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

• Status: ACTIVE_NOT_RECRUITING
• Trial end date: 2026-01-01

Summary

The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standard of care who have not achieved tHcy target levels. Study details include:

• Total Study duration: up to 38 weeks
• Screening:

• Initial Screening duration: up to 4 weeks
• Pre-treatment Diet Standardization Period duration: up to 6 weeks
• Blinded Treatment Duration: 24 weeks

• 2-week blinded dose titration period
• 22-week blinded assessment period
• Safety Follow-Up: 4 weeks after last dose (as applicable for those not enrolling in the long term extension study, ENSEMBLE)

• Phase: PHASE3

Details

• Lead Sponsor: Travere Therapeutics, Inc.