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)
Sponsor
Travere Therapeutics, Inc.
Enrollment
150 participants
Start Date
Jan 1, 2017
Study Type
OBSERVATIONAL
Conditions
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
Eligibility
Min Age: 1 YearMax Age: 65 Years
Inclusion Criteria4
- Patients who are clinically diagnosed with homocystinuria
- Male/female patients aged 1 to 65 years
- Patients who consented and/or assented
- Patients who are willing and able to comply with all study-related procedures.
Exclusion Criteria2
- Medically significant postnatal complications or congenital anomalies that are not associated with homocystinuria
- Received any experimental therapy for homocystinuria during the 6 months prior to enrollment or expected to receive any such therapy during duration of the study
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Locations(11)
View Full Details on ClinicalTrials.gov
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NCT02998710