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GSDIa patients monitored for 10 years after gene therapy

NCT ID NCT06636383

Knowledge-focused 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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Aug 27, 2026 · Updated 5 times

Summary

This observational study follows people with Glycogen Storage Disease Type Ia who have already received the gene therapy DTX401. The goal is to track their health and safety for at least 10 years after treatment. No new drugs or treatments are given in this study. It will help researchers understand the long-term effects of DTX401.

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

About 140 people

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

Started

Nov 2024

Expected to finish

Dec 2036

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

All participants who received DTX401 in a qualifying clinical study will be expected and asked to participate in the DMP upon completion or early termination (ET) from the parent study, as required by regulatory agencies for all gene therapy studies.

Ages

2 years and older

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: * Patient who had: * DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or * Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2) * Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. Exclusion Criteria: * Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

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Conditions

The condition(s) this trial relates to.

glucose metabolism disease glycogen storage disease due to glucose-6-phosphatase deficiency type IA glycogen storage disease I

As listed by the trial registrant

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

Glycogen storage disease type IA

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

    19 sites in 9 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

  • Children's Hospital Colorado

    RECRUITING

    Denver, Colorado, 80045, United States

  • Children's Hospital of Orange County

    RECRUITING

    Orange, California, 92868, United States

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Fujita Health University Hospital

    RECRUITING

    Toyoake, 470-1192, Japan

  • Groningen University

    RECRUITING

    Groningen, Provincie Groningen, 9700 RB, Netherlands

  • Hospital Clínico Universitario de Santiago

    RECRUITING

    Santiago, 15706, Spain

  • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

  • Istituto G Gaslini Ospedale Pediatrico IRCCS

    RECRUITING

    Genoa, 16147, Italy

  • McGill University Health Center

    RECRUITING

    Montreal, Quebec, H4A3J1, Canada

  • Osaka City General Hospital

    RECRUITING

    Osaka, 534-0021, Japan

  • Primary Children's Hospital

    NOT_YET_RECRUITING

    Salt Lake City, Utah, 84108, United States

  • Rigshospitalet

    RECRUITING

    Copenhagen, 2700, Denmark

  • The Cleveland Clinic Foundation

    NOT_YET_RECRUITING

    Cleveland, Ohio, 44195, United States

  • University of Connecticut Health Center

    RECRUITING

    Hartford, Connecticut, 06106, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Naples

    RECRUITING

    Naples, Campania, 80131, Italy

  • University of Texas Health Science Center at Houston

    RECRUITING

    Houston, Texas, 77030, United States

  • Universitätsklinikum Hamburg Eppendorf

    RECRUITING

    Hamburg, 20246, Germany

More trials for these conditions

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