Clinical Trial

Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)

Recruiting
View on ClinicalTrials.gov →
Record status
This record was last updated November 22, 2024 (before its estimated April 2026 completion). Its status may not reflect the trial's current state.
Summary
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
Protocol Amendment History 21 amendments
This ClinicalTrials.gov record has been amended 21 times since 2016-12-16; most recent amendment 2024-11-19.
Status change: Not Yet Recruiting → Recruiting 2017-01-05
Trial Details
NCT Number NCT02998710
Lead Sponsor Travere Therapeutics, Inc.
Conditions Homocystinuria Due to CBS Deficiency
Enrollment 150 participants
Start Date 2017-01
Primary Completion 2026-04 (estimated)
Study Completion 2026-08 (estimated)
Updated on ClinicalTrials.gov 2024-11-22