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

Part of paid clinical trials in Culver City, California.

Sponsor
Travere Therapeutics, Inc.
Study ID
NCT02998710
Status
Recruiting
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Conditions

  • Homocystinuria Due to CBS Deficiency

Eligibility Criteria

Sex
ALL
Age
1 Year - 65 Years
Healthy Volunteers
Not accepted

Study Details

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 Dates

Start date
Jan 31, 2017
Status verified
Nov 2024
Primary completion
Apr 30, 2026
Completion
Aug 31, 2026

Study Design

Enrollment
150 participants (estimated)

Primary Outcome Measure

Changes in Met cycle metabolites levels - tHcy [ Time Frame: 6.5 years ]

Central Contacts

Locations (7)

FacilityCityStateZIPSite coordinators
Travere Investigational Site - Virtual SiteCulver CityCalifornia90230-
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)AuroraColorado80045-
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)Washington D.C.District of Columbia20010-
Travere Investigational SiteAtlantaGeorgia30322-
Travere Investigational SiteIndianapolisIndiana46202-
Travere Investigational SiteBostonMassachusetts02115-
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)PhiladelphiaPennsylvania19104-

Find similar trials in Culver City, CA

By research site
Travere Investigational Site - Virtual Site· Culver City, CATravere Investigational Site (Enrolling 1 to <5 Year-olds Only)· Aurora, COTravere Investigational Site (Enrolling 1 to <5 Year-olds Only)· Washington D.C., DCTravere Investigational Site· Atlanta, GATravere Investigational Site· Indianapolis, INTravere Investigational Site· Boston, MA