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New drug pegtibatinase tested for rare metabolic disorder over two years

NCT ID NCT06431893

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 100 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2024

Expected to finish

Aug 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

5 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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 Exclusion Criteria: * Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    52 sites in 14 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

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

More trials for these conditions

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