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Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment
Sponsor: Travere Therapeutics, Inc.
Summary
Researchers are looking for a better way to treat people living with classical homocystinuria (HCU), a rare inherited condition. It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme helps break down homocysteine, which is produced when the body processes methionine from protein-containing foods. This helps keep homocysteine and methionine at healthy levels. When this enzyme is not working, homocysteine and methionine build up in the blood and then spread into different tissues of the body and stop these body tissues from working normally. People living with HCU can experience problems with their vision, bones, blood vessels, thinking, learning, and memory. Available treatments for HCU include a low-methionine diet (typically achieved by limiting protein-containing foods), a methionine-free protein supplement to help meet daily protein needs, and betaine (Cystadane®). These treatments are either not sufficient or are hard to take for many patients. Pegtibatinase was designed by researchers to act like the CBS enzyme, which is missing or not working properly in people living with HCU. Researchers are studying whether adding pegtibatinase to standard treatment can lower homocysteine levels. Researchers want to learn how safe and effective pegtibatinase is, how it moves through and affects the body, and how it may affect the daily lives of people with HCU. The study plans to enroll participants from the US, Australia, Belgium, France, Germany, Ireland, Italy, Poland, Portugal, Qatar, Saudi Arabia, Spain, Turkey, and the United Kingdom. Many study visits may happen at home, depending on where you live and local regulations. Some visits may include a home nurse and telemedicine appointments with the study doctor.
Official title: A Phase 3, Parallel-Group Treatment, Blinded, Randomized, Placebo-Controlled Study To Assess The Efficacy And Safety Of Pegtibatinase Administered Subcutaneously In Addition To Standard Of Care In Participants With Classical Homocystinuria Due To Cystathionine Beta Synthase Deficiency (HARMONY)
Key Details
Gender
All
Age Range
12 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
70
Start Date
2023-12-28
Completion Date
2027-09
Last Updated
2026-10-08
Healthy Volunteers
No
Conditions
Interventions
Pegtibatinase
Pegtibatinase BIW
Placebo
volume-matched saline SC BIW
Locations (52)
Phoenix Children's Hospital
Phoenix, Arizona, United States
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 - Maine Medical Center
Portland, Maine, United States
Uncommon Cures
Chevy Chase, Maryland, United States
The Mount Sinai Hospital
New York, New York, United States
Science 37 - Virtual Site
Morrisville, North Carolina, United States
Oregon Health and Science University
Portland, Oregon, United States
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
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