HOMOCYSTINURIA

Clinical trials for HOMOCYSTINURIA explained in plain language.

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Trials to join now! 1 Not yet finished but already full! 3 Completed 1

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: HOMOCYSTINURIA

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

Last updated Jun 27, 2026 12:30 UTC

HOMOCYSTINURIA

Get alerted when new HOMOCYSTINURIA trials appear

New shot could tame rare metabolic disease