Great Ormond Street Hospital for Children

London WCIN 3JH

18 studies enrolling now · 55 studies all time

What they study most

Acute Graft-versus-host Disease, Acute Respiratory Distress Syndrome, Adenovirus, Adenovirus Infection, Adenovirus Infections, Adult Onset Still Disease, Angelman Syndrome, Anti-myostatin, Arginase I Deficiency, Arthritis, Juvenile, Atrophy, BK Virus Infection

Studies at this site

• A Phase 3 Trial to Compare IV BCV Versus IV CDV for Treatment of Adenovirus Infection After Allo-HCT — Recruiting now
• A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Obinutuzumab in Adolescents With Active Class III or IV Lupus Nephritis and the Safety and PK of Obinutuzumab in Pediatric Participants — Recruiting now
• A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome — Recruiting now
• A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy — Recruiting now
• Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease — Recruiting now
• A Study of Eptinezumab in Pediatric Participants With Episodic Migraine — Recruiting now
• A Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD) — Recruiting now
• A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy — Recruiting now
• A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome — Recruiting now
• A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study) — Recruiting now
• International Study of Cerebral Oxygenation and Electrical Activity During Major Neonatal Surgery — Recruiting now
• A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM). — Recruiting now
• Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease — Recruiting now
• A Study of the Efficacy and Safety of DMX-200 in Patients With FSGS Who Are Receiving an ARB — Recruiting now
• Simple Bone Cysts in Kids — Recruiting now
• Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients — Enrolling by invitation
• Long-term Extension of GTX-102 in Angelman Syndrome — Enrolling by invitation
• Efficacy and Safety of Erenumab in Pediatric Participants With Episodic Migraine — Running, not enrolling
• ARDS in Children and ECMO Initiation Strategies Impact on Neurodevelopment (ASCEND) — Running, not enrolling
• A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB) — Starting soon
• Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) — Running, not enrolling
• A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors — Running, not enrolling
• Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors — Running, not enrolling
• A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy — Running, not enrolling
• Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age — Running, not enrolling
• Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older — Enrolling by invitation
• A Long-term Follow-up Study in Participants Who Received CTX001 — Enrolling by invitation
• A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity — Completed
• A Long-Term Study of Bivamelagon in Participants With Hypothalamic Obesity (HO) — Enrolling by invitation
• Efficacy and Safety of Erenumab in Pediatric Subjects With Chronic Migraine — Completed
• A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling — Completed
• A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome — Completed
• Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF) — Completed
• Safety and Efficacy of Itacitinib in Combination With Corticosteroids for Treatment of Graft-Versus-Host Disease in Pediatric Subjects — Stopped early
• Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I) — Running, not enrolling
• Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab — Running, not enrolling
• Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency — Completed
• Tamoxifen Therapy for Myotubular Myopathy — Stopped early
• Gene Therapy Study for Children With CLN5 Batten Disease — Running, not enrolling
• Study of Posoleucel (ALVR105,Viralym-M) for Multi-Virus Prevention in Patients Post-Allogeneic Hematopoietic Cell Transplant — Stopped early
• Study to Evaluate Viralym-M (ALVR105) for the Treatment of Virus-Associated Hemorrhagic Cystitis (HC) — Stopped early
• Posoleucel (ALVR105) for the Treatment of Adenovirus Infection in Pediatric and Adult Participants Receiving Standard of Care Following Allogeneic Hematopoietic Cell Transplantation — Stopped early
• Glycosade v UCCS in the Dietary Management of Hepatic GSD — Completed
• A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH) — Completed
• Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) — Stopped early
• A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids — Stopped early
• A Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome — Completed
• BAX 855 Pediatric Study — Completed
• Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) — Completed
• Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B — Stopped early