Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Gene therapy could free GSD ia patients from constant cornstarch

NCT ID NCT05139316

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 2 times

Summary

This Phase 3 trial tested a gene therapy called DTX401 in 49 people with glycogen storage disease type Ia (GSD Ia). The goal was to see if a single infusion could reduce or eliminate the need for frequent cornstarch doses to keep blood sugar stable. Participants were randomly assigned to receive either the gene therapy or a placebo, and their cornstarch intake was measured over 48 weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
DTX401 (pariglasgene brecaparvovec) gene therapy
What this could lead to
If successful, this gene therapy could reduce or eliminate the need for frequent cornstarch doses to maintain normal blood sugar levels in people with GSD Ia.
What could go wrong
This is an early-stage Phase 3 trial with only 49 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or liver issues, and long-term effects are unknown.

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

49 people

The number who actually took part.

Started

Nov 2021

Finished

Feb 2026

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

8 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.

Key Inclusion Criteria: * Documented GSDIa with confirmation by molecular testing or enzymatic activity on liver biopsy * Currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations with stable nutrition, glycemic, and clinical status. * Willing and able to complete the informed consent process and to comply with study procedures and visit schedule * Females of childbearing potential and fertile males must consent to use highly effective contraception from the period following informed consent through the duration of the 144-week study and in cases of early withdrawal at least 48 weeks after the last dose of investigational product (IP). Female subjects must agree not to become pregnant. Male subjects must agree not to father a child or donate sperm Key Exclusion Criteria: * Detectable pre-existing antibodies to the AAV8 capsid * History of liver transplant, including hepatocyte cell therapy/ transplant * History of liver disease * Presence of liver adenoma \>5 cm in size * Presence of liver adenoma \>3 cm and ≤5 cm in size that has a documented annual growth rate of ≥0.5 cm per year * Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> upper limit of normal (ULN), total bilirubin \>1.5 × ULN, alkaline phosphatase \>2.5 × ULN * Non-fasting triglycerides ≥1000 mg/dL * Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study. * Current or previous participation in another gene transfer study * History of illicit drug use within 60 days prior to screening or positive results from an 8-panel urine drug screen during the Screening Period completed at 2 time points at least 4 weeks apart Note: additional inclusion/exclusion criteria may apply, per protocol

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Glycogen storage disease type IA are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

Contacts and locations

Locations

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Duke University

    Durham, North Carolina, 27710, United States

  • Fujita Health University Hospital

    Toyoake, Japan

  • Groningen University

    Groningen, 9700 RB, Netherlands

  • Hospital Clinico Universitario de Santiago

    Santiago de Compostela, A Coruna, 15706, Spain

  • Hospital de Clinicas de Porto Alegre

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

  • Istituto Giannina Gaslini

    Genova, Linguria, 16147, Italy

  • Kumamoto University Hospital

    Kumamoto, Japan

  • McGill University

    Montreal, Quebec, H3H 1P3, Canada

  • Mount Sinai

    The Bronx, New York, 10467, United States

  • Osaka City General Hospital

    Osaka, Japan

  • Primary Children's Hospital

    Salt Lake City, Utah, 84132, United States

  • Righospitalet

    Copenhagen, Capital, 2100, Denmark

  • University Medical Center Eppendorf

    Hamburg, 20251, Germany

  • University of Connecticut Health Center

    Farmington, Connecticut, 06030, United States

  • University of Naples

    Naples, 80131, Italy

  • University of Texas

    Houston, Texas, 77030, United States

More trials for these conditions

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