Actively Recruiting
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
Led by Travere Therapeutics, Inc. · Updated on 2024-11-22
150
Participants Needed
11
Research Sites
499 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinically diagnosed with homocystinuria
- Male or female patients aged 1 to 65 years
- Provided consent and/or assent
- Willing and able to comply with all study procedures
You will not qualify if you...
- Medically significant postnatal complications or congenital anomalies not related to homocystinuria
- Received experimental therapy for homocystinuria within 6 months before enrollment or expected to receive such therapy during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 11 locations
1
Travere Investigational Site - Virtual Site
Culver City, California, United States, 90230
Completed
2
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
4
Travere Investigational Site
Atlanta, Georgia, United States, 30322
Completed
5
Travere Investigational Site
Indianapolis, Indiana, United States, 46202
Completed
6
Travere Investigational Site
Boston, Massachusetts, United States, 02115
Completed
7
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
Travere Investigational Site
Dublin, Ireland, 1
Actively Recruiting
9
Travere Investigational Site
Doha, Qatar, Qatar
Actively Recruiting
10
Travere Investigational Site
Doha, Qatar
Actively Recruiting
11
Travere Investigational Site
Salford, Manchester, United Kingdom, M6 8HD
Completed
Research Team
T
Travere Call Center
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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