Dublin 1

• A study to investigate efficacy and safety with pegtibatinase compared to placebo in participants ≥12 to ≤65 years of age with classical hom … Pending
  2023-504135-40-00 5 Classical Homocystinuria
• ACUMEN: Acute High Dose Melatonin for Encephalopathy of the Newborn Pending
  2025-520538-49-00 1 Moderate-Severe Hypoxic-Ischaemic Encephalopathy (HIE)
• A Phase 2 Clinical Trial to Investigate Safety, Tolerability and Efficacy of TransCon CNP, Weekly Subcutaneous Injections, Compared with Pla … Recruiting
  2023-506091-27-00 4 2024-09-19 Achondroplasia in Children and Adolescents
• A Phase 3, Open-Label, Uncontrolled Study to Evaluate the Activity, Safety, Pharmacokinetics and Pharmacodynamics of Roxadustat for the Trea … Recruiting
  2022-501980-42-00 5 2023-12-21 Anemia associated with Chronic Kidney Disease
• A study of sepiapterin in participants with phenylketonuria (PKU) (EPIPHENY) Recruiting
  2024-514435-20-00 5 2025-12-23 Phenylketonuria
• A Phase 3 study of Omaveloxolone in participants with Friedreich’s Ataxia Ages 2 < 16 years Recruiting
  2025-520896-13-00 5 2025-12-04 Friedreich’s ataxia
• 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
• Efficacy and Safety of Navepegritide in Adolescents with Achondroplasia Recruiting
  2024-514208-15-00 4 2024-12-10 Achondroplasia in Children and Adolescents
• A study to investigate efficacy, safety, and tolerability of mavorixafor in participants with congenital and acquired primary autoimmune and … Recruiting
  2023-508482-32-00 5 2024-07-22 Neutropenias
• A Phase 3 Study of Nipocalimab in Pregnancies at Risk for Severe Hemolytic Disease of the Fetus and Newborn (HDFN) Recruiting
  2022-502629-16-00 5 2024-07-10 Hemolytic Disease of the Fetus and Newborn (HDFN)
• AttaCH: A Phase 2, Multicenter, Long-Term, Open Label Extension Trial Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of … Recruiting
  2022-502202-33-00 4 2023-07-06 Achondroplasia in Children and Adolescents
• HYPATIA: A prospective randomised controlled trial of HYdroxychloroquine to improve Pregnancy outcome in women with AnTIphospholipid Antibod … Recruiting
  2023-504661-23-00 5 2023-06-27 Antiphospholipid antibodies
• An open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dy … Ended recruitment
  2024-512828-12-00 6 2024-12-05 Duchenne Muscular Dystrophy
• COACH: A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepe … Ended recruitment
  2023-508341-40-00 4 2024-07-26 Children with Achondroplasia
• A Study to Examine the Efficacy and Safety of ZX008 in Subjects with CDKL5 Deficiency Disorder Ended recruitment
  2023-506269-78-00 5 2023-06-20 CDKL5 Deficiency disorder
• 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
• Study of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Ended recruitment
  2023-510351-31-00 7 2023-01-06 Duchenne muscular dystrophy
• Co-administration of Acetaminophen with Ibuprofen to Improve Duct-Related Outcomes in Extremely Premature Infants – The ACEDUCT Trial Ended
  2023-503209-13-00 4 2024-08-07 Patent ductus arteriosus (PDA)
• A phase 2b clinical trial to evaluate efficacy and safety of weekly doses of TransCon CNP compared with placebo in participants with achondr … Ended
  2024-515469-32-00 4 2023-03-02 Achondroplasia in Children and Adolescents
• A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients Ended
  2024-514698-23-00 5 2017-05-15 Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Sk

Showing 1–20 of 20 trials