Great Ormond Street hospital

London WC1N3JH

18 studies enrolling now · 66 studies all time

What they study most

ALK Fusion-positive Solid or CNS Tumors, AOSD, APDS, Adrenoleukodystrophy (ALD), Aicardi-Goutières Syndrome (AGS), Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Anaplastic Astrocytoma, Arginase I Deficiency, Arthritis, Juvenile, Arthritis, Psoriatic, Astrocytoma

Studies at this site

• A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids) — Recruiting now
• A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors — Recruiting now
• Obe-cel in Severe, Refractory Systemic Lupus Erythematosus (SLE) With Active Lupus Nephritis (LN) — Recruiting now
• A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome — Recruiting now
• EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety — Recruiting now
• A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease — Recruiting now
• A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) — Recruiting now
• A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's Disease — Recruiting now
• Study of Olutasidenib and Temozolomide in HGG — Recruiting now
• Targeted Pediatric High-Grade Glioma Therapy — Recruiting now
• Study of Ribociclib and Everolimus in HGG and DIPG or Ribociclib and Temozolomide in DHG, H3G34-mutant — Recruiting now
• A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, and Antiviral Activity of Maribavir for the Treatment of Cytomegalovirus (CMV) Infection in Children and Adolescents Who Have Received a Hematopoietic Stem Cell Transplant (HSCT) or a Solid Organ Transplant (SOT) — Recruiting now
• An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency — Recruiting now
• A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C — Recruiting now
• Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD). — Recruiting now
• Quality of Pediatric Resuscitation in a Multicenter Collaborative — Recruiting now
• Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations — Running, not enrolling
• A Study of Ustekinumab or Guselkumab in Pediatric Participants With Active Juvenile Psoriatic Arthritis — Completed
• A Long-term Extension Study of Ustekinumab in Pediatric Participants — Running, not enrolling
• Adalimumab Dose Adjustment for Pediatric Uveitis Treatment Trial — Starting soon
• An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment — Running, not enrolling
• A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension — Running, not enrolling
• Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis — Running, not enrolling
• A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria — Running, not enrolling
• Pediatric Patients Aged 1 to 6 Years With APDS — Running, not enrolling
• An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 — Enrolling by invitation
• Hemofiltration for Respiratory Failure After Bone Marrow Transplantation — Completed
• COMparison Between Anakinra and Tocilizumab in NORSE - "COMBAT-NORSE" — Starting soon
• Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE — Completed
• 24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG — Completed
• Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping — Running, not enrolling
• An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism — Completed
• A Study to Evaluate the Efficacy and Safety of Daratumumab in Pediatric and Young Adult Participants Greater Than or Equal to (>=)1 and Less Than or Equal to (<=) 30 Years of Age With Relapsed/Refractory Precursor B-cell or T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma — Completed
• Adalimumab in JIA-associated Uveitis Stopping Trial — Completed
• Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product — Running, not enrolling
• Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes — APPROVED_FOR_MARKETING
• Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C — Completed
• Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH) — Completed
• Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis — Completed
• A Study of PTC923 in Participants With Phenylketonuria — Completed
• N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) — Completed
• Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease — Stopped early
• Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies — Completed
• A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency — Completed
• A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency — Stopped early
• Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa — Completed
• A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) — Completed
• VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease — Completed
• A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM) — Completed
• A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects — Completed