NCT06247085Phase 3Recruiting

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

Study sponsor: Travere Therapeutics, Inc. · TVTX

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

Primary completion2027-08Estimated
Enrollment · participants70Estimated
Source updated2026-08-07Last update listed in the registry record
Version history

Changes to this trial

Last 12 months
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· 3 fields changed

Main study measure wording changed

Text changed; whether the study measures changed has not been verified. Saved text may be shortened.

Compare wording
− Before

Measure: Change from baseline in plasma tHcy levels

+ After

Measure: Change from baseline in plasma tHcy levels - Weeks 6 to 12

Primary completion date changed

− Before

Dec 2025

+ After

Aug 2027

Recruitment status changed

− Before

Active not recruiting

+ After

Recruiting

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