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Displaying 1 - 60 of 60

Research Database for Cell Transplant and Other Cellular Therapies

Condition: Transplant / Stem Cell Transplant
Investigator: Markus Mapara, MD
Status: Currently Recruiting
The Center for International Blood and Marrow Transplant Research (CIBMTR), the research program of the National Marrow Donor Program (NMDP)/Be The Match, invites you to take part in a Research Database. The CIBMTR does research with medical data from patients who have had a transplant or other cellular therapy and donors who donate bone marrow or blood…
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Study of Focused Ultrasound (FUS) with DEFINITY Microbubbles in Children and Young Adults with Progressive Diffuse Midline Glioma (DMG)

Condition: Pediatrics / Cancer
Investigator: James Hinkley Garvin Jr, MD
Status: Currently Recruiting
Children and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine…
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Pediatric Acute Leukemia (PedAL) Screening Trial - Developing New Therapies for Relapsed Leukemias (Children and Young Adults)

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study is called a screening study and the purpose of this study to find better ways to diagnose and treat leukemia that has come back after treatment or difficult to treat. Bone marrow, blood, and medical information about cancer and treatment will be collected. The results from this screening study may give other information about leukemia that is…
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Study of R-MVST Cells to Treat Patients with a Viral Infection

Condition: Transplant / Stem Cell Transplant
Investigator: Pawel Muranski, MD
Status: Currently Recruiting
The purpose of this study is to test whether giving an experimental cell product can treat viral infection in patients who have conditions that cause the poor function of their immune system. The patients with infections caused by viruses such as cytomegalovirus (CMV), Epstein-Barr virus (EBV), adenovirus (ADV), BK and JC polyomaviruses (PyVs) virus may…
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Early Age-Related Hearing Loss Investigation (EARHLI)

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Justin Golub, MD, MS
Status: Currently Recruiting
Early Age-Related Hearing Loss Investigation (EARHLI) is a study to obtain information on how a hearing aid-based intervention can prevent cognitive decline in those at risk for Alzheimer's Disease and Alzheimer's Disease Related Dementias (AD/ADRD). Age-related hearing loss (ARHL) is the third most common chronic condition in older adults. The…
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Study of Relatlimab and Nivolumab in Patients Under 30 Years Old with Classical Hodgkin Lymphoma (cHL) and Non-Hodgkin Lymphoma (NHL)

Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study will utilize an investigational drug combination (also known as the study drugs) called relatlimab and nivolumab as a possible treatment for recurrent/relapsed (returned) or refractory (treatment-resistant) cHL and NHL. An investigational drug combination is one that has not been approved by regulatory agencies, such as the United States (US)…
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A Study for Patients with Advanced Melanoma, Solid Tumors or Lymphoma Using Study Drug Pembrolizumab

Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
The purpose of this study is to determine if a study drug called pembrolizumab is safe and effective in the treatment of children and adolescents who have advanced melanoma, or advanced, relapsed, or refractory PD-L1 positive malignant solid tumor or other lymphoma, relapsed or refractory classical Hodgkin lymphoma (rrcHL), advanced, relapsed or refractory…
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Long-Term Follow-up Study for Participants of Kite-Sponsored Gene-Modified Therapy Studies

Condition: Transplant / Stem Cell Transplant
Investigator: Ran Reshef, MD
Status: Currently Recruiting
The purpose of this study is to monitor you for any delayed side effects that may possibly be related to the experimental gene-modified therapy product you received in the Kite-sponsored study for Axicabtagene ciloleucel (axi-cel). The main study does not involve treatment. In the re-treatment portion, you will be provided access to a second course of…
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Speech Perception in Hearing Impaired Listeners

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Nima Mesgarani, PhD
Status: Currently Recruiting
The purpose of this study is to learn more about the mechanisms of hearing loss, specifically to learn how abnormalities in the brain may produce certain experiences that are common in patients with hearing loss. Individuals with hearing loss will be compared to individuals with normal hearing.
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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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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 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 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 ALVR105 (Viralym-M) in Patients Undergoing Allogeneic Hematopoietic Cell Transplant (HCT)

Condition: Transplant / Stem Cell Transplant
Investigator: Prakash Satwani, MD
Status: Closed
Are you undergoing a stem cell transplant? You may be at high risk for infections after the transplant. This study is looking at the safety and effectiveness of an investigational medication for those who have undergone a stem cell transplant. Recovery after a stem cell transplant can already be challenging without the added worry of life-threatening viral…
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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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Study of GDC-8264 in Stem Cell Transplant Patients with Acute Graft-Versus-Host-Disease (acute GVHD)

Condition: Transplant / Stem Cell Transplant
Investigator: Ran Reshef, MD
Status: Closed
This study is testing a drug called GDC-8264, which is being developed to treat acute graft-versus-host-disease (acute GVHD). GDC-8264 is an experimental drug, which means health authorities have not approved GDC-8264 in combination with standard medications for the treatment of acute GVHD. The purpose of this study is to evaluate the effects, good or bad,…
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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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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 Itacitinib and Tocilizumab for the Treatment of Steroid Refractory Acute Graft versus Host Disease in Stem Cell Transplant

Condition: Transplant / Stem Cell Transplant
Investigator: Markus Mapara, MD
Status: Closed
Graft versus host disease (GVHD) is a common complication after allogeneic stem cell transplant. It happens when the immune system of the donor is attacking the organs, and usually causes a skin rash, diarrhea, nausea and vomiting, and liver abnormalities. While it is often treated with steroids, some patients are considered steroid-refractory, which means…
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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 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 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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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 of the Gene Therapy CGF166 in Patients with Severe-to-Profound Hearing Loss

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Lawrence Lustig, MD
Status: Closed
The purpose of this research is to find out whether it is safe to use a virus to deliver a new gene to the inner ear of people with hearing loss. This is a kind of gene therapy which involves gene transfer. Once the genetic material and vector are surgically administered to you, it is not possible to remove these agents. The study is designed to evaluate…
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Off the shelf T-cells therapy for viral infection post HCST

Condition: Transplant / Stem Cell Transplant
Investigator: Prakash Satwani, MD
Status: Closed
In this trial, we will evaluate whether partially-HLA matched allogeneic multivirus-specific VSTs, will be safe and produce anti-viral effects in immunodeficient recipients infected with one of more of the targeted viruses that are persistent despite conventional anti-viral therapy. This will be in patients who have received a hematopoietic stem cell…
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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 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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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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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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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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STAR HCST for Less Severe Sickle Cell Disease (SCD)

Condition: Transplant / Stem Cell Transplant
Investigator: Monica Bhatia, MD
Status: Closed
The primary objectives of this study are to measure the safety and effectiveness of bone marrow transplant (BMT) using specific chemotherapy in children with less severe SCD and to understand the long term effects of bone marrow transplant in children with SCD, by evaluating ovarian reserve, sickle cell related brain problems, sickle cell related kidney…
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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 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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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 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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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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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 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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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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A study to Assess Safety and Effectiveness of Study Drug OTO-104 in Patients at Risk of Hearing Loss From Cisplatin Chemotherapy

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Alice Lee, MD
Status: Closed
Cisplatin is a chemotherapy medicine that is used to treat a variety of cancers. At high total (cumulative) doses it can be associated with progressive and irreversible hearing loss. The purpose of this study is see if injections of OTO-104 can protect against hearing loss associated with cisplatin therapy in the treatment of cancer. OTO-104 contains an…
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A study for patients with chronic Graft Versus Host Disease (cGVHD) using study drug Ibrutinib

Condition: Transplant / Stem Cell Transplant
Investigator: Ran Reshef, MD
Status: Closed
This is a research study of a new drug for the treatment of new onset chronic graft versus host disease (cGVHD). Chronic GVHD is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted donor cells attack the transplant recipient's body. The current way to treat cGVHD is to use prednisone (a type of…
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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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A study for patients with sickle cell disease receiving stem cell transplants

Condition: Transplant / Stem Cell Transplant
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to see whether or not the medicine, Abatacept, when given after hematopoietic stem cell transplantation (HSCT) is effective in treating GVHD in children and adolescents with sickle cell disease (SCD). We are also planning to gather additional information about the safety of treating sickle cell disease with a hematopoietic stem…
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A study for patients undergoing Hematopoietic Stem Cell Transplant using study drug Defibrotide

Condition: Transplant / Stem Cell Transplant
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to test a new medicine to prevent liver venoocclusive disease (VOD) in patients undergoing hematopoietic stem cell transplant (HSCT) who are at high risk or very high risk for liver VOD following HSCT. Liver VOD can be a complication of bone marrow and/or stem cell transplant,which may be life threatening. The condition is a…
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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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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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A study for pediatric patients with acute graft vs host disease using study drug Remestemcel-L

Condition: Transplant / Stem Cell Transplant
Investigator: Prakash Satwani, MD
Status: Closed
The main purpose of the study is to find out about whether the study drug is safe in humans and aneffective treatment in pediatric patients with acute Graft versus Host Disease (GVHD). GVHD mayoccur after a bone marrow or stem cell transplant in which someone receives bone marrow tissue orcells from a donor (called an allogeneic transplant). The new,…
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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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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 Adults with Sudden Sensorineural Hearing Loss Using the drug Am-111 to Evaluate its Effectiveness in Restoring Hearing Loss

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Anil Lalwani, MD
Status: Closed
Acute inner ear hearing loss is the sudden onset of hearing loss, usually in one ear, without any known cause. Acute hearing loss can permanently destroy some of the hair cells (that govern the sense of hearing) and / or hearing nerve in the inner ear.The drug AM-111 has been developed to protect the hearing cells and nerve following sudden inner ear…
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Study of Drug for the Treatment of Cytomegalovirus (CMV) Infection in Stem Cell Transplant Recipients

Condition: Transplant / Stem Cell Transplant
Investigator: Ran Reshef, MD
Status: Closed
The purpose of this study is to see which study drug is the better treatment for CMV infection. The study also wants to see how safe the study drugs are. The study drugs are Maribavir and Valganciclovir. Those who participate in the study will not know which drug they are taking. The study team will not know either.
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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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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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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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A study for stem cell transplant patients using study drug CSJ148

Condition: Transplant / Stem Cell Transplant
Investigator: Markus Mapara, MD
Status: Closed
The purpose of this study is to see if investigational drug CSJ148 is safe and if it can prevent HCMV infection during the first 98 days after stem cell transplant. Information about any side effect that the study drug can cause will also be collected.Study group will be made up of 6 patients. All patients in this group will receive the study drug, CSJ148.…
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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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