Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
NCT02998710 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 150
Last updated 2024-11-22
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
Conditions
- Homocystinuria Due to CBS Deficiency
Sponsors & Collaborators
-
Travere Therapeutics, Inc.
lead INDUSTRY
Principal Investigators
-
Michael Imperiale, MD · Travere Therapeutics, Inc.
Eligibility
- Min Age
- 1 Year
- Max Age
- 65 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2017-01-31
- Primary Completion
- 2026-04-30
- Completion
- 2026-08-31
Countries
- United States
- Ireland
- Qatar
- United Kingdom
Study Locations
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