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Study on the Effect and Safety of Autologous Mesoangioblasts for Patients with Mitochondrial Myopathy Due to m.3243A>G Mutation

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

The trial focuses on individuals who carry the m.3243A>G mutation causing mitochondrial myopathy, a rare condition that leads to weakness and easy tiring of the arm muscles. The experimental treatment uses a patient’s own stem‑like cells called autologous mesoangioblasts, which are prepared from the person’s own muscle tissue (autologous muscle precursor cells) and delivered directly into the blood vessel that supplies the upper arm using an intra‑arterial injection.

The main aim of the study is to determine whether three separate administrations of the cell therapy improve muscle strength and reduce fatigue in the treated arm. Participants will receive the first cell infusion, followed by two additional infusions at set intervals, with visits for safety checks and simple strength tests before the first dose and after the final dose.

Safety is closely monitored through imaging of the blood vessels (angiography), observation of neurological signs for several hours after each infusion, and recording of any serious problems. Muscle performance is measured with a device that records force output (Biodex dynamometer). Small samples of muscle tissue (muscle biopsy) are also taken to look at the amount of the genetic mutation and to assess the mitochondrial respiratory capacity, which reflects how well the muscle cells produce energy.

The research process

The trial runs in 9 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 criteria such as age (18-64 years), gender (male or female), and presence of the m.3243A>G mutation.

    Written informed consent is required to participate.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to evaluate muscle strength and fatigue in both arms using a device called a Biodex dynamometer.

    Baseline measurements are taken before any treatment is administered.

  3. Step 3

    First administration

    The first administration of autologous mesoangioblasts is performed via intra-arterial injection into the left upper arm.

    This involves a cell suspension specifically prepared for injection.

  4. Step 4

    Monitoring after first administration

    After the first administration, monitoring is conducted for 8 hours to check neurological vital signs and ensure no serious adverse events (SAEs) occur.

    Angiography may be used to assess vascular conditions.

  5. Step 5

    Second administration

    A second administration of the cell suspension is performed in the same manner as the first, targeting the left upper arm.

  6. Step 6

    Monitoring after second administration

    Similar to the first administration, monitoring is conducted for 8 hours post-procedure to ensure safety and check for any adverse effects.

  7. Step 7

    Third administration

    The third and final administration of the cell suspension is carried out, again targeting the left upper arm.

  8. Step 8

    Monitoring after third administration

    Post-procedure monitoring is conducted for 8 hours to ensure safety and check for any adverse effects.

  9. Step 9

    Follow-up assessment

    A follow-up assessment is conducted 4-6 weeks after the third administration to evaluate changes in muscle strength and fatigue using the Biodex dynamometer.

    Muscle biopsies may be taken to assess muscle mass, morphology, mutation load, and mitochondrial respiratory capacity.

Who can join the trial?

4 criteria

  • The patient must provide written informed consent, which means they agree to participate in the study after being fully informed about it.
  • The patient must be between the ages of 18 and 64.
  • Both males and females can participate in the study.
  • The patient must have a specific genetic change called the m.3243A>G mutation, which is related to a condition affecting the muscles known as mitochondrial myopathy.

Who cannot join the trial?

4 criteria

  • Patients who do not have the m.3243A>G mutation, which is a specific change in the genetic material that can cause a condition called mitochondrial myopathy, cannot participate.
  • Patients who are not within the specified age range for the study cannot participate. The age range is not specified in the provided data, but it is important for eligibility.
  • Patients who are part of a vulnerable population, which means they might need special protection or care, are not included in the study.
  • Patients who have any other medical conditions or factors that the study team believes would make it unsafe or inappropriate for them to participate cannot join the study.
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Investigated drugs

Autologous Myogenic Stem Cell Therapy is a treatment that uses stem cells derived from the patient's own body. In this trial, the therapy involves administering these stem cells directly into the arteries of the upper left arm. The goal is to improve muscle strength and reduce fatigue in patients with a specific genetic mutation known as m.3243A>G. The therapy is given three times, and its safety is monitored by checking for any serious adverse effects, blockages in blood vessels, and changes in neurological health.

What is already known about the treatment

Autologous Myogenic Stem Cell Therapy – This therapy is administered intra-arterially, specifically targeting the upper left arm. It is currently under investigation in clinical trials for its potential to treat mitochondrial myopathy caused by the m.3243A>G mutation. The main therapeutic indication is to improve muscle strength and reduce fatigue in affected individuals. At the molecular level, the therapy involves the use of autologous myogenic stem cells, which are believed to aid in muscle repair and regeneration. It falls under the pharmacological classification of advanced therapy medicinal products (ATMPs).

Investigated diseases

Mitochondrial Myopathy – This condition is caused by a genetic mutation known as m.3243A>G, which affects the mitochondria, the energy-producing structures within cells. It primarily impacts muscle cells, leading to muscle weakness and fatigue. As the disease progresses, individuals may experience increasing difficulty with physical activities due to the muscles' reduced ability to generate energy efficiently. The condition can also affect other systems in the body, depending on the extent of mitochondrial dysfunction. Symptoms can vary widely among individuals, even within the same family. The progression of the disease is often gradual, with symptoms worsening over time.
Trial detailsLast updated 9 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-515129-27-00Estimated enrolment20 patientsSponsorAcademisch Ziekenhuis Maastricht

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