Not recruitingRare disease

Long-Term Safety Study of Avalglucosidase Alfa for Patients with Pompe Disease in France

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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 the long-term safety and effectiveness of a treatment for Pompe disease, also known as Glycogen storage disease type II. The treatment being tested is called avalglucosidase alfa, which is also known by the code name GZ402666. This medication is given as a solution through an intravenous infusion, which means it is administered directly into the bloodstream through a vein.

The purpose of the study is to assess the long-term safety of avalglucosidase alfa in patients who have previously participated in other studies related to this treatment. Participants in this study will receive the medication over a period of time, and their health will be monitored to identify any adverse events, which are any unwanted effects that might occur during the treatment. The study will also evaluate the participants' ability to walk a certain distance in six minutes, their lung function, and their overall quality of life. For those with late-onset Pompe disease, additional assessments will include motor function tests and specific quality of life evaluations. For those with infantile-onset Pompe disease, evaluations will include a pediatric disability inventory and a heart function test.

This study is an extension of previous research and will continue until the medication is reimbursed in France or until December 2025, whichever comes first. Participants will be closely monitored throughout the study to ensure their safety and to gather important data on the effectiveness of avalglucosidase alfa in managing Pompe disease.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Participation begins after completing a previous study related to Pompe disease.
Eligibility requires a negative pregnancy test for females of childbearing potential and agreement to use contraception if sexually active.
Step 2
Treatment administration
The medication used is Nexviadyme, which contains the active substance avalglucosidase alfa.
It is administered as a solution for infusion through an intravenous route.
The frequency and dosage are determined by the study protocol and medical team.
Step 3
Monitoring and assessments
Regular monitoring for any adverse events, including reactions to the infusion, is conducted.
Assessments include a six-minute walk test, motor function tests, and pulmonary function tests.
Quality of life is evaluated using specific surveys and scales tailored to Pompe disease.
Step 4
Study duration
The study continues until the medication is reimbursed in France or until December 2025, whichever occurs first.
Participation involves regular visits and assessments as outlined in the study protocol.

Who can join the trial?

5 criteria

The patient must have a condition called **Glycogen storage disease type II** and have completed one of the following studies in France: EFC14028, LTS13769, or ACT14132.
The patient or their parent/legal guardian must be willing and able to sign a document called **informed consent**, which shows they understand the study and agree to participate. If the patient is under 18 years old, they should also agree to participate if they are able to do so.
The patient and their legal guardian, if the patient is under 18, must be able to follow the rules and procedures of the study.
If the patient is a female who can have children, she must have a negative result on a pregnancy test at the start of the study. This test checks for a hormone called **beta-human chorionic gonadotropin (β-HCG)** in urine, which indicates pregnancy.
Female patients who can have children and male patients who are sexually active must either not engage in sexual activity or use two effective methods of birth control to prevent pregnancy.

Who cannot join the trial?

5 criteria

Patients who have not completed one of the previous studies: EFC14028, LTS13769, or ACT14132.
Patients who have not been diagnosed with Glycogen storage disease type II, a condition where the body has trouble breaking down a type of sugar called glycogen.
Patients who are not within the specified age ranges for the study.
Patients who are not able to follow the study procedures or attend the required visits.
Patients who have any other medical condition that might interfere with the study or make it unsafe for them to participate.

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Verified sites

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

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Assistance Publique Hopitaux De Marseille
Marseille, France
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CHU Gabriel-Montpied
Clermont Ferrand, France
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Centre Hospitalier Lyon Sud
Pierre Benite, France

Trial locations

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Investigated drugs

Avalglucosidase alfa is a medication used in the treatment of Pompe disease, a rare genetic disorder. This medication helps to replace an enzyme that is missing or not working properly in people with this condition. The trial aims to assess the long-term safety and effectiveness of this medication in patients who have previously participated in related studies.

Avalglucosidase Alfa

What is already known about the treatment

Avalglucosidase alfa – Avalglucosidase alfa is administered intravenously and is currently being studied in a Phase 4 clinical trial to evaluate its long-term safety and efficacy for treating Pompe disease, also known as glycogen storage disease type II. This medication is designed to replace the deficient enzyme in patients, helping to break down glycogen accumulation in cells. It is classified as an enzyme replacement therapy and works by targeting and degrading glycogen deposits, thereby improving muscle function and reducing disease symptoms.

Investigated diseases

Glycogen storage disease type II – Also known as Pompe disease, this is a genetic disorder that affects the body's ability to break down glycogen, a stored form of sugar used for energy. The disease is caused by a deficiency of the enzyme acid alpha-glucosidase, leading to the accumulation of glycogen in the body's cells. This accumulation primarily affects muscle tissues, including the heart and skeletal muscles, causing them to weaken over time. Symptoms can vary depending on the age of onset, with infantile-onset forms presenting more severe symptoms such as muscle weakness, poor muscle tone, and heart problems. In late-onset forms, symptoms may include progressive muscle weakness, respiratory issues, and difficulty walking. The progression of the disease can vary widely among individuals, with some experiencing rapid deterioration and others having a slower progression.