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

Recruiting now

Conditions studied: Homocystinuria Due to CBS Deficiency

In brief

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

Key facts

Study ID

NCT02998710

Run by

Travere Therapeutics, Inc.

People needed

150

Starts

2017-01-01

Expected to finish

2026-08-01

Last updated by the study team

2024-11-22

Who can join

Age: 1 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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

Where it is running

• Travere Investigational Site (Enrolling 1 to <5 Year-olds Only) — Philadelphia, Pennsylvania, United States (enrolling)
• Travere Investigational Site — Dublin, Ireland (enrolling)
• Travere Investigational Site — Doha, Qatar, Qatar (enrolling)
• Travere Investigational Site — Doha, Qatar (enrolling)
• Travere Investigational Site (Enrolling 1 to <5 Year-olds Only) — Washington D.C., District of Columbia, United States (enrolling)
• Travere Investigational Site (Enrolling 1 to <5 Year-olds Only) — Aurora, Colorado, United States (enrolling)
• Travere Investigational Site — Salford, Manchester, United Kingdom
• Travere Investigational Site — Atlanta, Georgia, United States
• Travere Investigational Site — Indianapolis, Indiana, United States
• Travere Investigational Site — Boston, Massachusetts, United States
• Travere Investigational Site - Virtual Site — Culver City, California, United States

Full record on ClinicalTrials.gov

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