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)

Homocystinuria Clinical Trial

— ENSEMBLE  

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)

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT06431893
• Other study ID #: TVTX-TVT058-302
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: April 30, 2024
• Est. completion date: January 2027

Study information

• Verified date: May 2024
• Source: Travere Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

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

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 information / eligibility

• Status: Enrolling by invitation
• Enrollment: 90
• Est. completion date: January 2027
• Est. primary completion date: January 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 65 Years

Eligibility

Inclusion Criteria:

• Participants active in the Phase 1/2 COMPOSE study or who completed the 24-week blinded period in the Phase 3 HARMONY study

Exclusion Criteria:

• Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance

Related Conditions & MeSH terms

• Homocystinuria

Intervention

Drug:
• Pegtibatinase
Full Target Dose of pegtibatinase BIW

Locations

Country	Name	City	State
United States	Travere Investigational Site - Virtual Site	Culver City	California

Sponsors (1)

Lead Sponsor	Collaborator
Travere Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment-emergent AEs	Incidence of treatment-emergent AEs	Week 1 - Week 56
Primary	Hypermethioninemia	Incidence of hypermethioninemia	Week 1 - Week 56
Primary	Hypomethioninemia	Incidence of hypomethioninemia	Week 1 - Week 56
Primary	Dietary Protein Rescue	Incidence of dietary protein rescue	Week 1 - Week 56
Secondary	Total Homocysteine (tHcy) Levels	Relative and absolute changes from baseline in tHcy levels	Week 1 - Week 56