A 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) (ENSEMBLE)

What this study involves

The registry does not currently label this study as recruiting. Ask about its status before planning a screening visit.

Study type

Interventional

Phase

Phase 3

Age range

5 years - 65 years

Sex eligible

All sexes

Healthy volunteers

Not accepted

Participants

100 Estimated

These are starting points, not an eligibility decision. The research team must review the full requirements.

About the research

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 blinded treatment in the Phase 3 HARMONY are eligible to participate.

Participants will be in this clinical study for a minimum of 108 weeks including:

a treatment period of up to 104 weeks a 4-week safety follow-up period

Who may be able to participate?

The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.

Inclusion Criteria:

• Participants active in the COMPOSE study or who completed the 24- week blinded period on study intervention (pegtibatinase or placebo) 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

Treatment, groups and participation

The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.

Assignment

Not applicable

Masking

No masking

What researchers are measuring

Outcome measures describe what is being studied. They are not proof that a treatment works.

Primary measures

Secondary measures

Research locations (52)

The overall study status and a local site’s status can differ. Confirm that location’s availability before traveling.

52 locations

Study team and timeline

Lead sponsor

Travere Therapeutics, Inc.

Study start

Apr 30, 2024 Actual

Primary completion

2030-08 Estimated

Study completion

2030-08 Estimated

First posted

May 29, 2024 Actual

Last updated

Nov 10, 2026 Actual

Official study contacts

No central contact is listed. Check the locations and the official record.