homocystinuria Clinical Trials in Philadelphia, Pennsylvania
2 recruitingPhiladelphia, Pennsylvania
Showing 1–2 of 2 trials
Recruiting
Phase 3
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
homocystinuria
Travere Therapeutics, Inc.70 enrolled52 locationsNCT06247085
Recruiting
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
Homocystinuria Due to CBS Deficiency
Travere Therapeutics, Inc.150 enrolled11 locationsNCT02998710