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)
Enrolling by invitation · Phase 3
Conditions studied: Homocystinuria
In brief
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
Key facts
- Study ID
- NCT06431893
- Run by
- Travere Therapeutics, Inc.
- People needed
- 100
- Starts
- 2024-04-30
- Expected to finish
- 2030-08-01
- Last updated by the study team
- 2026-05-28
Who can join
Age: 5 and older, up to 65. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance
Where it is running
- Yale University School of Medicine — New Haven, Connecticut, United States
- Emory University — Atlanta, Georgia, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- Maine Health Pediatric Specialty Care — Portland, Maine, United States
- Uncommon Cures — Chevy Chase, Maryland, United States
- Icahn School of Medicine at Mount Sinai — New York, New York, United States
- Science 37 - Virtual Site — Morrisville, North Carolina, United States
- Oregon Health and Science University — Portland, Oregon, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- University of Texas Southwestern Medical Center — Dallas, Texas, United States
- Utah Health - The University of Utah Primary Children's Hospital — Salt Lake City, Utah, United States
- Royal Adelaide Hospital — Adelaide, Australia
- Royal Children's Hospital Melbourne — Parkville, Australia
- Westmead Hospital — Westmead, Australia
- Cliniques Universitaires Saint-Luc — Brussels, Belgium
- Universitair Ziekenhuis Antwerpen (UZA) — Edegem, Belgium
- Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades — Paris, France
- CHRU Hôpitaux de Tours - Hôpital Bretonneau — Tours, France
- Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital d'enfants — Vandœuvre-lès-Nancy, France
- Universitätsklinikum Hamburg-Eppendorf — Hamburg, Germany
- Universitätsklinikum Leipzig — Leipzig, Germany
- Universitätsklinikum Münster — Münster, Germany
- Children's Health Ireland (CHI) at Temple Street — Dublin, Ireland
- Phoenix Children's Hospital — Phoenix, Arizona, United States
Full record on ClinicalTrials.gov
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