NCT06431893

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)

An Open-Label, Phase 3 Long-Term Extension (LTE) Study To Assess The Safety, Tolerability, And Efficacy Of Treatment With Pegtibatinase In Participants With Classical Homocystinuria (HCU) Due To Cystathionine Beta Synthase Deficiency (ENSEMBLE)

• Status: Enrolling By Invitation
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 100 (estimated)
• Sponsor: Travere Therapeutics, Inc. · Industry
• Sex: All
• Age: 5 Years – 65 Years
• Healthy volunteers: Not accepted

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

Detailed description

Overall Design: This is a global, multicenter, single-arm, open-label study. Participants who meet all eligibility criteria may transition from other pegtibatinase studies and enroll into this LTE study for long-term safety, efficacy, and clinical assessments of pegtibatinase treatment. All participants will follow a protocol of self-administration of pegtibatinase unless exempted. During the ENSEMBLE study, an optional protein tolerance modification (PTM) sub-study will also be conducted for eligible participants.

Conditions

• Homocystinuria

Interventions

Type	Name	Description
DRUG	Pegtibatinase	Full Target Dose of pegtibatinase BIW

Timeline

• Start date: 2024-04-30
• Primary completion: 2027-01-01
• Completion: 2027-01-01
• First posted: 2024-05-29
• Last updated: 2025-04-11

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study