Birmingham Womens and Childrens NHS Foundation Trust

Birmingham B5 6NH

8 studies enrolling now · 18 studies all time

What they study most

Achondroplasia, Alagille Syndrome, Atrial Flutter, Cystic Fibrosis, Fetal Hydrops, Fucosidosis, Homocystinuria, Hypothalamic Obesity, Intrahepatic Cholestasis of Pregnancy, Mucopolysaccharidosis II, PFIC1, PFIC2

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 Long-Term Observational Study of Patients With Fucosidosis — Recruiting now
• A Study of Sepiapterin in Participants With Phenylketonuria (PKU) — Recruiting now
• A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) — Recruiting now
• Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome — Running, not enrolling
• An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 — Enrolling by invitation
• 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
• Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC — Completed
• A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG — Running, not enrolling
• A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity — Completed
• A Long-Term Study of Bivamelagon in Participants With Hypothalamic Obesity (HO) — Enrolling by invitation
• This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2 — Completed
• Extension Study of Infigratinib in Children With Achondroplasia (ACH) — Enrolling by invitation
• BlueWind RENOVA iStim™ System for the Treatment of OAB — Completed
• Prospective Observational Cohort Study of Fetal Atrial Flutter & Supraventricular Tachycardia — Completed
• A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA) — Stopped early
• Study Evaluating Betrixaban in Pediatric Participants — Stopped early
• Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis — Completed