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)
- Registration Number
- NCT06431893
- Lead Sponsor
- Travere Therapeutics, Inc.
- Brief 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.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 100
- Participants active in the COMPOSE study or who completed the 24-week blinded period on the full target dose of study intervention in the Phase 3 HARMONY study
- Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description pegtibatinase Pegtibatinase -
- Primary Outcome Measures
Name Time Method Treatment-emergent AEs Week 1 - Week 108 Incidence of treatment-emergent AEs
Hypermethioninemia Week 1 - Week 108 Incidence of hypermethioninemia
Hypomethioninemia Week 1 - Week 108 Incidence of hypomethioninemia
Dietary Protein Rescue Week 1 - Week 108 Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia
- Secondary Outcome Measures
Name Time Method Total Homocysteine (tHcy) Levels Week 1 - Week 108 Relative and absolute changes from baseline in tHcy levels
Trial Locations
- Locations (2)
Travere Investigational Site
🇺🇸Philadelphia, Pennsylvania, United States
Travere Investigational Site - Virtual Site
🇺🇸Morrisville, North Carolina, United States