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Gene therapy for rare Sugar-Storage disease shows promise in Long-Term safety check

NCT ID NCT03970278

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Updated 1 time

Summary

This study checked the long-term safety of a one-time gene therapy (DTX401) in 12 adults with glycogen storage disease type Ia, a rare condition that causes dangerously low blood sugar. Researchers monitored side effects and how well the therapy helped control blood sugar levels over time. The goal was to see if the treatment remains safe and effective in the long run.

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

12 people

The number who actually took part.

Started

Jul 2019

Finished

Feb 2025

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 GSDIa previously enrolled in 401GSDIA01.

Ages

18 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: 1. Received DTX401 in study 401GSDIA01. 2. Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures being performed. 3. Willing and able to comply with all scheduled study visits, procedures, and requirements. Exclusion Criteria: 1. Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study. 2. Presence or history of any condition that, in the view of the Investigator, poses a risk to subject safety or places the subject at high risk of poor compliance or not completing the study or that would significantly affect the interpretation of study results.

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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 hereditary disease inborn carbohydrate metabolic disorder

As listed by the trial registrant

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

Glycogen storage disease type IA Von Gierke's disease (GSD type IA)

Contacts and locations

Locations

  • Complejo Hospitalario Universitario de Santiago

    Santiago de Compostela, A Coruna, 15706, Spain

  • Michigan Medicine University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • Montreal Children Hospital, McGill University Health Centre

    Montreal, Quebec, H4A3J1, Canada

  • UCONN Health

    Farmington, Connecticut, 06030-3213, United States

  • University Medical Center Groningen

    Groningen, 9700RB, Netherlands

  • University of Texas Health Science Center at Houston

    Houston, Texas, 77030, United States

More trials for these conditions

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