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)

NCT06431893 | Enrolling By Invitation Phase 3 DMC FDA Drug

• 1 other identifier: TVTX-TVT058-302
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 4

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

Conditions

Homocystinuria

Keywords

Classical homocystinuria; HCU; Cystathionine Beta Synthase Deficiency

Outcome Measures

Primary Outcomes (4)

• Treatment-emergent AEs

  Incidence of treatment-emergent AEs

  Week 1 - Week 108

• Hypermethioninemia

  Incidence of hypermethioninemia

  Week 1 - Week 108

• Hypomethioninemia

  Incidence of hypomethioninemia

  Week 1 - Week 108

• Dietary Protein Rescue

  Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia

  Week 1 - Week 108

Secondary Outcomes (1)

• Total Homocysteine (tHcy) Levels

  Week 1 - Week 108

Study Arms (1)

pegtibatinase

EXPERIMENTAL

Drug: Pegtibatinase

Interventions

Pegtibatinase DRUG

Full Target Dose of pegtibatinase BIW

Also known as: OT-58, TVT-058

pegtibatinase

Eligibility Criteria

• Age: 5 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Travere Therapeutics, Inc.: lead

Study Sites (4)

Travere Investigational Site

Portland, Maine, 04012, United States

Location

Travere Investigational Site

New York, New York, 10029, United States

Location

Travere Investigational Site - Virtual Site

Morrisville, North Carolina, 27560, United States

Location

Travere Investigational Site

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Homocystinuria

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hyperhomocysteinemia; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Michael Imperiale, MD

  Travere Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2024
• First Posted: May 29, 2024
• Study Start: April 30, 2024
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2027
• Last Updated: April 11, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will share
• Time Frame: Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication.
• Access Criteria: Requires submission and approval of intended use and a data sharing agreement.

Locations

US (4)