A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

• Status: ENROLLING_BY_INVITATION
• Trial end date: 2027-01-01

Summary

The goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU). Patients who are active in the Phase 1/2 COMPOSE study or those who complete the 24 weeks of treatment in the Phase 3 HARMONY are eligible to participate.

Participants will be in this clinical study for up to about 13 months including:

• a treatment period of up to 104 weeks
• a 4-week safety follow-up period

• Phase: PHASE3

Details

• Lead Sponsor: Travere Therapeutics, Inc.