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 shot could tame rare metabolic disease

Disease control Recruiting now

This Phase 3 trial tests pegtibatinase, an enzyme given as a shot, in 70 people aged 12–65 with classical homocystinuria. The goal is to see if it safely lowers high homocysteine levels when added to standard care. Participants receive either the drug or a placebo for 24 weeks.

Matched conditions: CLASSICAL HOMOCYSTINURIA DUE TO CYSTATHIONINE BETA SYNTHASE DEFICIENCY

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

Last updated Jun 27, 2026 12:30 UTC

CLASSICAL HOMOCYSTINURIA DUE TO CYSTATHIONINE BETA SYNTHASE DEFICIENCY

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

New shot could tame rare metabolic disease