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

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Top conditions

Top treatments

Nivolumab
Cyclophosphamide
Selumetinib
Prednisone
Erdafitinib
Vincristine Sulfate
Doxorubicin Hydrochloride
Palbociclib
Ulixertinib
Larotrectinib Sulfate

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This site is a part of Children's Hospital of the King's Daughters

Data sourced from clinicaltrials.gov

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Active trials

5 of 44 total trials

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (EXPEDITION)

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 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

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

The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDB...

Invitation-only
Duchenne Muscular Dystrophy
Drug: Ataluren

A study to learn about safety and find out maximum tolerable dose of palbociclib given in combination with chemotherapy (temozolomide with irinotecan...

Active, not recruiting
Neuroblastoma
Rhabdomyosarcoma
Drug: Cyclophosphamide
Drug: Temozolomide

Trial sponsors

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