Dublin 12

• An experimental trial to learn about hu14.18K322A treatment in children and adolescents with high-risk neuroblastoma. Pending
  2025-524397-42-00 10 Participants with relapsed HRNB and participants with HRNB refractory
• A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People with Hemophilia A Recruiting
  2025-522435-33-00 5 2026-06-17 Hemophilia A
• HELIOS: Long-term study of DISC-1459 (or Bitopertin) in patients with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). Recruiting
  2025-523275-27-00 5 2026-05-26 X-Linked Protoporphyria (XLP)
• Vedolizumab Subcutaneous Long-term Open-label Extension Study in Pediatric Subjects Recruiting
  2023-508804-39-00 5 2025-10-15 Ulcerative Colitis or Crohn’s Disease
• RLY-2608-201-Ph2 Study of RLY-2608 in PROS and PIK3CA Driven Malformations Recruiting
  2024-518895-30-00 4 2025-11-21 PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA
• A Phase III study to evaluate the efficacy of INM004 (Shiga antitoxin) in pediatric patients with Shiga toxin-producing Escherichia coli-ass … Recruiting
  2024-512412-22-00 5 2025-06-24 Hemolytic Uremic Syndrome
• Long-term study evaluating joint health in people with haemophilia A receiving real-world prophylactic treatment with efanesoctocog alfa Recruiting
  2024-517812-31-00 6 2025-05-21 Haemophilia A
• A study of aficamten (CK-3773274) in a Pediatric Population with Symptomatic Obstructive Hypertrophic Cardiomyopathy Recruiting
  2024-511377-30-00 10 2025-01-23 SYMPTOMATIC OBSTRUCTIVE HYPERTROPHIC CARDIOMYOPATHY
• Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis Subjects 1 Through 11 Years of Age Recruiting
  2024-513754-29-00 5 2023-03-06 Cystic Fibrosis
• FaR-RMS: An overarching study for children and adults with Frontline and Relapsed RhabdoMyoSarcoma Recruiting
  2024-510579-40-00 9 2020-10-07 Rhabdoyosarcoma
• A Repeated Dose-finding Study of Sarilumab in Children and Adolescents with Systemic Juvenile Idiopathic Arthritis (sJIA) Recruiting
  2024-512701-11-00 4 2018-08-29 Systemic Juvenile Idiopathic Arthritis (sJIA)
• Paediatric Hepatic International Tumour Trial Recruiting
  2024-516110-38-00 5 2018-03-06 Hepatoblastoma and Hepatocellular Carcinoma
• SIOP Ependymoma II: An International Clinical Program for the diagnosis and treatment of children, adolescents and young adults with ependym … Recruiting
  2024-512222-28-00 10 2015-06-02 Newly diagnosed with an intracranial or spinal ependymoma (all WHO gra
• APOLLO: A Randomized, Double-Blind, Placebo-Controlled Study of Bitopertin to Evaluate the Efficacy, Safety, and Tolerability in Participant … Ended recruitment
  2024-520407-27-00 5 2025-10-23 X-Linked Protoporphyria (XLP)
• LOGGIC/FIREFLY-2: DAY101 vs. Standard of Care Chemotherapy in Pediatric Patients with Low-Grade Glioma Requiring First-Line Systemic Therapy Ended recruitment
  2024-510742-13-00 5 2023-04-19 Pediatric low-grade glioma harboring an activating RAF alteration requ
• A Phase 3 Study of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Ended recruitment
  2024-514173-22-00 5 2023-02-28 Cystic Fibrosis
• Myechild 01: International Randomised Phase III Clinical Trial in Children with Acute Myeloid Leukaemia - Incorporating an Embedded Dose Fin … Ended recruitment
  2024-516112-21-00 5 2017-11-20 Newly diagnosed acute myeloid leukaemia (AML), high risk myelodysplast
• Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or … Ended
  2023-508884-59-00 5 2024-06-03 Hemophilia A or Hemophilia B

Showing 1–18 of 18 trials