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Children's Hospital of the King's Daughters | Novel Therapeutics and Gene Therapy

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Cyclophosphamide
Etoposide
cyclophosphamide
Vincristine Sulfate
vincristine sulfate
Doxorubicin Hydrochloride
Carboplatin
Cytarabine
Methotrexate
etoposide

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Active trials

25 of 267 total trials

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD...

Enrolling
Duchenne Muscular Dystrophy
Genetic: placebo
Genetic: delandistrogene moxeparvovec

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study...

Active, not recruiting
Duchenne Muscular Dystrophy
Genetic: placebo
Genetic: delandistrogene moxeparvovec

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD....

Active, not recruiting
Muscular Dystrophy, Duchenne
Genetic: delandistrogene moxeparvovec

The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants w...

Invitation-only
Duchenne Muscular Dystrophy
Genetic: delandistrogene moxeparvovec

The main purpose of this study is to evaluate the long-term efficacy of mirikizumab in pediatric participants with ulcerative colitis (UC) or Crohn's...

Enrolling
Ulcerative Colitis Chronic
Ulcerative Colitis
Drug: Mirikizumab

This phase II trial studies the side effects and how well nirogacestat works in treating patients less than 18 years of age with desmoid tumors that...

Active, not recruiting
Desmoid Fibromatosis
Unresectable Desmoid Fibromatosis
Drug: Nirogacestat
Procedure: Magnetic Resonance Imaging

The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atr...

Active, not recruiting
Muscular Atrophy, Spinal
Drug: Nusinersen

The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy...

Enrolling
Spinal Muscular Atrophy
Drug: Nusinersen

This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of...

Active, not recruiting
Neuromuscular Diseases
SMA
Drug: Placebo
Drug: taldefgrobep alfa

This is a Phase 1-2, multicenter, international, single-arm, open-label study designed to identify a recommended dose of bosutinib administered orall...

Active, not recruiting
Blastic Phase Chronic Myelogenous Leukemia
Chronic Phase Chronic Myelogenous Leukemia
Drug: Bosutinib

The participants of this study will be children, adolescents, and young adults with residual osteosarcoma, which cannot be removed completely through...

Enrolling
Osteosarcoma
Osteosarcoma in Adolescents and Young Adults
Drug: Cabozantinib
Other: Best Supportive Care (BSC)

This Phase 3 trial (Study SRK-015-003) is being conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spina...

Active, not recruiting
Anti-myostatin
Neuromuscular Manifestations
Drug: Placebo
Drug: Apitegromab

Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study in combination with etoposide for subjects with relapsed/refractory...

Enrolling
Neuroblastoma
Drug: Eflornithine

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with on...

Active, not recruiting
Spinal Muscular Atrophy Type III
Spinal Muscular Atrophy Type I
Biological: Onasemnogene Abeparvovec-xioi

The purpose of this study is to test the long-term safety of rimegepant in the acute treatment of moderate or severe migraine in children and adolesc...

Enrolling
Acute Treatment of Migraine
Drug: Rimegepant / BHV3000

This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofi...

Active, not recruiting
Neurofibromatosis Type 1 (NF1)
Plexiform Neurofibroma
Drug: Mirdametinib (PD-0325901) oral capsule or dispersible tablet

A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with st...

Enrolling
Neuroblastoma
Drug: DFMO
Drug: vorinostat

This is a Phase 1/2a, open-label, single-dose, PK study of VALTOCO with open-label safety period in pediatric epilepsy subjects age 2 to 5 years.

Active, not recruiting
Epilepsy
Drug: Diazepam Nasal Spray [Valtoco]

The overall goal of this study is to determine the clinical benefit and safety of antiviral therapy for asymptomatic congenital cytomegalovirus (cCMV...

Active, not recruiting
SNHL
Cmv Congenital
Drug: Valganciclovir
Drug: Simple Syrup

The purpose of this study is to test the safety and efficacy of BHV-3000 versus placebo in the acute treatment of moderate or severe migraine in chil...

Enrolling
Pediatric Migraine
Drug: Rimegepant/BHV3000
Drug: Matching placebo

Trial sponsors

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