Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
Recruiting
Observational Study
Homocystinuria Due to CBS Deficiency
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 1 – 65
- Sex
- Any
- Study type
- Observational
- Participants needed
- 150 (estimated)
- Sponsor
- Travere Therapeutics, Inc. · Industry
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About This Trial
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
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* 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 Criteria:
* Medically significant postnatal…
Contacts