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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age1-65
SponsorTravere Therapeutics, Inc.

About this trial

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

Eligibility criteria

Qualifiers

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.

Disqualifiers

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

Trial design

Treatments tested in this trial

  • Not listed

Trial groups

No trial groups listed

Sponsors and collaborators