CLASSICAL HOMOCYSTINURIA DUE TO CYSTATHIONINE BETA SYNTHASE DEFICIENCY

Clinical trials for CLASSICAL HOMOCYSTINURIA DUE TO CYSTATHIONINE BETA SYNTHASE DEFICIENCY explained in plain language.

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New drug pegtibatinase tested for rare metabolic disorder over two years

Disease control ENROLLING_BY_INVITATION

This study tests the long-term safety and effectiveness of pegtibatinase in people with classical homocystinuria (HCU), a rare genetic disorder that prevents the body from breaking down certain amino acids. About 100 participants who completed earlier studies will receive the dru…

Matched conditions: CLASSICAL HOMOCYSTINURIA DUE TO CYSTATHIONINE BETA SYNTHASE DEFICIENCY

Phase: PHASE3 • Sponsor: Travere Therapeutics, Inc. • Aim: Disease control

Last updated Jun 27, 2026 12:38 UTC

CLASSICAL HOMOCYSTINURIA DUE TO CYSTATHIONINE BETA SYNTHASE DEFICIENCY

Get alerted when new CLASSICAL HOMOCYSTINURIA DUE TO CYSTATHIONINE BETA SYNTHASE DEFICIENCY trials appear

New drug pegtibatinase tested for rare metabolic disorder over two years