Not recruitingRare disease

Study on the Effects of Pariglasgene Brecaparvovec for Adults with Glycogen Storage Disease Type Ia

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 is focused on studying a rare condition known as Glycogen Storage Disease Type Ia (GSDIa). This disease affects how the body stores and uses sugar, leading to low blood sugar levels and other health issues. The study is investigating a treatment called Pariglasgene brecaparvovec, which is a solution given through an infusion into the veins. This treatment involves a special type of virus that helps transfer a gene called Glucose-6-Phosphatase (G6Pase) into the body. The goal is to see if this gene transfer can help improve the condition in adults with GSDIa.

The purpose of the study is to evaluate the long-term safety of this treatment, known by its code name DTX401, after a single dose is given. Participants in the study will receive the treatment and be monitored over time to see how their bodies respond. The study will look at any side effects that occur and how they relate to the treatment. Additionally, researchers will observe changes in the time it takes for participants to experience low blood sugar events during a controlled fasting period.

This study is part of ongoing research to better understand and manage Glycogen Storage Disease Type Ia. By participating, researchers hope to gather important information that could lead to improved treatments for this condition in the future. The study is expected to continue until 2026, 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.

Step 1
Joining the study
Participation begins after receiving a single dose of DTX401 in a previous study (Study 401GSDIA01).
Written informed consent is required before any study-related procedures.
Step 2
Initial assessment
An initial assessment is conducted to evaluate the current health status.
This includes reviewing medical history and any previous reactions to DTX401.
Step 3
Administration of medication
Pariglasgene brecaparvovec is administered as a solution for infusion through an intravenous route.
This is a one-time administration aimed at transferring the gene for Glucose-6-Phosphatase (G6Pase).
Step 4
Monitoring and follow-up
Regular follow-up visits are scheduled to monitor the long-term safety of DTX401.
The primary focus is on observing any adverse events (AEs) or serious adverse events (SAEs).
Step 5
Evaluation of efficacy
The study evaluates the change in time to the first hypoglycemic event during a controlled fasting challenge.
This is assessed over time following the intravenous administration of DTX401.
Step 6
Completion of study
The study is estimated to conclude by November 3, 2026.
Final assessments will be conducted to summarize the findings related to safety and efficacy.

Who can join the trial?

3 criteria

Must have received **DTX401** in a previous study called **Study 401GSDIA01**.
Must be willing and able to provide written permission to participate after understanding the study details. This is called **informed consent**.
Must be willing and able to attend all scheduled study visits and follow all procedures and requirements.

Who cannot join the trial?

3 criteria

Patients who are not adults cannot participate. This means only adults are allowed.
Patients who are not diagnosed with Glycogen Storage Disease Type Ia cannot participate. This is a specific condition related to how the body stores sugar.
Patients who are part of a vulnerable population cannot participate. This refers to groups of people who might need special protection or care.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Universidade De Santiago De Compostela
Verified
Santiago De Compostela, Spain
Universitair Medisch Centrum Groningen
Verified
Groningen, The Netherlands

Trial locations

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Investigated drugs

DTX401 is a gene therapy being studied for its potential to treat Glycogen Storage Disease Type Ia (GSDIa). This therapy uses a virus called Adeno-Associated Virus Serotype 8 (AAV8) to deliver a healthy copy of the gene responsible for producing the enzyme glucose-6-phosphatase (G6Pase). The goal of this therapy is to help the body produce the enzyme it lacks, which is essential for breaking down glycogen into glucose, thereby improving the symptoms and health of individuals with GSDIa.

Pariglasgene Brecaparvovec

What is already known about the treatment

DTX401 – DTX401 is administered as a single intravenous (IV) dose and is currently being studied in clinical trials for its safety and efficacy. It is primarily being investigated for the treatment of Glycogen Storage Disease Type Ia, a rare genetic disorder. The medication works by using an adeno-associated virus (AAV) vector to deliver a functional copy of the glucose-6-phosphatase (G6Pase) gene to the liver, aiming to correct the underlying enzyme deficiency at the molecular level. DTX401 falls under the pharmacological classification of gene therapy, representing a novel approach in the treatment of genetic disorders.

Investigated diseases

Glycogen storage disease type Ia – This is a genetic disorder that affects the body's ability to break down glycogen into glucose, which is necessary for energy. It is caused by a deficiency of the enzyme glucose-6-phosphatase, leading to the accumulation of glycogen in the liver and kidneys. Symptoms often include low blood sugar, an enlarged liver, and growth delays. Over time, individuals may experience issues with maintaining normal blood sugar levels, especially during fasting. The disease can also lead to increased levels of certain fats in the blood and may affect kidney function. It is typically diagnosed in infancy or early childhood.