Clinical trials for Homocystinuria
6 recruiting or active trials synced from ClinicalTrials.gov.
ℹ
Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- RECRUITINGNCT06556615Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency)University Children's Hospital, Zurich
- ACTIVE NOT RECRUITINGN/ANCT06495567Proof of Concept Creatine Supplementation for Homocystinuria StudyUniversity of British Columbia · PI: Rajavel Elango, PhD
- ENROLLING BY INVITATIONPhase 3NCT06431893A Phase 3 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)Travere Therapeutics, Inc. · PI: Michael Imperiale, MD
- ACTIVE NOT RECRUITINGPhase 3NCT06247085A 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 TreatmentTravere Therapeutics, Inc. · PI: Michael Imperiale, MD
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT03406611Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)Travere Therapeutics, Inc. · PI: Michael Imperiale, MD
- RECRUITINGNCT02998710Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)Travere Therapeutics, Inc. · PI: Michael Imperiale, MD