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Study on the Safety of ATA-200 Gene Therapy for Patients with Limb-Girdle Muscular Dystrophy Type R5

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 rare genetic condition called limb-girdle muscular dystrophy type R5 (LGMDR5), which affects the muscles around the hips and shoulders. The study is testing a new treatment called ATA-200, which is a type of gene therapy. Gene therapy involves using a specially designed virus to deliver a healthy copy of a gene into the body. In this case, the virus used is called adeno-associated virus serotype 8, and it carries the human gamma-sarcoglycan gene, which is important for muscle function.

The purpose of the study is to evaluate the safety of two different doses of ATA-200 when given through an intravenous infusion, which means it is delivered directly into the bloodstream. The study will involve children who can walk and have been diagnosed with LGMDR5. Participants will receive the treatment and be monitored for any side effects or changes in their health. The study aims to find the best dose for future research.

Throughout the study, participants will undergo various assessments to check their muscle function and overall health. These assessments include physical exams, muscle strength tests, and imaging tests like MRI to look at the muscles. The study will also collect information on how the treatment affects the participants' daily activities and quality of life. The goal is to ensure the treatment is safe and to gather information that could help improve the lives of people with LGMDR5 in the future.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, diagnosis of limb-girdle muscular dystrophy type R5, and ability to perform specific physical tasks.

    A blood test is performed to ensure the absence of antibodies against the viral vector used in the treatment.

  2. Step 2

    Treatment administration

    The treatment involves the administration of ATA-200, a gene therapy delivered through an intravenous infusion. This is done in a controlled setting to monitor safety and tolerability.

    Two different dosage levels are tested to determine the most effective and safe dose for future studies.

  3. Step 3

    Monitoring and follow-up

    Regular follow-up visits are scheduled to monitor the patient's health and response to the treatment. This includes physical examinations, vital signs checks, and assessments of muscle function and strength.

    Additional tests such as muscle imaging and biopsies are conducted to evaluate the treatment's impact on muscle tissue.

  4. Step 4

    Safety and efficacy evaluation

    Throughout the study, any adverse effects are recorded, and the overall safety of the treatment is assessed.

    The effectiveness of the treatment is evaluated through various tests, including muscle function tests, respiratory assessments, and patient-reported outcomes.

  5. Step 5

    Study completion

    The study is expected to conclude by January 2030. At this point, a final evaluation is conducted to summarize the findings and determine the recommended dose for future research.

Who can join the trial?

6 criteria

  • Children who can walk, both boys and girls, aged at least 6 but less than 12 years old at the time of screening.
  • Must have a confirmed diagnosis of **limb-girdle muscular dystrophy type R5 (LGMDR5)** before turning 10 years old. This diagnosis is based on symptoms and a genetic test showing changes in the **SGCG gene**.
  • Must be able to walk 10 meters (about 33 feet) in 15 seconds or less without needing help like a cane, crutches, or special leg braces. They should also be able to stand up from a regular chair with or without using their arms.
  • Must have signed a written agreement to participate in the study before any study-related activities begin.
  • The child's health should be stable enough, and both the child and their parents or legal guardians should be able to follow the study schedule and requirements, according to the study doctor.
  • Must not have certain antibodies, called **neutralizing antibodies against AAV8**, in their blood. These antibodies could affect how the treatment works.

Who cannot join the trial?

9 criteria

  • Patients who have a different type of muscular dystrophy other than limb-girdle muscular dystrophy type R5 cannot participate. This is a specific type of muscle disease.
  • Patients who are not able to walk on their own are excluded. The study is for those who can walk.
  • Patients who have other serious health conditions that might interfere with the study are not allowed to join. This means if you have another major illness, you might not be able to participate.
  • Patients who have had a recent surgery or are planning to have surgery soon cannot take part. This is to ensure safety during the study.
  • Patients who are taking certain medications that could affect the study results are excluded. Some medicines might interfere with the study treatment.
  • Patients who have participated in another clinical trial recently are not eligible. This is to avoid any overlap with other treatments.
  • Patients who have allergies to the study medication or its ingredients cannot participate. This is to prevent allergic reactions.
  • Patients who are pregnant or breastfeeding are not allowed to join the study. This is to protect the health of the mother and baby.
  • Patients who are unable to follow the study procedures or instructions are excluded. This means you need to be able to understand and follow the study rules.
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Investigated drugs

ATA-200 is an experimental therapy being tested in this clinical trial. It is an adeno-associated viral vector that carries the human gamma-sarcoglycan gene. This therapy is designed to help patients with gamma-sarcoglycanopathy, a type of limb-girdle muscular dystrophy known as LGMDR5. The goal of the trial is to evaluate the safety and tolerability of this treatment when given intravenously to pediatric patients. The study aims to determine the best dose for future research.

What is already known about the treatment

ATA-200 – This medication is administered intravenously and is currently being studied in clinical trials for its safety and tolerability. It is designed to treat limb-girdle muscular dystrophy type R5, a genetic disorder affecting muscle function. ATA-200 works by using an adeno-associated viral vector to deliver the human gamma-sarcoglycan gene, which is crucial for muscle integrity. It falls under the category of gene therapy, aiming to correct the underlying genetic defect causing the disease.

Investigated diseases

Limb-Girdle Muscular Dystrophy Type R5 – This is a genetic disorder characterized by progressive muscle weakness, primarily affecting the muscles around the hips and shoulders. Over time, individuals with this condition may experience difficulty in performing tasks that require muscle strength, such as climbing stairs or lifting objects. The disease progresses as muscle tissue is gradually replaced by fat and connective tissue, leading to further loss of muscle function. Symptoms often begin in childhood or adolescence and can vary in severity among affected individuals. As the condition advances, it may also impact respiratory and cardiac muscles, although the extent can differ. The progression of muscle weakness is typically slow, but it can lead to significant physical limitations over time.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase I/IITrial ID2023-506440-16-00Protocol codeATA-003-GSAREstimated enrolment5 patientsSponsorAtamyo Therapeutics

sourced from the EU Clinical Trials Register and site verification

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