Birmingham Children´s Hospital NHS Foundation Trust

Birmingham B46NH

1 studies enrolling now · 16 studies all time

What they study most

Cystic Fibrosis, Eosinophilic Granulomatosis With Polyangiitis, Excessive Bleeding During Surgery, Haemophilia A, Hunter Syndrome, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy (MLD), Methylmalonic Acidemia, Microscopic Polyangiitis, Niemann-Pick Disease, Type C, Phenylketonuria, Polyarteritis Nodosa

Studies at this site

• Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients — Recruiting now
• Natural History Study of Patients With Succinic Semialdehyde Dehydrogenase (SSADH) Deficiency — Recruiting now
• An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705 — Recruiting now
• A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy — Completed
• A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria — Running, not enrolling
• Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older — Completed
• A Study to Evaluate the Efficacy and Safety of NexoBrid in Children With Thermal Burns Compared the Standard of Care — Completed
• Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities — Completed
• A Study of PTC923 in Participants With Phenylketonuria — Completed
• Pediatric Vasculitis Initiative — Status unconfirmed
• Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease — Stopped early
• A Study of Safety and Efficacy of Fibrin Sealant Grifols as an Adjunct to Haemostasis During Surgery in Paediatric Participants — Completed
• Study of Aztreonam for Inhalation in Children With Cystic Fibrosis and New Infection of the Airways by Pseudomonas Aeruginosa Bacteria — Stopped early
• A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase® — Completed
• Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) — Completed
• A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype — Completed