Homocystinuria

15 registered clinical trials studyying Homocystinuria — 3 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency) NCT06556615	University Children's Hospital, Zurich	—
Active Not Recruiting	Proof of Concept Creatine Supplementation for Homocystinuria Study NCT06495567	University of British Columbia	N/A
Enrolling By Invitation	A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in NCT06431893	Travere Therapeutics, Inc.	Phase 3
Active Not Recruiting	A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Y NCT06247085	Travere Therapeutics, Inc.	Phase 3
Unknown	Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan NCT05910151	West Kazakhstan Medical University	—
Completed	Baby Detect : Genomic Newborn Screening NCT05687474	Centre Hospitalier Universitaire de Liege	—
Completed	Safety, Tolerability and Pharmacodynamics of SYNB1353 in Healthy Adult Volunteers NCT05462132	Synlogic	Phase 1
Completed	Evaluation of the Express Plus Range NCT05051657	Vitaflo International, Ltd	N/A
Completed	Effects of Exercise on Metabolic Parameters in Classical Homocystinuria NCT04021732	University of Lausanne	—
Active Not Recruiting	Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Defi NCT03406611	Travere Therapeutics, Inc.	Phase 1 / Phase 2
Enrolling By Invitation	Early Check: Expanded Screening in Newborns NCT03655223	RTI International	—
Completed	Betaine METABOLISM OF PATIENTS With Homocystinuria NCT02404337	Assistance Publique - Hôpitaux de Paris	Phase 2
Completed	Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine NCT01192828	University of Colorado, Denver	Phase 1 / Phase 2
Completed	Homocystinuria: Treatment With N-Acetylcysteine NCT00483314	McGill University Health Centre/Research Institute of the McGill University Health Centre	Phase 2
Completed	Study of Homocysteine Metabolism in Homocystinuria NCT00004356	National Center for Research Resources (NCRR)	—