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

Recruiting

Phase 3

PegtibatinaseA 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

Sponsor:

Travere Therapeutics, Inc.

Code:

NCT06247085

Conditions

Homocystinuria

Eligibility Criteria

Sex: All

Age: 12 - 65

Healthy Volunteers: Not accepted

Interventions

Pegtibatinase

Placebo

Study Details

Eligibility Criteria

Study Design

Interventions and Outcome Measures

Central Contacts and Locations

More Information

Trial information was received from ClinicalTrials.gov and was last updated on 2026-05-24. This information was provided to ClinicalTrials.gov by Travere Therapeutics, Inc. on 2026-05-08.