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
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)
1 other identifier
interventional
100
14 countries
52
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 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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Apr 2024
Longer than P75 for phase_3
52 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 30, 2024
CompletedFirst Submitted
Initial submission to the registry
May 8, 2024
CompletedFirst Posted
Study publicly available on registry
May 29, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 1, 2030
May 28, 2026
May 1, 2026
6.3 years
May 8, 2024
May 27, 2026
Conditions
Keywords
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 (3)
Maximum Drug Concentration
Pharmacokinetic Assessment Period - 4 weeks & 8 weeks
Area under the Curve within the Dosing Interval
Pharmacokinetic Assessment Period - 4 weeks & 8 weeks
Total Homocysteine (tHcy) Levels
Week 1 - Week 108
Study Arms (1)
pegtibatinase
EXPERIMENTALInterventions
Eligibility Criteria
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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (52)
Phoenix Children's Hospital
Phoenix, Arizona, 85006, United States
Yale University School of Medicine
New Haven, Connecticut, 06519, United States
Emory University
Atlanta, Georgia, 30322, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Maine Health Pediatric Specialty Care
Portland, Maine, 04012, United States
Uncommon Cures
Chevy Chase, Maryland, 20815, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Science 37 - Virtual Site
Morrisville, North Carolina, 27560, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Utah Health - The University of Utah Primary Children's Hospital
Salt Lake City, Utah, 84113, 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
Ospedale Pediatrico Giovanni XXIII
Bari, Italy
IRCCS Istituto Giannina Gaslini
Genova, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Policlinico di Milano)
Milan, Italy
Fondazione IRCCS San Gerardo Dei Tintori - Ospedale San Gerardo
Monza, Italy
Azienda Ospedaliera di Padova
Padova, Italy
SP ZOZ Szpital Uniwersytecki w Krakowie
Krakow, Poland
Instytut Matki i Dziecka
Warsaw, Poland
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Poland
Unidade Local de Saúde de Coimbra, EPE - Hospitais da Universidade de Coimbra
Coimbra, Portugal
Unidade Local de Saúde de Santa Maria, EPE - Hospital de Santa Maria
Lisbon, Portugal
Unidade Local de Saúde de São João, EPE - Hospital São João
Porto, Portugal
Hamad General Hospital
Doha, Qatar
Sidra Hospital
Doha, Qatar
King Abdullah International Medical Research Center
Riyadh, Saudi Arabia
King Faisal Specialist Hospital and Research Centre (KFSHRC) - Riyadh
Riyadh, Saudi Arabia
Hospital Sant Joan de Déu
Barcelona, Spain
Hospital Universitario 12 de Octubre
Madrid, Spain
Hospital Universitario Ramón y Cajal
Madrid, Spain
Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario
Santiago de Compostela, Spain
Hospital Universitario Virgen del Rocío
Seville, Spain
Gazi University Hospital
Ankara, Turkey (Türkiye)
Ege Üniversitesi Hastanesi Bornova
Bornova, Turkey (Türkiye)
Istanbul University Cerrahpasa Hospital
Istanbul, Turkey (Türkiye)
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
University College London Hospitals NHS Foundation Trust - National Hospital for Neurology and Neurosurgery
London, United Kingdom
Salford Royal NHS Foundation Trust - Salford Royal Hospital
Salford, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Michael Imperiale, MD
Travere Therapeutics, Inc.
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)
August 1, 2030
Study Completion (Estimated)
August 1, 2030
Last Updated
May 28, 2026
Record last verified: 2026-05
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.
Requests for clinical trial data, including language stating its intended use, should be directed to datarequest@travere.com. If approved, the requested information will be provided to the requestor after signing a data access agreement. 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. Travere reserves the right to decline or recommend modifications to a request if it does not comply with the data sharing policy or if it is determined that the request is made by a biased source.