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RecruitingNCT02998710

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

A Multicenter, Observational, Prospective, Natural History Study of Homocystinuria Due to Cystathionine Beta-synthase Deficiency in Pediatric and Adult Patients (ACAPPELLA)

Status
Recruiting
Phase
Study type
Observational
Enrollment
150 (estimated)
Sponsor
Travere Therapeutics, Inc. · Industry
Sex
All
Age
1 Year – 65 Years
Healthy volunteers
Not accepted

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

Timeline

Start date
2017-01-01
Primary completion
2026-04-01
Completion
2026-08-01
First posted
2016-12-20
Last updated
2024-11-22

Locations

11 sites across 4 countries: United States, Ireland, Qatar, United Kingdom

Source: ClinicalTrials.gov record NCT02998710. Inclusion in this directory is not an endorsement.

Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA) (NCT02998710) · Clinical Trials Directory