HomeTrialNCT06431893
🔬NCT06431893Phase 3ENROLLING BY INVITATION

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)

Study 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

Conditions
Interventions / Drugs
View on ClinicalTrials.gov ↗
Study Details
Enrollment100 participants
Primary Completion
Lead SponsorTravere Therapeutics, Inc.
Data Retrieved2026-04-11 04:30:13.942984+00:00
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