New drug pegtibatinase tested for rare metabolic disorder over two years

NCT ID NCT06431893

ENROLLING_BY_INVITATION Disease control Sponsor: Travere Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests the long-term safety and effectiveness of pegtibatinase in people with classical homocystinuria (HCU), a rare genetic disorder that prevents the body from breaking down certain amino acids. About 100 participants who completed earlier studies will receive the drug for up to 2 years. The goal is to see if pegtibatinase can help manage the condition with fewer dietary restrictions.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Pegtibatinase (a drug given by injection to help break down harmful amino acids)

What this could lead to

If successful, this could provide a long-term treatment option for people with classical homocystinuria, potentially reducing the need for strict dietary restrictions.

What could go wrong

This is an extension study, so it builds on earlier results. However, long-term safety and effectiveness are still being evaluated, and side effects like abnormal methionine levels may occur.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

classic homocystinuria homocystinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HOMOCYSTINURIA

Contacts and locations

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Locations

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

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

Paris, France
Azienda Ospedaliera di Padova

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

Birmingham, United Kingdom
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
Children's Health Ireland (CHI) at Temple Street

Dublin, Ireland
Cliniques Universitaires Saint-Luc

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

Santiago de Compostela, Spain
Ege Üniversitesi Hastanesi Bornova

Bornova, Turkey (Türkiye)
Emory University

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

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

Monza, Italy
Gazi University Hospital

Ankara, Turkey (Türkiye)
Hamad General Hospital

Doha, Qatar
Hospital Sant Joan de Déu

Barcelona, Spain
Hospital Universitario 12 de Octubre

Madrid, Spain
Hospital Universitario Ramón y Cajal

Madrid, Spain
Hospital Universitario Virgen del Rocío

Seville, Spain
IRCCS Istituto Giannina Gaslini

Genova, Italy
Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States
Instytut Matki i Dziecka

Warsaw, Poland
Instytut Pomnik-Centrum Zdrowia Dziecka

Warsaw, Poland
Istanbul University Cerrahpasa Hospital

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

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

Riyadh, Saudi Arabia
Maine Health Pediatric Specialty Care

Portland, Maine, 04012, United States
Oregon Health and Science University

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

Bari, Italy
Phoenix Children's Hospital

Phoenix, Arizona, 85006, United States
Royal Adelaide Hospital

Adelaide, Australia
Royal Children's Hospital Melbourne

Parkville, Australia
SP ZOZ Szpital Uniwersytecki w Krakowie

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

Salford, United Kingdom
Science 37 - Virtual Site

Morrisville, North Carolina, 27560, United States
Sidra Hospital

Doha, Qatar
The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Uncommon Cures

Chevy Chase, Maryland, 20815, United States
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
Universitair Ziekenhuis Antwerpen (UZA)

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

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

Birmingham, United Kingdom
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
Universitätsklinikum Hamburg-Eppendorf

Hamburg, Germany
Universitätsklinikum Leipzig

Leipzig, Germany
Universitätsklinikum Münster

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

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

Westmead, Australia
Yale University School of Medicine

New Haven, Connecticut, 06519, United States