NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

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

CompletedN/A

Sponsor: Travere Therapeutics, Inc.

Conditions: Homocystinuria Due to CBS Deficiency

Countries: United States, Ireland, Qatar, United Kingdom

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 overview

Sex: ALL

Age: 1 Year to 65 Years

Healthy volunteers: No

Study type: OBSERVATIONAL

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 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

Locations (11)

Aurora, Colorado, United States
Washington D.C., District of Columbia, United States
Atlanta, Georgia, United States
Indianapolis, Indiana, United States
Boston, Massachusetts, United States
Morrisville, North Carolina, United States
Philadelphia, Pennsylvania, United States
Dublin, Ireland
Doha, Qatar, Qatar
Doha, Qatar
Salford, Manchester, United Kingdom