A 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)

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 blinded treatment in the Phase 3 HARMONY are eligible to participate. Participants will be in this clinical study for a minimum of 108 weeks including: * a treatment period of up to 104 weeks * a 4-week safety follow-up period

NCT Number	NCT06431893
Lead Sponsor	Travere Therapeutics, Inc.
Conditions	Homocystinuria
Enrollment	100 participants
Start Date	2024-04-30
Primary Completion	2030-08 (estimated)
Study Completion	2030-08 (estimated)
Updated on ClinicalTrials.gov	2026-05-28