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Study on Long-Term Safety and Pregnancy Outcomes of Pariglasgene Brecaparvovec in Patients with Glycogen Storage Disease Type Ia (GSDIa)

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on a rare condition known as Glycogen Storage Disease Type Ia (GSDIa). This is a genetic disorder that affects how the body stores and uses sugar, which can lead to low blood sugar levels and other health issues. The study is examining a treatment called Pariglasgene brecaparvovec, also known by its code name DTX401. This treatment is given as a solution through an intravenous infusion, which means it is delivered directly into the bloodstream through a vein.

The main purpose of the study is to evaluate the long-term safety of DTX401 in patients with GSDIa, as well as to monitor pregnancy outcomes in these patients. Participants in the study may have previously received DTX401 in an earlier clinical trial or as part of their regular treatment. The study will track any serious side effects that might occur, including those related to the infusion process or any other medications the patients might be taking, such as steroids. It will also look at any potential effects on the liver, the development of new or worsening cancers, and other health issues that might arise over time.

Throughout the study, various assessments will be conducted to gather information on the participants' nutritional status, clinical and laboratory findings, and overall health experiences. This will help researchers understand the impact of DTX401 on patients' lives and identify any major clinical events that occur during the study period. The study is expected to continue for several years, allowing for comprehensive monitoring and data collection.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after providing informed consent. If underage or with cognitive limitations, a legally authorized representative must provide consent.

    Eligibility requires previous administration of DTX401 in a clinical study or post-marketing setting.

  2. Step 2

    Medication administration

    Pariglasgene brecaparvovec is administered as a solution for infusion through an intravenous route.

    The medication is part of the study to evaluate long-term safety and pregnancy outcomes in patients with Glycogen Storage Disease Type Ia (GSDIa).

  3. Step 3

    Monitoring and assessments

    Regular monitoring for any serious adverse events (SAEs) related to DTX401 is conducted.

    Assessments include checking for specific effects such as increased liver enzyme levels, malignancy, nerve effects, and other potential risks associated with the therapy.

  4. Step 4

    Pregnancy monitoring

    Monitoring includes tracking pregnancy outcomes in patients or their partners who have been treated with DTX401.

  5. Step 5

    Nutritional and clinical assessments

    Nutritional assessments and various clinical evaluations are conducted, including laboratory tests and imaging.

    Patient experiences are documented through clinical interviews.

  6. Step 6

    Study duration

    The study is expected to continue until December 31, 2036.

    Recruitment for the study is anticipated to start on October 16, 2024.

Who can join the trial?

5 criteria

  • The patient must have received **DTX401** (either a full or partial dose) in a previous clinical study or have been prescribed **DTX401** in a real-world setting.
  • The patient must be willing and able to give their agreement to participate in the study after it has been explained to them. If the patient is a child or an adult with cognitive limitations, they must be willing to agree to participate, and a legally authorized representative must provide consent on their behalf.
  • The study is open to both male and female participants.
  • The study includes participants from different age groups, including children, teenagers, and adults.
  • The study may include individuals who are considered part of a vulnerable population, meaning they might need extra protection or care.

Who cannot join the trial?

5 criteria

  • Patients who do not have Glycogen Storage Disease Type Ia (GSDIa) cannot participate. This is a specific condition related to how the body stores sugar.
  • Patients who are not within the specified age ranges cannot participate. The study includes certain age groups, so if you are outside these, you may not be eligible.
  • Patients who are not part of the specified clinical trial groups cannot participate. This means the study is looking for people with certain characteristics or conditions.
  • Patients who are not female or male subjects cannot participate. The study is open to both genders.
  • Patients who are not considered part of a vulnerable population cannot participate. This refers to groups that might need special protection or consideration in research.
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Investigated drugs

DTX401 is a medication being studied for its long-term safety and effects on pregnancy outcomes in patients with Glycogen Storage Disease Type Ia (GSDIa). This trial aims to monitor how patients respond to the treatment over an extended period, focusing on any potential side effects and the health of pregnant patients receiving the medication.

What is already known about the treatment

DTX401 – DTX401 is administered through intravenous infusion, allowing the medication to be delivered directly into the bloodstream. It is currently under investigation in clinical trials, specifically for its long-term safety and effects on pregnancy outcomes in patients with Glycogen Storage Disease Type Ia (GSDIa). The main therapeutic indication for DTX401 is the treatment of GSDIa, a rare genetic disorder affecting glycogen metabolism. At the molecular level, DTX401 works by delivering a functional copy of the gene responsible for producing the enzyme glucose-6-phosphatase, which is deficient in individuals with GSDIa. This medication is classified as a gene therapy, aiming to correct the underlying genetic defect causing the disease.

Investigated diseases

Glycogen Storage Disease Type Ia (GSDIa) – This is a rare genetic disorder that affects the body's ability to break down glycogen into glucose, leading to an accumulation of glycogen in the liver and kidneys. It is caused by a deficiency of the enzyme glucose-6-phosphatase, which is crucial for glucose production. As a result, individuals with GSDIa often experience low blood sugar levels, especially between meals or during fasting. Over time, this can lead to symptoms such as an enlarged liver, growth delays, and increased levels of certain fats in the blood. The disease is typically diagnosed in infancy or early childhood, and its progression can vary depending on the severity of the enzyme deficiency. Regular monitoring and dietary management are essential to help manage the symptoms and prevent complications.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2023-510219-20-00Protocol codeDTX401-CL401Estimated enrolment148 patientsSponsorUltragenyx Pharmaceutical Inc.

sourced from the EU Clinical Trials Register and site verification

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