Current Environment:


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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


Homocystinuria Due to CBS Deficiency

Recruitment Status


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



Min Age

1 Year

Max Age

65 Years

Download Date

April 9, 2024

Principal Investigator


Primary Contact Information

Travere Call Center


For more information on this trial, visit


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