Status: Recruiting
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices
Participants needed: 150
Trial details
Age: 1-65Biological sex: AllType: ObservationalSponsor: Travere Therapeutics, Inc.Updated: Nov 22, 2024Locations: 11
Eligibility criteria
Patients who are clinically diagnosed with homocystinuria [+3]
Medically significant postnatal complications or congenital anomalies that are n... [+1]