Homocystinuria due to CBS deficiency

Clinical trials for Homocystinuria due to CBS deficiency explained in plain language.

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Completed 1

A multicenter, observational, prospective, natural history study of homocystinuria due to cystathionine beta-synthase deficiency in pediatric and adult patients (ACAPPELLA)

Completed

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

Matched conditions: HOMOCYSTINURIA DUE TO CBS DEFICIENCY

Sponsor: Travere Therapeutics, Inc.

Last updated Sep 09, 2026 17:00 UTC

Homocystinuria due to CBS deficiency

Get alerted when new Homocystinuria due to CBS deficiency trials appear

A multicenter, observational, prospective, natural history study of homocystinuria due to cystathionine beta-synthase deficiency in pediatric and adult patients (ACAPPELLA)