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Displaying 1 - 9 of 9

Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies

Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study is for 1 to 21-year-olds whose hematologic cancer has relapsed to take medication called Tagraxofusp. Relapse means that cancer has come back after treatment. Tagraxofusp is a drug approved by the FDA (Food and Drug Administration) but is not approved to treat the cancers of people in this study. This study is being done to find out if…
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Pediatric Acute Leukemia (PedAL) Screening Trial - Developing New Therapies for Relapsed Leukemias (Children and Young Adults)

Condition: Cancer / 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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Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients (ages 18-21)

Condition: 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 Ibrutinib in Pediatric and Young Adult Patients With Relapsed or Refractory Mature B-cell non-Hodgkin Lymphoma (Ages 1-30)

Condition: Cancer / Lymphoma
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a 2-part, multicenter study of a study drug in pediatric and young adult patients with mature B-cell non-Hodgkin Lymphoma which has come back or has not responded to the standard therapy. The study treatment is considered an experimental because the study drug is not approved by the United States (US) Food and Drug Administration (FDA) for treating…
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Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia

Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to find the best dose of Asciminib that can be given safely in children with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) having previously been treated with one or more tyrosine kinase inhibitors (TKIs). Tyrosine kinase inhibitors are a group of medications that block chemical…
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Study of VX15/2508 in Children, Adolescents, or Young Adults with Recurrent or Relapsed Solid Tumors

Condition: Cancer / Solid Tumors
Investigator: Luca Szalontay, MD
Status: Closed
This is a Phase 1/2 multicenter study of VX15/2503 in pediatric patients and young adults with solid tumors which have come back or have not responded to standard therapy. The study treatment is considered experimental because VX15/2503 is not approved by the United States (US) Food and drug Administration (FDA) for treating pediatric patients and young…
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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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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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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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