RECRUITING

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

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Conditions

Homocystinuria Due to CBS Deficiency

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

A Multicenter, Observational, Prospective, Natural History Study of Homocystinuria Due to Cystathionine Beta-synthase Deficiency in Pediatric and Adult Patients (ACAPPELLA)

Quick Facts

Study Start:2017-01
Study Completion:2026-08
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT02998710

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Year to 65 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Patients who are clinically diagnosed with homocystinuria
  2. * Male/female patients aged 1 to 65 years
  3. * Patients who consented and/or assented
  4. * Patients who are willing and able to comply with all study-related procedures.
  1. * Medically significant postnatal complications or congenital anomalies that are not associated with homocystinuria
  2. * 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

Contacts and Locations

Study Contact

Travere Call Center
CONTACT
1-877-659-5518
medinfo@travere.com

Principal Investigator

Michael Imperiale, MD
STUDY_DIRECTOR
Travere Therapeutics, Inc.

Study Locations (Sites)

Travere Investigational Site - Virtual Site
Culver City, California, 90230
United States
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Aurora, Colorado, 80045
United States
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Washington, District of Columbia, 20010
United States
Travere Investigational Site
Atlanta, Georgia, 30322
United States
Travere Investigational Site
Indianapolis, Indiana, 46202
United States
Travere Investigational Site
Boston, Massachusetts, 02115
United States
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Philadelphia, Pennsylvania, 19104
United States

Collaborators and Investigators

Sponsor: Travere Therapeutics, Inc.

  • Michael Imperiale, MD, STUDY_DIRECTOR, Travere Therapeutics, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2017-01
Study Completion Date2026-08

Study Record Updates

Study Start Date2017-01
Study Completion Date2026-08

Terms related to this study

Additional Relevant MeSH Terms

  • Homocystinuria Due to CBS Deficiency