Esplugues De Llobregat

• A Study to Determine the Effect of Triheptanoin Compared with Even-chain MCT on MCEs in Pediatric Patients with LC-FAOD Ended recruitment
  2023-509809-76-00 5 2023-02-28 Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
• 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
• A study to test Taldefgropeb Alfa in patients with Spinal Muscular Atrophy. Ended recruitment
  2024-511852-42-00 5 2023-01-19 Spinal Muscular Atrophy
• 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
• Towards a treatment for accelerated maturation in girls testing spiomet. Ended recruitment
  2024-518675-55-00 4 2022-12-02 Girls with advanced puberty and accelerated bone maturation. Polycysti
• Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor Long-term Safety and Efficacy in Subjects Without F508del Ended recruitment
  2024-515637-14-00 5 2022-11-08 Cystic Fibrosis
• A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria Ended recruitment
  2023-509229-31-00 5 2022-09-28 Metabolic Disorders - Phenylketonuria
• A research study to compare somapacitan once a week with Norditropin® once a day in children who need help to grow Ended recruitment
  2023-506927-27-00 5 2022-08-29 Children with short stature either born small for gestational age or w
• A study to evaluate the efficacy and safety of treatment with JR-141 Ended recruitment
  2024-512289-33-00 5 2022-08-24 Mucopolysaccharidosis type II
• Study of setmelanotide in patients with obesity with specific genetic variants Ended recruitment
  2023-507634-24-00 5 2022-05-20 Improper function of certain messenger materials in the body that cont
• A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular A … Ended recruitment
  2023-506761-65-00 10 2022-04-08 Spinal Muscular Atrophy (SMA)
• Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children aged ≥ 1 to < 7 Years with Neurofibromatosis Type 1 … Ended recruitment
  2023-506357-38-00 9 2022-02-17 Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN)
• A Study to Evaluate Safety and Efficacy of Ocrelizumab in Comparison with Fingolimod in Children and Adolescents with Relapsing-Remitting Mu … Ended recruitment
  2023-506516-40-00 5 2022-02-11 Relapsing-Remitting Multiple Sclerosis
• A study to learn how well finerenone works; how safe it is; how it moves into, through, and out of the body; and the effects it has on the b … Ended recruitment
  2023-504884-17-00 5 2022-02-04 Proteinuria
• A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscu … Ended recruitment
  2024-511492-15-00 5 2021-12-09 Duchenne Muscular Dystrophy
• Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen … Ended recruitment
  2023-505637-27-00 5 2021-07-12 Spinal Muscular Atrophy
• A research study to find out if the study treatment alpelisib (BYL719) is safe and can help others who have confirmed diagnosis of PIK3CA-re … Ended recruitment
  2023-508530-34-00 4 2021-04-19 PIK3CA-Related Overgrowth Spectrum (PROS)
• Tumor-Agnostic Precision Immuno-Oncology and Somatic Targeting Rational for You (TAPISTRY) Platform Study Ended recruitment
  2023-507418-28-00 4 2021-03-15 Solid tumors
• A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 … Ended recruitment
  2023-505964-13-00 5 2021-03-09 Hemophilia A
• A Study of Ixekizumab (LY2439821) in Children With Juvenile Idiopathic Arthritis Categories of Enthesitis-related Arthritis (Including Juven … Ended recruitment
  2023-507184-19-00 5 2021-02-22 Juvenile Psoriatic Arthritis  Enthesitis Related Arthritis
• Parent and/or Legal Guardian ICF and Adult patient ICF: A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Parallel-Group Study wi … Ended recruitment
  2022-501012-34-00 5 2020-02-27 Pulmonary Arterial Hypertension
• Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have … Ended recruitment
  2023-505088-35-00 9 2019-12-12 Pediatric Solid tumors and primary central nervous system (CNS) tumors
• AN OPEN-LABEL, MULTICENTER STUDY IN MALE PEDIATRIC PATIENTS WITH CEREBRAL X-LINKED ADRENOLEUKODYSTROPHY (CALD) TO ASSESS THE EFFECTS OF MIN- … Ended recruitment
  2024-513774-21-00 4 2019-09-13 CEREBRAL X-LINKED ADRENOLEUKODYSTROPHY (CALD)
• A study to learn if combination pyrimidine nucleosides is safe, how well it works and how it moves through the bloodstream in participants w … Ended recruitment
  2023-510427-29-00 4 2019-09-04 Thymidine kinase 2 deficiency

Showing 217–240 of 317 trials