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

Last updated: November 19, 2024
Sponsor: Travere Therapeutics, Inc.
Overall Status: Active - Recruiting

Phase

N/A

Condition

N/A

Treatment

N/A

Clinical Study ID

NCT02998710
CBS-HCY-NHS-01
  • Ages 1-65
  • All Genders

Study 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 Criteria

Inclusion

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

Exclusion Criteria:

  • Medically significant postnatal complications or congenital anomalies that are notassociated with homocystinuria

  • Received any experimental therapy for homocystinuria during the 6 months prior toenrollment or expected to receive any such therapy during duration of the study

Study Design

Total Participants: 150
Study Start date:
January 01, 2017
Estimated Completion Date:
August 31, 2026

Connect with a study center

  • Travere Investigational Site

    Dublin, 1
    Ireland

    Active - Recruiting

  • Travere Investigational Site

    Doha,
    Qatar

    Active - Recruiting

  • Travere Investigational Site

    Salford, Manchester M6 8HD
    United Kingdom

    Completed

  • Great Ormond Street Hospital NHS Foundation Trust

    London, WC1N 3JH
    United Kingdom

    Site Not Available

  • Travere Investigational Site

    Culver City, California 90230
    United States

    Active - Recruiting

  • Travere Investigational Site - Virtual Site

    Culver City, California 90230
    United States

    Completed

  • Travere Investigational Site

    Aurora, Colorado 80045
    United States

    Active - Recruiting

  • Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)

    Aurora, Colorado 80045
    United States

    Active - Recruiting

  • Travere Investigational Site

    Washington, District of Columbia 20010
    United States

    Active - Recruiting

  • Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)

    Washington, District of Columbia 20010
    United States

    Active - Recruiting

  • Travere Investigational Site

    Atlanta, Georgia 30322
    United States

    Completed

  • Travere Investigational Site

    Decatur, Georgia 30033
    United States

    Site Not Available

  • Travere Investigational Site

    Indianapolis, Indiana 46202
    United States

    Completed

  • Travere Investigational Site

    Boston, Massachusetts 02115
    United States

    Completed

  • Travere Investigational Site

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

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