New hope for rare metabolic disorder: phase 3 drug trial targets toxic amino acid buildup

NCT ID NCT06247085

NEW Recruiting now Disease control Sponsor: Travere Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen May 08, 2026 · Last updated May 08, 2026

Summary

This study tests an experimental drug called pegtibatinase in 70 people aged 12 to 65 with classical homocystinuria, a rare genetic condition that causes dangerously high levels of the amino acid homocysteine. Participants receive weekly injections of the drug or a placebo for 24 weeks, alongside their usual care. The main goal is to see if pegtibatinase safely lowers homocysteine levels in the blood.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago
NOT_YET_RECRUITING

Chicago, Illinois, 60611, United States
Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades
NOT_YET_RECRUITING

Paris, France
Azienda Ospedaliera di Padova
NOT_YET_RECRUITING

Padova, Italy
Birmingham Women's and Children's NHS Foundation Trust
NOT_YET_RECRUITING

Birmingham, United Kingdom
CHRU Hôpitaux de Tours - Hôpital Bretonneau
NOT_YET_RECRUITING

Tours, France
Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital d'enfants
NOT_YET_RECRUITING

Vandœuvre-lès-Nancy, France
Children's Health Ireland (CHI) at Temple Street
NOT_YET_RECRUITING

Dublin, Ireland
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Cliniques Universitaires Saint-Luc
NOT_YET_RECRUITING

Brussels, Belgium
Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario
NOT_YET_RECRUITING

Santiago de Compostela, Spain
Ege Üniversitesi Hastanesi Bornova
NOT_YET_RECRUITING

Bornova, Turkey (Türkiye)
Emory University
RECRUITING

Atlanta, Georgia, 30322, United States
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Policlinico di Milano)
NOT_YET_RECRUITING

Milan, Italy
Fondazione IRCCS San Gerardo Dei Tintori - Ospedale San Gerardo
NOT_YET_RECRUITING

Monza, Italy
Gazi University Hospital
NOT_YET_RECRUITING

Ankara, Turkey (Türkiye)
Hamad General Hospital
NOT_YET_RECRUITING

Doha, Qatar
Hospital Sant Joan de Déu
NOT_YET_RECRUITING

Barcelona, Spain
Hospital Universitario 12 de Octubre
NOT_YET_RECRUITING

Madrid, Spain
Hospital Universitario Ramón y Cajal
NOT_YET_RECRUITING

Madrid, Spain
Hospital Universitario Virgen del Rocío
NOT_YET_RECRUITING

Seville, Spain
IRCCS Istituto Giannina Gaslini
NOT_YET_RECRUITING

Genova, Italy
Instytut Matki i Dziecka
NOT_YET_RECRUITING

Warsaw, Poland
Instytut Pomnik-Centrum Zdrowia Dziecka
NOT_YET_RECRUITING

Warsaw, Poland
Istanbul University Cerrahpasa Hospital
NOT_YET_RECRUITING

Istanbul, Turkey (Türkiye)
King Abdullah International Medical Research Center
NOT_YET_RECRUITING

Riyadh, Saudi Arabia
King Faisal Specialist Hospital and Research Centre (KFSHRC) - Riyadh
NOT_YET_RECRUITING

Riyadh, Saudi Arabia
Maine Health - Maine Medical Center
RECRUITING

Portland, Maine, 04102, United States
Oregon Health and Science University
NOT_YET_RECRUITING

Portland, Oregon, 97239, United States
Ospedale Pediatrico Giovanni XXIII
NOT_YET_RECRUITING

Bari, Italy
Phoenix Children's Hospital
NOT_YET_RECRUITING

Phoenix, Arizona, 85006, United States
Royal Adelaide Hospital
NOT_YET_RECRUITING

Adelaide, Australia
Royal Children's Hospital Melbourne
NOT_YET_RECRUITING

Parkville, Australia
SP ZOZ Szpital Uniwersytecki w Krakowie
NOT_YET_RECRUITING

Krakow, Poland
Salford Royal NHS Foundation Trust - Salford Royal Hospital
NOT_YET_RECRUITING

Salford, United Kingdom
Science 37 - Virtual Site
RECRUITING

Morrisville, North Carolina, 27560, United States
Sidra Hospital
NOT_YET_RECRUITING

Doha, Qatar
The Mount Sinai Hospital
RECRUITING

New York, New York, 10029, United States
Uncommon Cures
RECRUITING

Chevy Chase, Maryland, 20815, United States
Unidade Local de Saúde de Coimbra, EPE - Hospitais da Universidade de Coimbra
NOT_YET_RECRUITING

Coimbra, Portugal
Unidade Local de Saúde de Santa Maria, EPE - Hospital de Santa Maria
NOT_YET_RECRUITING

Lisbon, Portugal
Unidade Local de Saúde de São João, EPE - Hospital São João
NOT_YET_RECRUITING

Porto, Portugal
Universitair Ziekenhuis Antwerpen (UZA)
NOT_YET_RECRUITING

Edegem, Belgium
University College London Hospitals NHS Foundation Trust - National Hospital for Neurology and Neurosurgery
NOT_YET_RECRUITING

London, United Kingdom
University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital Birmingham
NOT_YET_RECRUITING

Birmingham, United Kingdom
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
University of Texas Southwestern Medical Center
RECRUITING

Dallas, Texas, 75390, United States
Universitätsklinikum Hamburg-Eppendorf
NOT_YET_RECRUITING

Hamburg, Germany
Universitätsklinikum Leipzig
NOT_YET_RECRUITING

Leipzig, Germany
Universitätsklinikum Münster
NOT_YET_RECRUITING

Münster, Germany
Utah Health - The University of Utah Primary Children's Hospital
NOT_YET_RECRUITING

Salt Lake City, Utah, 84113, United States
Westmead Hospital
NOT_YET_RECRUITING

Westmead, Australia
Yale University School of Medicine
RECRUITING

New Haven, Connecticut, 06519, United States

Conditions

Explore the condition pages connected to this study.

HOMOCYSTINURIA