Great Ormond Street Hospital NHS Foundation Trust

The trials here

What the 8 trials at this site are about, from their registry records.

Conditions

• Duchenne muscular dystrophy3 trials
• Epilepsy2 trials
• ANCA-associated vasculitis1 trial
• Dilated cardiomyopathy1 trial
• Giant cell arteritis1 trial
• Pompe disease1 trial

Phase

• Phase 23 trials
• Phase 1/21 trial

Status

• Active, not recruiting2 trials
• Recruiting2 trials
• Completed1 trial
• Enrolling by invitation1 trial
• Not yet recruiting1 trial
• Terminated1 trial

Lead sponsor

• BioMarin Pharmaceutical2 trials
• University College, London2 trials
• Amicus Therapeutics1 trial
• Cambridge University Hospitals NHS Foundation Trust1 trial
• Institute of Child Health1 trial
• and 1 more

Running trials

• Enrolling by invitation

  An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy

  NCT07573631· Phase 2· BioMarin Pharmaceutical· Source

• Recruiting

  The Children's Adaptive Deep Brain Stimulation for Epilepsy Trial

  NCT06924086· University College, London· Source

  Contact: Rory Piper, MRCS

• Not yet recruiting

  Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age

  NCT06643442· Phase 2· Sebastiano Lava· Source

• Recruiting

  A Global Prospective Observational Registry of Patients With Pompe Disease

  NCT06121011· Amicus Therapeutics· Source

  Contact: For Site

• Active, not recruiting

  A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

  NCT06280209· Phase 1/2· BioMarin Pharmaceutical· Source

• Active, not recruiting

  UNICORNS: Uveitis in Childhood Prospective National Cohort Study

  NCT05385757· Institute of Child Health· Source

Recruiting at the same time

How many trials recruit for the same indication here at once. A count, not a rating.

• Duchenne muscular dystrophy2 recruiting
• Dilated cardiomyopathy1 recruiting
• Epilepsy1 recruiting
• Pompe disease1 recruiting

Closed trials

• Completed

  Children's Adaptive Deep Brain Stimulation for Epilepsy Trial (CADET): Pilot

  NCT05437393· University College, London· Source

• Terminated

  Biologics in Refractory Vasculitis: A Trial of Biologic Therapy for Refractory Primary Non-ANCA Associated Vasculitis

  NCT05168475· Phase 2· Cambridge University Hospitals NHS Foundation Trust· Source