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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

2024-11-22

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