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Gene therapy for rare liver disease: Long-Term safety check

NCT ID NCT03636438

Disease control Sponsor: Ultragenyx Pharmaceutical 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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 follows 11 adults with late-onset OTC deficiency who received a single dose of gene therapy (DTX301) in an earlier trial. Researchers are checking long-term safety and how well the body makes urea and controls ammonia levels. The goal is to see if the treatment remains safe over time.

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.

Participants

11 people

The number who actually took part.

Started

Aug 2018

Expected to finish

Dec 2029

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.

Who is studied

Subjects 18 years of age or older with OTC previously enrolled in Study 301OTC01

Ages

18 years and older

Sex

Anyone

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: 1. Completed the Week 52 visit in Study 301OTC01. 2. Willing and able to provide written informed consent. 3. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements. Exclusion Criteria: 1. Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX301 during the duration of this study. 2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study.

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Conditions

The condition(s) this trial relates to.

ornithine carbamoyltransferase deficiency urea cycle disorder

As listed by the trial registrant

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

Ornithine transcarbamylase (OTC) deficiency

Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Hopital Femme Mere Enfant

    Bron, Rhone, 69677, France

  • Hospital Clinico Universitario de Santiago

    Santiago de Compostela, Coruna, 15706, Spain

  • Hospital Universitario de Cruces. Servicio de Pediatria

    Barakaldo, Vizcaya, 48903, Spain

  • Icahn School of Medicine

    New York, New York, 10029, United States

  • M.A.G.I.C. Clinic

    Calgary, Alberta, T2E 7Z7, Canada

  • Queen Elizabeth Hospital, Department of Endocrinology

    Birmingham, B15 2TH, United Kingdom

  • The Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • University Hospital Cleveland Medical Center/Case Western Reserve University

    Cleveland, Ohio, 44106, United States

More trials for these conditions

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