A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) — ENSEMBLE  

Homocystinuria Clinical Trial

Official title: 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

Clinical Trial 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 52 weeks

• a 4-week safety follow-up period

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Homocystinuria

• NCT number: NCT06431893
• Study type: Interventional
• Source: Travere Therapeutics, Inc.
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: April 30, 2024
• Completion date: January 2027