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Displaying 1 - 15 of 15

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 C2 CryoBalloon Focal Ablation System for Patients with Barrett's Esophagus

Condition: Gastrointestinal and Digestive Diseases / Barrett's Esophagus
Investigator: Julian Abrams, MD
Status: Currently Recruiting
This research study will look at a newer treatment option for Barrett's Esophagus (BE) which uses nitrous oxide to freeze unwanted tissue. The most commonly used treatment method is Radiofrequency Ablation (RFA) which uses heat to destroy the pre-cancerous tissue. Nitrous Oxide ablation using the C2 CryoBalloon Focal Ablation System, an FDA-cleared…
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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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Eradicating Barrett's Esophagus Using Radiofrequency Ablation or a Novel Hybrid Argon Plasma Coagulation Technique (BURN)

Condition: Gastrointestinal and Digestive Diseases / Barrett's Esophagus
Investigator: Julian Abrams, MD
Status: Closed
The purpose of the study is to compare two types of endoscopic treatments in patients with Barretts esophagus (BE) with abnormal cells (dysplasia); radiofrequency ablation (RFA) and hybrid argon plasma coagulation (H-APC). H-APC is a newer technique in the treatment of BE and has not been adequately studied and is not approved by the FDA for routine use.…
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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 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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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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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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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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A study for patients with untreated dysplastic Barrett's Epithelium using the Coldplay CryoBallon Focal Ablation System

Condition: Gastrointestinal and Digestive Diseases / Barrett's Esophagus
Investigator: Julian Abrams, MD
Status: Closed
C2 Therapeutics is currently enrolling patients to participate in the Coldplay 3 clinical trial. Coldplay 3 is evaluating the effectiveness of the Coldplay CryoBalloon Focal Ablation System in patients with Barrett's esophagus who have not yet received treatment. Participants will undergo treatment with the Coldplay CryoBalloon Focal Ablation System…
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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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A study for patients with Barrett's esophagus using a Wide Area Transepithelial Sample Esophageal Biopsy (WATS) kits for analysis

Condition: Gastrointestinal and Digestive Diseases / Barrett's Esophagus
Investigator: Julian Abrams, MD
Status: Closed
The purpose of this study is to evaluate a new technique called Wide Area Transepithelial Sample 3-Dimensional Esophageal Biopsy (WATS) with computer assisted method of analysis of brush biopsy. This study will assess whether performing standard biopsies in addition to the WATS brush biopsies identifies more patients with dysplasia than does performing…
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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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