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New shot could tame rare metabolic disease

NCT ID NCT06247085

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 study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Aug 07, 2026 · Updated 2 times

Summary

This Phase 3 trial tests pegtibatinase, an enzyme given as a shot, in 70 people aged 12–65 with classical homocystinuria. The goal is to see if it safely lowers high homocysteine levels when added to standard care. Participants receive either the drug or a placebo for 24 weeks.

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 lab-made enzyme given as a shot under the skin)
What this could lead to
If it works, this could offer a new treatment option to help control homocystinuria by lowering harmful homocysteine levels.
What could go wrong
This is a relatively small Phase 3 trial (70 people). The drug may not lower homocysteine enough or could cause side effects. It is not a cure—patients will still need standard care.
Why investors are watching

Travere Therapeutics is running a phase 3 trial of pegtibatinase for classical homocystinuria, a rare genetic disease. The trial compares the drug against placebo in 70 patients over 24 weeks, and a positive result could give the small company a new approved treatment for a condition with few options. For a company of this size, this single readout carries outsized weight because it tests whether the drug actually controls the disease.

If it works: If the trial shows pegtibatinase safely reduces homocystine levels better than placebo, Travere could gain a new product for a niche patient population. That outcome would expand its pipeline beyond its existing portfolio and support a regulatory submission.

If it fails: The trial could fail to show a meaningful difference from placebo, or safety issues could emerge, which would likely end the program. Trials in rare diseases often miss their goals, and a negative result would leave Travere without this potential new therapy.

AI-written from the trial record. Speculative, and not investment advice.

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 70 people

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

Started

Dec 2023

Expected to finish

Sep 2027

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

12 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: * 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

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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 study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

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

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. 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

Study contacts

  • Contact

    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

    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

    RECRUITING

    Phoenix, Arizona, 85006, United States

  • Royal Adelaide Hospital

    RECRUITING

    Adelaide, Australia

  • Royal Children's Hospital Melbourne

    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

    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

    RECRUITING

    Salt Lake City, Utah, 84113, United States

  • Westmead Hospital

    RECRUITING

    Westmead, Australia

  • Yale University School of Medicine

    RECRUITING

    New Haven, Connecticut, 06519, United States

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Other studies related to the condition(s) this trial covers.