD
Status: Closed
The main purpose of this research study is to determine if the experimental product, KTE-C19, when administered after you receive 3 days of chemotherapy, is safe and effective in treating your leukemia or non-Hodgkin lymphoma.
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ApricityCare: Smartphone App Study for Patients with Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
The therapy you are receiving for your cancer has known side effects. If those side effects are caught early, they may be less serious, easier to treat, and less likely to lead to discontinuation of your cancer therapy. Apricity Health has created the Apricity CARE program, which includes a smartphone app called ApricityCare for monitoring and reporting…
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Studies of Pediatric Liver Transplantation (SPLIT)

Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Closed
The purpose of the study is to collect scientific data on pediatric liver transplantation to learn more about how children do after liver transplant. Collecting these data is expected to help researchers learn more about liver transplant in children. This information may improve medical care of your child and other children who need and who have received…
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REST: Sleep Study for High School Students

Condition: Pediatrics
Investigator: Brooke Aggarwal, EdD, MS, FAHA
Status: Closed
High school students ages 14-18 and their parent(s) may be eligible to participate in a 12-week (consisting of a 6-week pre-pilot and 6-week pilot) sleep health education program.
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Lung Cancer Patient Survey Study on Technology Support for Treatment Preparedness

Condition: Cancer / Lung Cancer
Investigator: Alicia Matthews
Status: Closed
We are conducting a survey to gather feedback about lung cancer patients experience with treatment and the challenges you faced. Your insights can help shape better care for future patients and explore how technology can be of help! The survey takes 30-40 minutes, and eligible participants who complete it will receive a gift card. Interested in…
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PEPN2113: Study of Uproleselan in Children with AML, MDS, or MPAL

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 study of a drug called uproleselan. We are testing new experimental drugs such as uproleselan in the hopes of finding a treatment that may be effective against acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that has come back or that has not responded to standard therapy. This study looks at how well…
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Study of CLN-081 in Patients with Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
This is a research study evaluating a new investigational drug, called CLN-081. CLN-081 is a type of drug called a tyrosine kinase inhibitor. It targets a mutation in the tumor cell called EGFR exon 20 insertion with the goal to slow down or stop cancer cell growth. It is taken orally (by mouth). There are several different parts to this study, each with…
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Study of Osimertinib (TAGRISSOTM) in Patients with Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study to learn more about whether the study drug osimertinib (TAGRISSOTM) is more effective than placebo in participants with epidermal growth factor receptor (EGFR) mutated stage IA2 or IA3 Non-Small Cell Lung Cancer (NSCLC) after complete surgical removal of the tumor. Stages IA2 and IA3 refer to tumor size. A placebo is an inactive…
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Study of Adagrasib Alone or in Combination with Nivolumab Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Benjamin Herzberg, MD
Status: Closed
The purpose of this study is to assess the benefits and risks of the study drug, adagrasib, when given either by itself or with another drug, nivolumab, to participants with non-small cell lung cancer and a specific cancer mutation, called KRAS G12C, and whether the study drug or the combination may treat or shrink tumors when given before cancer surgery.…
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Natural History Study of Children with SCID (Severe Combined Immunodeficiency)

Condition: Pediatrics
Investigator: Olatundun Williams, MD
Status: Closed
This study is being done to identify the best outcomes for hematopoietic cell transplantation (HCT) or other treatment (enzyme replacement or gene therapy), which is life-saving therapy for children with SCID (severe combined immunodeficiency), leaky SCID, Omenn syndrome and reticular dysgenesis.
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Study of Ramucirumab Plus Pembrolizumab vs. Standard of Care in Patients with Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Stephanie Smith-Marrone, MD
Status: Closed
The purpose of this study is to compare the drugs ramucirumab and pembrolizumab to standard-of-care treatment in patients whose lung cancer has progressed on immunotherapy. The study doctors hope to learn if the drug combination will help patients with stage IV or recurrent non-small cell lung cancer (NSCLC) live longer. The study is set up to find out if…
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Learn More About How We Decide Which Children With Headaches Need to Have a Picture of Their Brain Taken While They Are in the ED

Condition: Pediatrics
Investigator: Daniel Tsze, MD
Status: Closed
The purpose of this research study is to create a decision-making tool that will help doctors decide which children with headaches require a CT ("CAT scan," "computed tomography") or MRI scan of their head as part of their emergency department care.
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Impact on Learning and Development in Down Syndrome(CHILD-DS)

Condition: Pediatrics / Down Syndrome
Investigator: Michael DiLorenzo, MD
Status: Closed
Down syndrome (DS) occurs in roughly 1 out of 700 births in the U.S. Half of all children with Down syndrome have congenital heart disease (CHD), often requiring corrective heart surgery. Even though CHD in children without DS is known to be associated with developmental problems, few studies have explored how CHD can affect the development and behavior of…
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Understanding the Life Course of Primary Immune Regulatory Disorders (PIRD) Patients and Their Family Members

Condition: Pediatrics
Investigator: Olatundun Williams, MD
Status: Closed
The purpose of this study is to understand the life course of PIRD patients and their family members that have the same gene change but do not have the same symptoms. PIRD is mostly due to inherited changes in the genes that normally have immune cells fight infections and regulate the immune system. There are many types of problems that patients with PIRD…
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Testing Whether the Use of Brain Scans Alone vs. Brain Scans Plus Brain Radiation Affects Lifespan in Patients With Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Simon Cheng, MD, PhD
Status: Closed
We are doing this study because we want to find out if MRI brain scans alone is better or worse than MRI brain scans plus preventative brain radiation for patients with small lung cancer. The usual approach for patients with small cell lung cancer is prophylactic cranial irradiation. If you decide to take part in this study, you will either receive PCI for…
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Study of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

Condition: Pediatrics
Investigator: Dana Goldner, MD
Status: Closed
This study evaluates whether odevixibat is safe and effective compared to placebo in children with biliary atresia who have recently undergone a Kasai procedure. This is a double-blind study, so the study doctors and the child/parent will not know whether the child is receiving odevixibat or placebo. Participation will involve several research visits over…
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Study of Quizartinib in Combination with Chemotherapy for Children and Young Adults with Recurrent /Refractory Acute Myeloid Leukemia (AML)

Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with…
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Study of Savolitinib in Combination with Osimertinib vs. Chemotherapy in Patients with Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to learn more about an investigational drug called savolitinib in combination with osimertinib for the treatment of non-small cell lung cancer (NSCLC) compared to platinum-based doublet chemotherapy (a standard of care treatment). Participants will be randomly assigned to one of two study groups at the beginning of this study.…
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Learn About Nasal Spray Medication That Helps Children Stay Calm and Hold Still When They Have a Cut in Their Skin That Needs to be Fixed

Condition: Pediatrics
Investigator: Daniel Tsze, MD
Status: Closed
The purpose of this research study is to find out what is the best dose of intranasal midazolam for children to help them stay calm and hold still while they are having a cut in their skin fixed.
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FAST TRIAL REGISTRY - Study for Female, Pregnant Patients Affected by Atrial Flutter (AF) or Supraventricular Tachycardia (SVT)

Condition: Pediatrics
Investigator: Stephanie Levasseur, MD
Status: Closed
Although supraventricular tachycardia (SVT), including atrial flutter (AF), are the most common causes of intended in-utero fetal therapy, none of the medication used to date has been evaluated for their effects on the mother and her baby in a randomized controlled clinical trial (RCT). In the absence of such evidence, there is no consensus for the optimal…
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Study of Nivolumab in Combination with 5-Azacytidine in Acute Myeloid Leukemia (AML) - Children and Adults Age 1 to 30

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs…
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Study of Osimertinib (TAGRISSOTM) in Non-Small Cell Lung Cancer (NeoADAURA) Patients

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to see whether the medication osimertinib (TAGRISSOTM) alone or when used in combination with chemotherapy is more effective than chemotherapy alone for the treatment of early stage Non-Small Cell Lung Cancer prior to surgery. Osimertinib is an EGFR TKI therapy that targets EGFR mutations. When a tumor is found to have this type…
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Study of Patritumab Deruxtecan in Combination with Osimertinib in Patients with Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to determine the combination dose of patritumab deruxtecan and osimertinib that can be given to patients safely and to understand how well patritumab deruxtecan alone or in combination with osimertinib works to treat lung cancer. Osimertinib is an EGFR TKI therapy that targets EGFR mutations. Osimertinib has already been…
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A Study for Children with Acute Lymphoblastic Leukemia Using Study Drug Carfilzomib

Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to learn more about the treatment of children and teenagers whose leukemia has come back (has relapsed) or has not gone away after other treatments (is refractory). Participants will receive the study drug, called carfilzomib in combination with standard chemotherapy, that are dexamethasone, daunorubicin, vincristine and PEG-…
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ICON3: A Phase 3 Study of Eltrombopag vs. Standard Therapy for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

Condition: Pediatrics
Investigator: Cindy Neunert, MD
Status: Closed
The purpose of this study is to investigate the safety and effectiveness of eltrombopag (investigational drug) in treating children and adolescents with newly-diagnosed immune thrombocytopenia (ITP), which is a rare blood disorder.
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Liver-Directed Radiotherapy Along with Chemotherapy for Patients with Extensive Stage Small Cell Lung Cancer (ES-SCLC) and Liver Metastases

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
The purpose of this study is to evaluate whether radiation treatment directed at liver metastases can be safely added to standard of care treatment for extensive-stage small cell lung cancer (ES-SCLC). The current standard treatment for people who have ES-SCLC is chemotherapy, called carboplatin and etoposide, that is combined with a type of immunotherapy…
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Study of Combining Osimertinib with and without Ramucirumab in Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to compare the usual treatment for Non-Small Cell Lung Cancer (osimertinib) to a combination therapy (osimertinib plus ramucirumab). The combination therapy arm is testing if ramucirumab can help osimertinib control the abnormal EGRG protein for a longer time and keep the disease inactive longer. EGRF protein typically helps the…
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Study of Berzosertib Compared to Chemotherapy for Patients with Non-Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Benjamin Herzberg, MD
Status: Closed
The purpose of this study is to compare the usual treatment (carboplatin, gemcitabine, and pembrolizumab) alone to using berzosertib (M6620, VX-970), plus the usual treatment for patients with squamous non-small cell lung cancer. The addition of berzosertib (M6620, VX-970) to the usual treatment could prevent your cancer from getting worse. But, it could…
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Fetal Atrial Flutter & Supraventricular Tachycardia (FAST) Therapy Trial (Fetus diagnosed with a fast heart rate)

Condition: Pediatrics / Cardiology
Investigator: Stephanie Levasseur, MD
Status: Closed
Although supraventricular tachycardia (SVT), including atrial flutter (AF), are the most common causes of intended in-utero fetal therapy, none of the medication used to date has been evaluated for their effects on the mother and her baby in a randomized controlled clinical trial (RCT). In the absence of such evidence, there is no consensus for the optimal…
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Study of Atezolizumab and Tiragolumab Alone or in Combination with Chemotherapy in Patients with Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to assess the effects, good or bad, of atezolizumab and tiragolumab given with or without chemotherapy (in this study, chemotherapy will be a combination of either paclitaxel with carboplatin, pemetrexed with carboplatin or cisplatin, or gemcitabine with carboplatin or cisplatin) on patients who are able to have surgery to…
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Tomivosertib With Anti-PD-(L)1 in Subjects With Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
The purpose of the study is to see whether tomivosertib in combination with pembrolizumab treatment slows down the growth of cancers. The study drug, tomivosertib, works by inhibiting the activity of proteins in a pathway that allows tumor cells to grow and go unnoticed by your immune system. This study will evaluate the safety and antitumor activity of…
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Study of Pembrolizumab Alone or in Combination with Chemotherapy in Older Patients with Non-Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
This trial studies the side effects of pembrolizumab with or without chemotherapy in treating patients with non-small cell lung cancer that has come back and has spread to other places in the body. Pembrolizumab is an immunotherapy that may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and…
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Study in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC) whose Disease has Gotten Worse After Osermitinib Treatment

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to gain a better understanding of why some patients with non-small cell lung cancer and an epidermal growth factor receptor (EGFR-sensitizing mutation, fail first-line treatment with the drug osermitinib. Additionally, the purpose is to explore different drug therapies for patients whose cancer has progressed (gotten worse)…
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Learn More About Positive Pressure Ventilation for Asthma

Condition: Pediatrics / Child and Adolescent Health
Investigator: Maria Kwok, MD, MPH
Status: Closed
The purpose of this study is to evaluate the effectiveness and adverse reactions of the various types of noninvasive positive pressure supportive use in the management of children with moderate to severe acute asthma exacerbation.
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Study of Olanzapine in Children Receiving Chemotherapy for Hematopoietic Stem Cell Transplant (HSCT)

Condition: Pediatrics / Cancer
Investigator: Diane George, MD
Status: Closed
This research study is to find out whether adding olanzapine to standard antiemetics will be helpful in controlling chemotherapy-induced vomiting (CINV) in children receiving high-dose cyclophosphamide for Blood/Bone Marrow Transplant (BMT) conditioning.
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Study of MTX110 in Combination with Gadolinium in Children with Newly Diagnosed Brain Cancer (Diffuse Midline Gliomas)

Condition: Pediatrics / Cancer
Investigator: James Hinkley Garvin Jr, MD
Status: Closed
The purpose of this study is to find the best dose of a drug called MTX110 and a contrast agent called gadolinium that can be given safely in children with newly diagnosed diffuse midline gliomas, which is a kind of brain tumor. All patients enrolled in the study will receive infusion of MTX110 and Gadolinium delivered with a pump directly into the tumor…
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Study of Amivantamab and Carboplatin-Pemetrexed Therapy, versus Carboplatin-Pemetrexed, in Participants With Non-Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to see if amivantamab in combination with administration of standard of care chemotherapy (carboplatin and pemetrexed) is safe and useful for treating patients with Non-Small Cell Lung Cancer (NSCLC), specifically in patients with a gene abnormality called EGFR Exon 20 insertions. There are two study arms for this trial.…
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Study of Study Drug JNJ-61186372 in Patients with Advanced Non-Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to see if the study drug, JNJ-61186372, is safe and useful for treating patients with advanced, metastatic, non-small cell lung cancer (NSCLC). There are 2 parts of this study. The first part is to determine the dose of JNJ-61186372 that is safe to give to patients with non-small cell lung cancer. The second part of the study is…
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Study of Durvalumab with Platinum-based Chemotherapy in Completely Resected Stage II-III NSCLC (MeRmaiD-1)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
This study is aimed at finding patients who have small amounts of cancer DNA in their blood (called circulating tumor DNA or ctDNA) after surgery. This may indicate that you have minimal residual disease (MRD), meaning that even after surgery, there are still some remaining cancer cells releasing cancer DNA into your bloodstream. MRD after surgery may mean…
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Study of Capmatinib for Patients with Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this research study is to find out if a drug called capmatinib is safe and effective (can help people) in people who have non-small cell lung cancer (NSCLC) who are also eligible for surgery. Capmatinib is an oral drug that targets particular processes, which may not be working properly in the cancer cells (dysregulation) in the body. Prior…
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Study of JNJ-93841937 Participants with Advanced Non-Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to see if lazertinib and JNJ-61186372 are safe and useful for treating patients with a type of lung cancer called advanced non-small cell lung cancer (NSCLC) as well as looking at the effects that lazertinib and JNJ-61186372 have when taken together. Lazertinib is taken orally and JNJ-61186372 is an intravenous drug. Lazertinib…
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Study of Cabergoline in Adolescents and Adult Women with Endometriosis

Condition: Pediatrics
Investigator: Jin Hee Kim, MD
Status: Closed
In this research study we want to learn more about a potential new therapy for pain associated with endometriosis. We want to determine if a non-hormonal medication, cabergoline, will help patients with endometriosis. Cabergoline is a medication that is approved by the Food and Drug Administration (FDA) for treatment of other medical conditions. In this…
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Teens' Thoughts About Depression and Mental Health Related Discrimination

Condition: Pediatrics
Investigator: Doron Amsalem, MD
Status: Closed
We are conducting a research study to explore discrimination around depression and barriers to care in youth ages 14-18. We will use what we learn to create videos to reduce such discrimination. Parental permission is required for this study. Adolescents (ages 14-18) will participate in an individual interview that will be approximately 45 minutes long. We…
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Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients (6 months-17 years)

Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find out the effects and safety of an investigational (experimental) new drug called copanlisib in pediatric patients from 6 months to 21 years of age. This study consists in two-phase, Phase I part will determine which is the right dose of copanlisib to be used in children/adolescents/adults with relapsed (has returned) or…
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Study of Drug Injection with Chemotherapy in Children, Adolescents, and Young Adults with Relapse of Actue Lymphoblastic Leukemia

Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of Vincristine Sulfate Liposome Injection (Marqibo) in combination with UK ALL R3 induction chemotherapy. The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children with acute lymphoblastic leukemia (ALL) who have relapsed…
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Study of Flotetuzumab for Children, Adolescents, and Young Adults with Relapsed or Refractory Acute Myeloid Leukemia (AML)

Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the highest dose of Flotetuzumab that can be given safely to patients who have a certain type of leukemia called Acute Myeloid Leukemia (AML) that has relapsed (has come back after treatment) or is refractory (never responded to treatment). Flotetuzumab is an experimental treatment that works by targeting certain…
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Study of Selinexor in Pediatric Solid Tumors, including CNS Tumors

Condition: Cancer / Pediatric Solid Tumors
Investigator: Nobuko Hijiya, MD
Status: Closed
We are testing new experimental drugs such as selinexor in the hopes of finding a treatment that may be effective against tumors that have come back or that have not responded to standard therapy. The goals of this study are: To find the highest safe dose of selinexor that can be given without causing severe side effects; To learn what kind of side effects…
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Study of Maralixibat in Infants with Biliary Atresia Who Have Had Kasai Surgery

Condition: Pediatrics
Investigator: Jennifer Vittorio, MD
Status: Closed
The purpose of this study is to see how effective and safe the drug maralixibat is when compared to placebo in infants with biliary atresia who have had Kasai surgery within 3 weeks before participation. This is a 26-week, multicenter, double-blind, placebo-controlled, randomized parallel-group study, followed by an open-label extension (OLE). Approximately…
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Spearhead 1 Study in Subjects With Advanced Synovial Sarcoma or Myxoid/Round Cell Liposarcoma

Condition: Pediatrics / Cancer
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to find out if an investigational drug, called ADP-A2M4, is safe and how well it works in attacking Synovial Sarcoma or Myxoid/Round Cell Liposarcoma (MRCLS) cancer cells. The study involves taking certain types of immune cells from the blood, called T cells, and changing them in a laboratory before putting them back into the…
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Study of Durvalumab + Tremelimumab in Non-Small Cell Lung Cancer Patients After Adjuvant Treatment

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this research study is to see how effective durvalumab andtremelimumab is in patients with early stage non-small cell lung cancer (NSCLC). A a special blood test will be done to see if subjects have circulating tumor DNA (ctDNA) in their blood. ctDNA are pieces of genetic material shed from the tumor cells that can be detected in the blood,…
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Social and Contextual Impact on Children Undergoing Liver Transplant

Condition: Pediatrics
Investigator: Jennifer Vittorio, MD
Status: Closed
The social determinants of health have a large impact on health. We have previously demonstrated that an index of neighborhood socioeconomic deprivation was associated with an increased risk of medication non-adherence in children after liver transplant and an increased risk of graft failure and death in children after transplant. The present proposal seeks…
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Study of Metronomic and Targeted Anti-Angiogenesis Therapy For Children with Medullobastoma (Brain Tumor)

Condition: Pediatrics / Cancer
Investigator: Stergios Zacharoulis, MD
Status: Closed
The purpose of this study is to learn more about the treatment of children and young adults patient with relapsed (has returned) and progressive medulloblastoma (brain tumor). This study is being done to find out if bevacizumab can be safely given in combination with five study drugs given by mouth, thalidomide, fenofibrate, celecoxib and alternating every…
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A study for infants with pre-symptomatic Spinal Muscular Atrophy (SMA)

Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Darryl De Vivo, MD
Status: Closed
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can…
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Study of M7824 and cCRT in Patients with Unresectable Stage III Non-small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
The purpose of this study is to assess the overall safety and efficacy (how well the drug works) of study drug M7824 in the treatment of unresectable, non-small cell lung cancer. Another purpose is to determine whether use of the study drug in combination with chemoradiation (chemotherapy plus radiation therapy) followed by the study drug will result in…
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A study for patients with advanced Non-Small-Cell Lung Cancer (NSCLC) using study drug MEDI4736 in combination with Tremelimumab

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
The purpose of this study is to evaluate the combination of MEDI4736 and tremelimumab in treating non-small cell lung cancer. This study will look to see whether MEDI4736 combined with tremelimumab or MEDI4736 alone are more effective than standard of care chemotherapy in treating lung cancer and to collect information on the safety of these drugs. Both…
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Study of Patients Receiving an Allogeneic Stem Cell Transplantation for Non-Malignant Disease

Condition: Pediatrics
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant condition.
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HUDSON: Study of Multiple Treatments in Patients with Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
This is an open-label, Phase II study in patients with metastatic non-small cell lung cancer (NSCLC) whose cancer has gotten worse on an anti-programmed cell death-1/anti-programmed cell death ligand 1 (anti-PD-1/PD-L1) containing therapy. This study consists of a number of treatment cohorts to test the efficacy, safety, and tolerability of multiple…
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MLN4924 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome (1 month to 17 years old)

Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in children with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) that has either come back or has not responded to the standard therapy. Pevonedistat works by blocking some of the enzymes that are needed for cell growth.
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A study for children with refractory solid tumors, including CNS tumors using study drug ramucirumab

Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
The goals of this study are to find the highest safe dose of ramucirumab that can be given without causing severe side effects, to learn what kind of side effects ramucirumab can cause, to learn more about the effects of ramucirumab on cells and proteins in the blood and to determine whether ramucirumab is a beneficial treatment for patients with solid…
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Testing combination drugs in children with solid tumors that have come back or are resistant to current anti-cancer therapy

Condition: Cancer / Pediatric Solid Tumors
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of pevonedistat in combination with Temozolomide and Irinotecan in pediatric patients with refractory solid tumors, including CNS tumors and lymphoma. The study treatment is considered experimental because pevonedistat is not approved by the United States (US) Food and Drug Administration (FDA) for treating pediatric…
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Study for children with recurrent or refractory solid tumors using study drug axitinib

Condition: Cancer / Pediatric Solid Tumors
Investigator: Julia Glade Bender, MD
Status: Closed
The purpose of this study is to assess the safety and effectiveness of a study drug called axitinib on treating tumors that have returned (recurrent) or have not responded to standard therapy (refractory). Children will take axitinib twice a day for 28 days (1 cycle). Children may take study drug for up to 24 cycles (2 years).
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A clinical trial for children with 15Q duplication syndrome or CDKL5 deficiency disorder to help control seizures.

Condition: Pediatrics
Investigator: Wendy Chung, MD, PhD
Status: Closed
The purpose of this research is to determine whether a new investigational drug called TAK-935 is safe and whether it may help treat epilepsy in children with 15q duplication syndrome or CDKL5 deficiency disorder.
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Adjuvant Lung Cancer Enrichment Marker Identification and Sequencing Trial (ALCHEMIST)

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
This purpose of this study is to determine whether the tumor cells of lung cancer patients contain changes to the DNA sequence of two proteins found on the cells surface- ALK and EGFR. If the tumor contains changes to the DNA of one of these proteins, the patient may be invited to participate on a trial that will look at drugs which may target tumors that…
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Study of Entinostat in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors and Lymphoma

Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
This study is being carried out by the Childrens Oncology Group (COG) Phase 1 Consortium. COG is an international research group that consists of more than 200 hospitals that treat children with cancer in the United States, Canada, Australia, and Switzerland. The Phase 1 Consortium is the group within COG that consists of 21 hospitals, and participation in…
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A Study to Evaluate Immunotherapy Combinations in Participants With Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
This research study is testing two potential new drugs called AB928 and AB122. AB928 is an investigational drug that has been shown in animal studies to improve the functioning of the immune system, without causing any immune effects on its own. This research study will help us understand whether AB928, the study drug, can be safely given in combination…
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A study for patients with non-small cell lung cancer using study drug AZD9291

Condition: Cancer / Lung Cancer
Investigator: Sewanti Limaye, MD
Status: Closed
The FLAURA Study is a new clinical research study underway in NSCLC and we are looking for people who would like to join. This study will examine an investigational drug, called AZD9291, and compare it tostandard, approved drugs that are already used to treat lung cancer. These drugs are called gefitinib and erlotinib. When we talk about investigational, it…
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CD34 Stem Cell Selection for Malignant Condition

Condition: Pediatrics / Cancer
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a malignant condition.
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Tacrolimus/Everolimus vs. Tacrolimus/MMF in Pediatric Heart Transplant Recipients Using the MATE Score (TEAMMATE) (Pediatric)

Condition: Pediatrics / Cardiology
Investigator: Irene Lytrivi, MD
Status: Closed
This is a Phase 3 multi-center open label randomized clinical trial. The purpose of this study is to learnmore about which anti-rejection (or immunosuppressive) medications best protect infants, children,adolescents, and young adults after they have had a heart transplant. Two different drug regimens will bewill be compared. All of these drugs are FDA-…
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Study of Drug in Combination with Traditional Chemotherapy for Patients with Stage IV Small-Cell Lung Cancer (SCLC)

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
This trial will look at how the study drug will work in combination with traditional chemotherapy at treating extensive-stage small cell lung cancer in the first-line setting. The study will also determine if these drugs cause any side effects compared to traditional chemotherapy alone. The study drug is Durvalumab by itself or with Tremelimumab. Both…
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Decitabine and Vorinostat Followed by Fludarabine, Cytarabine, and G-CSF (FLAG) in Children & Young Adults with Acute Myeloid Leukemia

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of Decitabine (DEC) and Vorinostat (VOR) followed by the standard chemotherapy drugs (Fludarabine, Cytarabine and G-CSF (FLAG)). The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children with acute myeloid leukemia (AML)…
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Test of an inhaled medicine (Aerosurf) to treat premature lung disease in babies born at 26 to 28 weeks gestation.

Condition: Pediatrics
Investigator: Tina Leone, MD
Status: Closed
Babies born prematurely frequently have difficulty breathing because of immature lung development. A substance called surfactant is usually present in mature lungs to help keep the lungs inflated normally. The treatment for breathing problems in premature infants can include giving surfactant as a medicine. When surfactant is given as a medicine a…
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Reducing Surgical Complications in Newly Diagnosed Lung Cancer Patients Who Smoke Cigarettes

Condition: Cancer / Lung Cancer
Investigator: Bryan Stanifer, MD
Status: Closed
This randomized phase III trial studies how well management of a tobacco treatment intervention works in reducing surgical complications in patients with newly diagnosed lung cancer who smoke cigarettes. Management of a tobacco treatment intervention compares varenicline (a drug that reduces the craving and withdrawal symptoms that occur with abstinence…
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A study for patients with brain metastases from non-small cell lung cancer (NSCLC) using radiosurgery

Condition: Cancer / Lung Cancer
Investigator: Teri Kreisl, MD
Status: Closed
The purpose of this study is to obtain information on the safety and effectiveness of NovoTTF-100M (study device) in subjects with brain metastases (tumors) as a result of non-small cell lung cancer (NSCLC).
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Study of EBV-CTLs in Patients with Epstein-Barr Virus-Associated Viremia or Lymphoma

Condition: Pediatrics / Cancer
Investigator: Ran Reshef, MD
Status: Closed
Epstein-Barr Virus (EBV) is the virus that causes mononucleosis, also known as mono. Almost all adults have had EBV. For most people, EBV never causes a health problem. However, when their immune system is not working properly (people who have had a transplant and those who have immune disorders), EBV can cause certain diseases where cells grow abnormally.…
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Evaluating the safety and effectiveness of study drug AMG 228 in patients with Advanced Solid Tumors

Condition: Cancer / Pediatric Solid Tumors
Investigator: Yvonne Saenger, MD
Status: Closed
This study is for patients with the following cancers: advanced non-small cell lung cancer, head and neck cancer, melanoma, colon cancer, or bladder cancer. The purpose of this study is to find out more about the effects of AMG 228 in people and on their cancer. AMG 228 is an experimental drug that is being developed to stimulate the bodys immune system to…
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Radiation and Immune Checkpoints Blockade in Metastatic Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
The purpose of this study is to test whether local radiotherapy combined with Ipilimumab is safe. The study will try to find out if it can help patients with metastatic non-small cell lung cancer better than treatment with Ipilimumab and Nivolumab alone.
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Study for patients with advanced Non-small Cell Lung Cancer using study drug MEDI4736

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
The purpose of this study is to evaluate the combination of study drug MEDI4736 and tremelimumab in treating advanced non-small cell lung cancer that is resistant to or returned after receiving standard therapies, for which there is no available standard therapies, or for individuals who have declined standard therapies. MEDI4736 and tremelimumab are fully…
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Study of Hematopoietic Stem Cell Transplantation (HSCT) in non-cancer disease

Condition: Pediatrics
Investigator: Monica Bhatia, MD
Status: Closed
The overall purpose of this research is to treat diseases that are not cancer but are improved by bone marrow transplantation with mild and safer chemotherapy as pretransplant conditioning. This treatment is chosen because it is milder and is likely to have fewer long and short term side effects unlike older chemotherapy and radiation treatments. Yet, it…
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Study of Drug in Patients with Early Stage Non-Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
The purpose of this study is to find if adding the study drug, nivolumab (also known as OPVIDO), will limit lung cancer from growing back in patients with early stage non-small cell lung cancer. Nivolumab is a drug that may turn on the body's immune system to attack any cancer cells that may remain after surgery. The addition of nivolumab may help…
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Study of Experimental Drug in Pediatric Patients with Solid Tumors, including CNS Tumors

Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
This is a Phase 1 multicenter study of a drug called ABI-009 (nab-Rapamycin) in combination withtemozolomide and irinotecan in children and young adults with solid tumors (including CNS tumors)which have come back or have not responded to standard therapy. The study is considered experimental because ABI-009 (nab-Rapamycin) is not approved by the United…
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A study for children with high-risk neuroblastoma using study drug unituxin

Condition: Pediatrics / Cancer
Investigator: Alice Lee, MD
Status: Closed
The purpose of this research study is to collect information about the percentage of people that develop HACA when they are treated with Unituxin and determine if HACA affects the blood levels of Unituxin. Unituxin is a type of antibody. Antibodies are made by the body to attack tumors and fight infections. Unituxin is a chimeric antibody which means it is…
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Study of Telaglenastat in First-Line, Metastatic KEAP1/NRF2-Mutated, Nonsquamous, Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
The purpose of this study is to determine the amount (dose) of telaglenastat that is safe and tolerable when given together with the recommended treatment regimen of pembrolizumab, carboplatin, and pemetrexed and to determine if the combination of standard drugs with telaglenastat is more active against non-small cell lung cancer (NSCLC) than the standard…
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Study of New Drug with Radiation Therapy in Children with Diffuse Intrinsic Pontine Gliomas (DIPG) Brain Stem Cancer

Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
This is a phase I, multicenter, dose escalation study of MK-1775 in combination with radiation. The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children and young adults with newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG). MK-1775 is an oral…
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Study of Stepped Palliative Care versus Early Integrated Palliative Care in Patients with Advanced Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Craig Blinderman, MD, MA
Status: Closed
Research has shown that early involvement of a team of clinicians that specialize in lessening (or palliating) many of these distressing physical and emotional symptoms and in helping patients and their family cope with a serious illness improves patients and their loved ones experience with their cancer. This team is called palliative care, and consists of…
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Study of Nivolumab Monotherapy and Nivolumab in Combination with Ipilimumab in Subjects with Central Nervous System Malignancies

Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find out more about the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab (also known as BMS-936558) when given alone or when combined with ipilimumab in children/ adolescents/ young adults with high grade primary central nervous system (CNS) malignancies (certain…
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Identifying tumor neoantigens during immune checkpoint blockade in patients with non-small cell lung cancer

Condition: Cancer / Lung Cancer
Investigator: Adrian Sacher
Status: Closed
This study is for adults who have non-small cell lung cancer and have had surgery to remove their lung tumor. People who have lung cancer and undergo surgery still have a significant risk of their cancer returning outside of the lung. The purpose of this study is to determine if a new type of immune-based therapy can increase your chance of being cured.…
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Breathe Easy Early ORBEX Study

Condition: Pediatrics
Investigator: Meyer Kattan, MD
Status: Closed
The Breathe Easy Early ORBEX Study is a three -year study to identify if a daily capsule of Broncho-Vaxom may help prevent or delay young children from developing wheezing or asthma- like symptoms. Other studies have found that Broncho-Vaxom can help prevent upper respiratory infections and stimulate immune defenses, and has been safely and effectively used…
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A study for male adolecents with muscular dystrophy using study drug Eteplirsen

Condition: Pediatrics / Neuromuscular
Investigator: Jacinda Sampson, MD
Status: Closed
The purpose of this study is to find out if eteplirsen can help people with the muscle disorder called Duchenne Muscular Dystrophy (DMD) and find out if eteplirsen is safe to take without causing too many side effects. Eteplirsen is an investigational (experimental) drug and can only be used in research studies. DMD is caused by a mutation (a change) in the…
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Study of Whole-Brain Radiation With or Without Hippocampal (part of Brain) Avoidance in Patients with Small Cell Lung Cancer (SCLC)

Condition: Cancer / Lung Cancer
Investigator: Tony J. Wang, MD
Status: Closed
The purpose of this study is to compare any good and bad effects of avoiding the hippocampus during whole brain radiation to the usual whole-brain radiation. The hippocampus is part of the brain that is important for memory. Avoiding the hippocampus during whole-brain radiation could decrease the chance of side effects on memory and thinking. It also is…
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A study for patients with advanced or metastatic lung cancer using study drug Nivolumab

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
The purpose of this study is to test the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab on patients with advanced or metastatic non-small cell lung cancer . Nivolumab is an antibody (a type of human protein) that is being tested to see if it will allow the bodys immune system to work against…
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Study of a New Treatment (Atezolizumab) in Combination with Platinum-Based Chemotherapy in Patients with Non-Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to compare the effects of atezolizumab, an immunotherapy drug, with chemotherapy or placebo with chemotherapy in patients with resectable NSCLC to find out which is better. In this study, you will receive treatment before undergoing surgery to remove the tumor.
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MUPPITS-2: Study of Asthma Medication in Children with Asthma

Condition: Pediatrics
Investigator: Meyer Kattan, MD
Status: Closed
Research funded by the NIH is being done to learn if an asthma medication, given along with standard asthma care, makes children less likely to have asthma attacks.
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A study for children and adolescents with relapsed or refractory acute myeloid leukemia using study drug Lenalidomide

Condition: Pediatrics / Cancer
Investigator: Alice Lee, MD
Status: Closed
This study is for children with relapsed or refractory acute myeloid leukemia (rrAML). Acute myeloid leukemia is a fast growing cancer of the blood and bone marrow. This study examines the effects of study drug Lenalidomide on children with rrAML. Lenalidomide is an anti-cancer drug that belongs to a group of drugs known as immunomodulating drugs (IMiDs), a…
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Study to Evaluate Monotherapy and Combination Immunotherapies in Participants With PD-L1 Positive Non-small Cell Lung Cancer (ARC-7)

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
The purpose is to study the safety and the effect of AB122, AB154, and AB928 in participants with stage IV non-small cell lung cancer. Researchers will study the treatment response of AB122 alone as well as in combination with AB154 and AB928. There are three possible groups a participant can be randomly assigned to. Group 1 will include AB122 alone, Group…
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A study for patients with advanced Non small cell lung cancer using study drug Nivolumab

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this master study is to test the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab (also known as BMS-936558) alone or in combination with standard of care therapies.
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Study of Weekly MOD-4023 Compared to Daily Genotropin Therapy In Pre-Pubertal Children With Growth Hormone Deficiency

Condition: Pediatrics
Investigator: Ilene Fennoy, MD
Status: Closed
Your child has been asked to participate in this study because he/she may have or has growth hormone deficiency (i.e. not enough growth hormone in his/her body) and your doctor has already determined that your child may be suitable to be screened for this study. OPKO Biologics Ltd., the Sponsor of this clinical study, has developed an…
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Study of Combinations of REGN2810, Chemotherapy, and Ipilimumab vs. Pembrolizumab Monotherapy in Patients with Non-Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
This study is being done to determine if REGN2810 (anti-PD-1) in combination with ipilimumab (anti-CTLA-4) plus initial platinum-based doublet chemotherapy, or REGN2810 in combination with ipilimumab will prolong how long you live without your disease progressing compared to the standard of care pembrolizumab (anti-PD-1) monotherapy treatment. Neither…
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Study of Entrectinib in Children and Adolescents with Solid Tumors and Primary CNS Tumors

Condition: Cancer / Pediatric Solid Tumors
Investigator: Stergios Zacharoulis, MD
Status: Closed
The purpose of this study is to find out more about how the study drug called entrectinib will be used in children, adolescents and young adults with treatment-resistant solid tumor in brain or body. This is a phase 1/1b study, divided into a dose escalation portion (different dose will be tested) and a dose expansion portion (one dose will be tested) in…
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A study for patients with small cell lung cancer using study drug SGI-110

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
This study will investigate the safety and effectiveness of the study drug SGI-110 in combination with two immunotherapy drugs named durvalumab and tremelimumab in patients with small cell lung cancer who have progressed on other treatments. SGI-110 is a form of chemotherapy known as a "hypomethylating agent" and may help the immunotherapy work…
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Study of Combination Therapies in Children with Relapsed Acute Lymphoblastic Leukemia (ALL) or Non-Hodgkins Lymphoma (NHL)

Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of temsirolimus in combination with etoposide and cyclophosphamide. The study treatment is considered experimental because it is not approved by the Unites States (US) Food and Drug Administration (FDA) for treating children with acute lymphoblastic leukemia (ALL) and non-Hodgkins lymphoma (NHL) who have relapsed. The…
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A study for children with pediatric acute Lymphoblastic Leukemia using study drug Moxetumomab Pasudotox

Condition: Pediatrics / Cancer
Investigator: Maria Sulis, MD
Status: Closed
The purpose of this study is to determine the effectiveness and safety of the study drug moxetumomab pasudotox in treating children with relapsed or refractory pALL or Lymphoblastic Lymphoma of B-cell origin. Moxetumomab pasudotox is made up of two parts: 1) a modified mouse antibody that attaches to a protein from the immune system, called CD22, which can…
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GSK3359609 Plus Tremelimumab for the Treatment of Advanced Solid Tumors (Non-small Cell Lung Cancer)

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
This research study is being done to test the combination of two new drugs, GSK3359609 and Tremelimumab, in certain cancers. These two drugs are experimental which means they have not been fully tested yet and are not yet approved to treat any disease. There are two parts to this study. Part 1 determines the best dose for the study drugs and part 2 compares…
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A study for patients with locally advanced or metastatic non-small cell lung cancer using study drug AZD9291

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
The purpose of this study is to see if study drug AZD9291 is effective in treating non-small cell lung cancer (NSCLC) and if so, how it compares with chemotherapy. Only patients with NSCLC that has been confirmed as having the EGFR mutation are eligible to participate. Before patients can participate in the main study a tumor sample will be taken to check…
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Improvement of exercise capacity in patients with Fontan physiology

Condition: Pediatrics / Cardiology
Investigator: Marc Richmond, MD
Status: Closed
Over time, children and young adults who have had a Fontan operation may find they are more tired, have difficulty breathing and cannot exercise as well as before. Currently, there are no approved medicines to prevent this decline. The purpose of this research study is to learn whether children who have had the Fontan operation can improve their ability to…
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Mothers' Attitude Towards Breastfeeding in Mothers with Babies with Congenital Heart Disease

Condition: Pediatrics
Investigator: Leonardo Liberman, MD
Status: Closed
The purpose of this study is to investigate how mothers with babies with congenital heart disease feel about breastfeeding. We will ask you to fill out a survey while you are in the hospital at around the time of delivery and another survey approximately three months later to see how you feel about breastfeeding. In addition to the survey data, the study…
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Study of Drug vs. Placebo in Patients with Early Stage Non-Small Cell Lung Cancer (NSCLC)

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
The purpose of this study is to determine if the study drug is safe and evaluate whether adjuvant therapy with the study drug will improve overall survival. The study drug is crizotinib (also known as XALKORI). The study will also collect tumor tissue and blood samples for future research.
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Study of Drug in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors

Condition: Pediatrics / Cancer
Investigator: Alice Lee, MD
Status: Closed
This is a Phase 1 multicenter study of LY2606368, a CHK1/2 inhibitor, in pediatric patients with refractory solid tumors, including CNS tumors. The study treatment is considered experimental because LY2606368 is not approved by the United States (US) Food and Drug Administration (FDA) for treating pediatric patients with refractory solid tumors, including…
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A study for pediatric patients with mild to moderate Ulcerative Colitis using study drug MMX Mesalamine/Mesalazine

Condition: Pediatrics
Investigator: Ali Mencin, MD
Status: Closed
This study is evaluating an investigational medication to see if it is safe and if it works well in children andadolescents who have mild to moderate ulcerative colitis (UC) or are in remission. Ulcerative colitis (UC) is a chronic disease of the last section of the intestine, which is also known as the colon. If you have UC, the lining of your colon…
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A study for patients with non-small cell lung cancer (NSCLC) using study drug atezolizumab with standard chemotherapy to shrink tumors

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
This study is for patients who are being considered for surgical removal of a specific kind of lung cancer called "non-small lung cancer" (NSCLC), which is a description of the way it looks under a microscope. The purpose of this study is to determine whether adding an investigational treatment called atezolizumab (also called MPDL3280A) to…
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Study of Combination Treatment to Reduce Occurrence and Severity of Chronic Graft Versus Host Disease (GVHD)

Condition: Pediatrics / Cancer
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to compare two different combinations of treatment plans to a standard transplant procedure in order to see whether one or both of them are better at reducing the occurrence and severity of chronic GVHD. The research portion of this study involves manipulation of your stem cell product by removing T-cells, which cause chronic…
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Biomarker-Driven Study for Second Line Therapy of Squamous Cell Lung Cancer (Lung-MAP)

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
The purpose of this study screening step is to perform genetic tests on your tumor sample for certain features that investigational targeted agents used in this study are specifically designed to work against. Then the purpose is to assign you to a treatment sub-study that is chosen based on the results (genes and proteins called biomarkers) of this genetic…
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Study of MK-1775 in Combination with Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumor

Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
This is a phase 1/2 multicenter study of AZD1775 (MK-1775) that will be given in combination with irinotecan. The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children and young adults with solid tumors which have come back or have not responded to standard…
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A study for children with an Inammatory Bowel Disease (IBD) such as Crohn's disease or ulcerative colitis

Condition: Pediatrics
Investigator: Joel Lavine, MD
Status: Closed
Researchers at Columbia University are conducting a clinical research study of children with inammatory bowel disease (IBD). This study will examine the eectiveness and safety of an investigational medication inchildren with IBD.
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Flex CMT Muscle Cramps Study

Condition: Pediatrics / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
Moving forward in Charcot-Marie-Tooth disease. A clinical research study assessing the safety and effectiveness of an investigational product in people with Charcot-Marie-Tooth (CMT) disease experiencing muscle cramps is now enrolling. Participation is entirely voluntary. Contacting us does not mean that you are required to take part in this study. If you…
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A study of children with relapsed or refractory solid tumors and large cell lymphoma using study drug, PF-02341066 for treatment

Condition: Cancer / Pediatric Solid Tumors
Investigator: Julia Glade Bender, MD
Status: Closed
The purpose of this study is to examine the effectiveness and tolerability of a study drug called PF-02341066. Additional goals of this study are to learn how the body handles the study drug as well as determine whether or not PF-02341066 is a beneficial treatment for your tumor This study is for children with a recurrent or progressive tumor or lymphoma…
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Study of Erlotinib in Patients with Non-Small Cell Lung Cancer (NSCLC) with EGFR Mutations

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
The purpose of this study is to look at the effects of treating Patients with Completely Resected Epidermal Growth Factor Receptor (EGFR) Mutant Non-small Cell Lung Cancer with the standard treatment or standard treatment in addition to Erlotinib. Subjects will be randomized to one of the two study groups. Subjects will be on the study for up to two years.
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A study for patients with advanced non-small cell lung cancer using study drug Alectinib

Condition: Cancer / Lung Cancer
Investigator: Sewanti Limaye, MD
Status: Closed
The purpose of this study is to compare the effects, good or bad, of drugs alectinib and crizotnib on patients with lung disease to find out which is better for patients with a special type of non-small cell lung cancer (NSCLC) that is called anaplastic lymphoma kinase (ALK)-positive NSCLC. In this study, participants will receive either alectinib or…
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Bowel Prep Study

Condition: Pediatrics
Investigator: Ali Mencin, MD
Status: Closed
This research study is for children age 12 to 16. In this research study, we will evaluate the safety, effectiveness and tolerability of two different doses of a bowel cleansing medication referred to as BLI-800. One group of study participants will receive the larger dose (6 oz.), which dose has been approved by the FDA for use by adults. A second group of…
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Early Developmental Changes, Risk for Diabetes, and the Microbiome in Children

Condition: Pediatrics
Investigator: Sharon Oberfield, MD
Status: Closed
We are recruiting boys between 3-9 years old and girls between 3-8 years old to participate in a research study. Children who would like to participate must be healthy and not showing signs of puberty. The goal of the study is to understand the relationship between early body development, metabolism (sugars and fats processing), and the saliva microbiome (…
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Study of Treatment Combinations in Patients with Metastatic Non-Small Cell Lung Cancer (NSCLC) (Morpheus-Lung)

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
This study is being done to test the safety and efficacy of immunotherapy based treatment combinations in patients with metastatic non-small cell lung cancer. Participants will be assigned to one of two groups, Cohort 1 or Cohort 2. Each cohort will be split into Stage 1 and Stage 2 phases.
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Body Composition Changes after Bariatric Surgery in Adolescents

Condition: Pediatrics
Investigator: Wei Shen, MD
Status: Closed
This is a study of body changes that take place in teenagers who undergo bariatric surgery as a treatment for obesity and obesity-related conditions. Changes will be measured by serial MRI andmetabolomic profiling.
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A study in children, adolescents, and young adults recurrent or refractory solid tumors using study drug nivolumab

Condition: Cancer / Pediatric Solid Tumors
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to learn what kind of side effects nivolumab as a single agent and nivolumab in combination with ipilimumab can cause and if these treatments are beneficial for solid tumors. Nivolumab (Dose Level 1) will be given as an intravenous infusion every 14 days of a 28 day cycle. If Dose Level 1 is not tolerable, then it will be…
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Study of RO7121661, a PD-1/TIM-3 Bispecific Antibody, in Participants With Advanced and/or Metastatic Solid Tumors

Condition: Cancer / Lung Cancer
Investigator: Brian Henick, MD
Status: Closed
The purpose of this study is to determine the doses of RO7121661 that can be administered safely to patients and what the side effects of RO7121661 are. In addition, how RO7121661 affects your body and, in particular, your tumor will be measured. The study also aims to investigate if RO7121661 can slow down or stop the growth of tumors in patients with…
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A study to identify the benefits of study drug pembrolizumab in patients with advanced or recurrent cancers

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
This is a research study to test the effectiveness of pembrolizumab on cancer types having a known relationship to smoking carcinogen exposure. The study will enroll 120 subjects age 18 and above with advanced solid tumor related to smoking exposure. All patients will receive the treatment of pembrolizumab. The study aims to determine if tumor genetics can…
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Effects of varying bubble nasal continuous airway pressure in very low birth weight infants

Condition: Pediatrics
Investigator: Rakesh Sahni, MD
Status: Closed
The purpose of this study is to see if the use of bubble nasal continuous positive airway pressure (NCPAP) at a pressure of 6-7 centimeters of water (cm H2O) in very low birth weight (VLBW) infants is associated with improved gas exchange without any related changes in vital signs.
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Study of Drug in Non-Small Cell Lung Cancer

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
The purpose of this study is to test the safety of the study drugs and how well your body tolerates it. The study will also test how well the study drugs work to slow down the growth of you tumor or stop your cancer. The study drugs are Cergutuzumab Amunaleukin and Atezolizumab. Atezolizumab is FDA approved for the treatment of patients with metastatic non-…
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Study of Posaconazole (POS) in Immunocompromised Pediatric Subjects with Neutropenia (Low Neutrophils Type of White Blood Cell Count)

Condition: Pediatrics / Cancer
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to: 1) Test the safety and tolerability of the research study drug, MK-5592 posaconazole (POS), intravenously (IV) and in powder for oral suspension (PFS), 2) Evaluate the pharmacokinetics (how the study drug is absorbed and broken down in the body), of POS IV solution and POS PFS, and 3) Evaluate the taste and reaction to the…
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A study for children and adolescents with advanced non central nervous system (CNS) tumors using study drug Talimogene laherparepvec

Condition: Cancer / Pediatric Solid Tumors
Investigator: Stergios Zacharoulis, MD
Status: Closed
The purpose of this study is to find out more about how the study drug called talimogene laherparepvec will be used in children/adolescents/young adults with advanced solid tumors not involving the central nervous system that are available for direct injection. This is a phase 1 study, multicenter, open-label study, that means all subject enrolled in the…
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Small Fiber Neuropathy in Juvenile Fibromyalgia

Condition: Pediatrics
Investigator: Alexis Boneparth, MD
Status: Closed
Juvenile fibromyalgia (JFM) is a cause of chronic, whole-body pain. The cause of JFM is unknown. One of the possible causes of JFM is a problem with nerves called "small fiber neuropathy." This is a problem where small nerve fibers in the skin are not working properly and patients can have pain as a result. Recent studies have found evidence of…
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A study for patients with spinal muscular atrophy using study drug, ISIS 396443 to test safety and effectiveness

Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Claudia Chiriboga, MD, MPH
Status: Closed
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in…
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Study of Crizotinib (Xalkori) for ALK-Positive Non-Small Cell Lung Cancer (NSCLC) Patients Versus Placebo

Condition: Cancer / Lung Cancer
Investigator: Naiyer Rizvi, MD
Status: Closed
The purpose of this study is to compare the effects of using crizotinib (also known as Xalkori) after completion of surgery, and in some cases, after chemotherapy and/or radiation therapy for ALK-positive non-small cell lung cancer patients versus placebo. Participants will be randomized to crizotinib or placebo. Participants will take crizotinib/placebo…
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A study for subjects with non-small cell lung cancer using study drug Ruxolitinib

Condition: Cancer / Lung Cancer
Investigator: Balazs Halmos, MD
Status: Closed
The purpose of this study is to find out whether the drug, ruxolitinib, is effective, tolerable and safe in combination with two chemotherapy medicines called pemetrexed and cisplatin. The study will test if this combination has beneficial effects in people diagnosed with certain types of non-small cell lung cancer. Only participants with Stage IIIB,…
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Brave Trial, A Clinical Research Study for Duchenne Muscular Dystrophy (DMD)

Condition: Pediatrics / Neuromuscular
Investigator: Darryl De Vivo, MD
Status: Closed
Our clinic is currently participating in the Brave Trial, a clinical research study for Duchenne muscular dystrophy (DMD). This study is being conducted to learn whether a study drug can improve motor functions in boys with DMD. If your child takes part, he'll receive at no cost: study drug or placebo, study-related procedures, reimbursement for…
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Study to Evaluate Novel Combination Therapies in Subjects with Previously Treated EGFRm NSCLC

Condition: Cancer / Lung Cancer
Investigator: Catherine Shu, MD
Status: Closed
The purpose of this study is to look at whether the combination of MEDI9447 and osimertinib or MEDI9447 and AZD4635 will improve the outcome in subjects with advanced epidermal growth factor receptor mutant (EGFRm) non-small cell lung cancer (NSCLC).
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Study of Glecaprevir/Pibrentasvir in Pediatric Subjects with Chronic Hepatitis C Virus (HCV) Infection

Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Closed
Open-labeled hepatitis C pediatric treatment trial with a two drug direct acting viral oral combination (already approved in adults) across all genotypes for naive or treatment experienced subjects.
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