Current Environment:

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

Conditions

Homocystinuria Due to CBS Deficiency

Recruitment Status

Recruiting

Eligibility Criteria

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 Criteria:

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

Gender

All

Min Age

1 Year

Max Age

65 Years

Download Date

April 9, 2024

Principal Investigator

N/A

Primary Contact Information

Travere Call Center

1-877-659-5518

medinfo@travere.com

For more information on this trial, visit clinicaltrials.gov.

Contact

For more information and to contact the study team:

Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA) NCT02998710 Travere Call Center 1-877-659-5518 medinfo@travere.com