N
← Back to Search
Sponsored

Accelerate Your Clinical Trial Recruitment

Reach thousands of high-intent patients matching your specific inclusion criteria.

Start Campaign
RECRUITING Phase 3 ClinicalTrials.gov

Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment

Sponsor: Travere Therapeutics, Inc. • ClinicalTrials.gov ID: NCT06247085

Registry updated: 2026-10-08
Ad

Recruiting for Similar Conditions?

Sponsors are looking for patients like you. See if you qualify for compensated research studies.

Check Eligibility →

Description

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.

Interventions

  • Pegtibatinase (Drug)
    Pegtibatinase BIW
  • Placebo (Other)
    volume-matched saline SC BIW

Eligibility criteria

Inclusion Criteria: * Must be ≥12 to ≤65 years of age, at the time of signing the informed consent * Must have a diagnosis of classical HCU based on clinical, biochemical, and/or molecular genetic testing * Plasma tHcy ≥80 µM at Screening visit, with allowance for up to 18 participants who may be enrolled with a Screening plasma tHcy ≥50 to \<80 µM * Participants who can become pregnant must have a negative pregnancy test before starting the study and must use a highly effective form of birth control (less than 1% risk of pregnancy per year) during the study and for at least 4 weeks after the last dose. * Willing to maintain a generally stable diet for the duration of the study (unless changes are required based on medical/safety reasons) * Willing to maintain generally stable intake and doses of betaine, pyridoxine, and medical food for the duration of the study (unless changes are required based on medical/safety reasons) Exclusion Criteria: * Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorder of cobalamin metabolism * Concurrent disease or condition (eg, history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease) that would interfere with study participation or safety (excluding complications of HCU). * History of major thrombotic event (eg, cerebrovascular accident, myocardial infarction, pulmonary embolism) in the previous 6 months. * Body weight ≥160 kg. * Use or planned use of any injectable drugs containing PEG (excluding PEG-containing vaccines) * Any previous exposure to pegtibatinase and/or previous participation in a clinical study that included administration of pegtibatinase or pegtarviliase * Prior severe immune reaction to a PEG-containing product

Conditions

Homocystinuria

Locations

  • 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
Sponsored

Partner with Nurenyx

Showcase your study to thousands of motivated patients matching your criteria.

Learn More

Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

View Official Record