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

Verified siteRegistered drugNo 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 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.

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

    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. Step 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. Step 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. Step 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. Step 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. Step 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 trial?

8 criteria

  • 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 trial?

10 criteria

  • 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.
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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.

What is already known about the treatment

GNT0006 – This medication is administered intravenously and is currently being studied in clinical trials for its safety and effectiveness. It is designed to treat FKRP-related limb-girdle muscular dystrophy (LGMD R9), a genetic muscle disorder. GNT0006 works by using an adeno-associated viral vector to deliver the FKRP gene to patients, aiming to correct the underlying genetic defect. It falls under the category of gene therapy, which involves modifying or manipulating genes to treat or prevent disease.

Investigated diseases

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 detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2023-506677-36-00Protocol codeATA-001-FKRPEstimated enrolment9 patientsSponsorAtamyo Therapeutics

sourced from the EU Clinical Trials Register and site verification

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