Gene Therapy Study for Patients with Limb-Girdle Muscular Dystrophy Using GNT0006

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What is this study about?

This clinical trial is focused on studying a condition known as FKRP-related limb-girdle muscular dystrophy, which is a type of muscle disease that affects the muscles around the hips and shoulders. The study is testing a new treatment called GNT0006, which is a gene therapy. This treatment uses a special virus, called an adeno-associated virus, to deliver a gene that may help improve muscle function in people with this condition. The purpose of the study is to evaluate the safety and effectiveness of this treatment.

The study is divided into two stages. In the first stage, participants will receive the GNT0006 treatment through an intravenous infusion, which means it will be given directly into a vein. This stage will help determine the best dose of the treatment. In the second stage, the selected dose will be used to further assess how well the treatment works over a period of one year. Some participants may receive a placebo, which is a substance with no active ingredients, to compare the effects of the treatment.

Participants in the study will undergo various assessments to monitor changes in their muscle function and overall health. These assessments may include tests of muscle strength and function, as well as imaging tests like MRI to look at the muscles. The study will also track changes in respiratory function and other health markers. The goal is to see if the GNT0006 treatment can improve the symptoms of FKRP-related limb-girdle muscular dystrophy and enhance the quality of life for those affected by this condition.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria such as age, diagnosis, and ability to perform certain physical tasks.

Informed consent is required before any study-related procedures begin.

2 stage 1: safety and tolerability assessment

The first stage involves the administration of GNT0006, a gene therapy delivered intravenously.

Two different dosage levels are tested to assess safety and tolerability in patients with limb-girdle muscular dystrophy.

The goal is to determine the recommended dose for the next stage.

3 stage 2: efficacy demonstration

In the second stage, the efficacy of the selected dose of GNT0006 is evaluated over a period of one year.

The primary focus is on changes in lung function, specifically the forced vital capacity (FVC) percentage, compared to a placebo group.

4 secondary assessments

Additional assessments include changes in muscle function, MRI parameters, respiratory assessments, and muscle biomarkers.

Patient-reported outcomes and quality of life are also evaluated.

5 medication details

The study involves the use of GNT0006 administered intravenously.

Other medications such as methylprednisolone and prednisolone may be used as part of the study protocol.

6 study duration

The estimated end date for the study is February 1, 2029.

The recruitment for the study began on February 1, 2022.

Who Can Join the Study?

Must be a male or female who is at least 16 years old.
Must have a confirmed diagnosis of LGMDR9, which is a type of muscle disorder, based on symptoms and genetic testing showing changes in the FKRP gene.
Must be able to walk 10 meters (about 33 feet) in 30 seconds with help, like using a cane or crutches, or devices below the knees.
Must be able to stand up from a regular chair with or without using arm support.
Must have some weakness in the diaphragm, which is a muscle that helps with breathing, shown by a forced vital capacity (FVC) between 40% and 80% of what is expected. FVC is a test that measures how much air you can blow out of your lungs.
Must agree to use effective birth control methods.
Must sign a written consent form agreeing to participate in the study before any study-related activities begin.
Must have a stable medical condition and be able, along with parents or legal guardians, to follow the study schedule and requirements, as judged by the study doctor.

Who Cannot Join the Study?

Patients who are not able to walk on their own.
Patients who have other serious health conditions that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial recently.
Patients who have allergies to the study medication or its ingredients.
Patients who have a history of drug or alcohol abuse.
Patients who are unable to follow the study procedures or instructions.
Patients who have received certain medications that might affect the study results.
Patients who have a history of certain heart problems.
Patients who have an infection that requires treatment.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Rigshospitalet	Copenhagen	Denmark
Ujneinvsrv Hygdrabrs Piqyp Starwtebghd Chfxcnk Fsgj	Paris	France

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	01.02.2022
France	Not recruiting	01.02.2022

Trial locations

Investigated drugs:

GNT0006 is an experimental therapy being tested in this clinical trial. It is an adeno-associated viral vector that carries the FKRP gene. This therapy is designed to be administered intravenously to patients with FKRP-related limb-girdle muscular dystrophy (LGMDR9). The goal of the trial is to evaluate the safety and effectiveness of this gene therapy in improving the condition of patients with this specific type of muscular dystrophy.

FKRP-related limb-girdle muscular dystrophy (LGMD R9) – This is a genetic disorder characterized by progressive muscle weakness, primarily affecting the muscles around the hips and shoulders. It is caused by mutations in the FKRP gene, which leads to abnormal muscle function and structure. Over time, individuals with this condition may experience difficulty with activities such as climbing stairs, lifting objects, or walking. The progression of muscle weakness can vary, with some individuals experiencing a slow decline in muscle strength, while others may have a more rapid progression. As the disease advances, it can also affect respiratory muscles, leading to breathing difficulties. This condition is considered a rare disease, affecting a small number of individuals worldwide.

Trial ID:

2023-506677-36-00

Protocol code:

ATA-001-FKRP

NCT ID:

NCT05224505

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Gene Therapy Study for Patients with Limb-Girdle Muscular Dystrophy Using GNT0006

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