Hospital Sant Joande Déu

Barcelona 8950

8 studies enrolling now · 30 studies all time

What they study most

AOSD, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Alpers Disease, Alpers Syndrome, Anti-myostatin, Atrophy, Autosomal Recessive Hypophosphatemic Rickets, Becker Muscular Dystrophy, CNS Metastases, Dravet Syndrome, Drug Resistant Epilepsy, Duchenne Muscular Dystrophy

Studies at this site

• A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment — Recruiting now
• A Study to Find Out if BI 764198 Helps Adults and Adolescents With a Kidney Condition Called Focal Segmental Glomerulosclerosis (FSGS) — Recruiting now
• Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B — Recruiting now
• The NODE-202 Study (Study of Etripamil Nasal Spray in Pediatric Patients) — Recruiting now
• The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency — Recruiting now
• EFESO: Study on Juvenile Onset Eosinophilic Fasciitis — 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
• Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow — Running, not enrolling
• Natural History Study Protocol in PMM2-CDG (CDG-Ia) — Completed
• A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) — Running, not enrolling
• A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE) — Enrolling by invitation
• Study Evaluating Brexucabtagene Autoleucel (KTE-X19) in Pediatric and Adolescent Participants With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma — Completed
• Pivotal-Safety and Therapeutic Measures of tDCS in Patients With Refractory Focal Epilepsy — Completed
• A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS) — Running, not enrolling
• A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy — Stopped early
• Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE — Completed
• Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam — Completed
• An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects — Enrolling by invitation
• A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease — Completed
• 24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG — Completed
• Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy — Stopped early
• An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome — Completed
• Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy — Completed
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) — Completed
• An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy — Completed
• 131I-omburtamab Radioimmunotherapy for Neuroblastoma Central Nervous System/Leptomeningeal Metastases — Stopped early
• A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) in Children and Young Adults With Dravet Syndrome — Completed
• Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa — Completed
• Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis — Completed
• Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) — Completed