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Dmd06-Mab

A groundbreaking clinical trial is underway to evaluate the safety and efficacy of DMD06-Mab, an innovative cell therapy for non-ambulant patients with Duchenne Muscular Dystrophy (DMD). This phase I/IIa study aims to restore dystrophin production in affected muscles through a single injection of genetically modified autologous mesoangioblasts (MABs). The trial focuses on patients with exon 51 skippable mutations and could potentially offer new hope for those living with this challenging condition.

Table of Contents

What is DMD06-MAB?

DMD06-MAB is an experimental treatment being studied for Duchenne Muscular Dystrophy (DMD). It is classified as an advanced therapy medicinal product, specifically a type of cell therapy.[1] This means it uses cells that have been modified to potentially treat the disease.

Target Condition: Duchenne Muscular Dystrophy

DMD06-MAB is being developed to treat Duchenne Muscular Dystrophy. DMD is a genetic disorder that causes progressive muscle weakness and loss of muscle mass. It primarily affects boys and is usually diagnosed in early childhood.[1]

How DMD06-MAB Works

DMD06-MAB works through a process called cell-mediated exon skipping. Here’s a simplified explanation of how it’s intended to work:

  1. Cells called mesoangioblasts (MABs) are taken from the patient’s own body.
  2. These cells are genetically modified in a laboratory to produce a special type of RNA that can “skip” a problematic part of the dystrophin gene (specifically, exon 51).
  3. The modified cells are then injected back into the patient’s muscles.
  4. The goal is for these cells to help the body produce a functional form of the dystrophin protein, which is missing or defective in patients with DMD.[1]

Clinical Trial Details

The clinical trial for DMD06-MAB is a Phase I/IIa study, which means it’s an early-stage trial primarily focused on safety and initial signs of effectiveness. Key details include:

  • It’s a non-randomized, open-label study.
  • The trial plans to enroll five non-ambulant patients (patients who can no longer walk) with DMD.
  • Each patient will receive a single injection of the modified cells into a specific foot muscle called the Extensor Digitorum Brevis (EDB).[1]

Eligibility Criteria

To participate in this trial, patients must meet certain criteria. Some key inclusion criteria are:

  • Age between 12 and 18 years old
  • Unable to walk at the time of recruitment
  • Confirmed diagnosis of DMD with a specific type of genetic mutation (one that can be helped by skipping exon 51)
  • Muscle degeneration not exceeding 50% reduction of muscle mass (as determined by an MRI scan)[1]

There are also several exclusion criteria, which are conditions that would prevent a person from participating in the trial. These include certain infections, severe scoliosis, significant heart or lung problems, and participation in other clinical trials.

Safety and Efficacy Measures

The trial has two main goals:

  1. Safety: Researchers will monitor for any side effects or adverse events for one year after the injection.
  2. Efficacy: They will check if the treatment leads to the production of dystrophin (the protein missing in DMD) in the injected muscle. The goal is to see at least 10% of the amount found in a healthy person’s muscle.[1]

How DMD06-MAB is Administered

DMD06-MAB is given as a suspension for injection. In this trial, it will be administered through a single intramuscular injection into a foot muscle. This means the medication is injected directly into the muscle tissue.[1]

Aspect Details
Study Type Phase I/IIa, non-randomized, open-label
Intervention DMD06-Mab (genetically modified autologous mesoangioblasts)
Administration Single intramuscular injection into foot muscle
Target Population Non-ambulant DMD patients, ages 12-18, with exon 51 skippable mutations
Primary Objectives Safety assessment and efficacy in restoring dystrophin production
Follow-up Period One year for safety, three months for efficacy
Key Exclusion Criteria Severe scoliosis, significant organ dysfunction, immune disorders, ongoing participation in other trials

FAQ

  • What is DMD06-Mab? DMD06-Mab is a cell therapy product consisting of genetically modified autologous mesoangioblasts (MABs). These cells are taken from the patient, modified to skip exon 51 of the dystrophin gene, and then injected back into the patient's muscles to potentially restore dystrophin production.
  • Who is eligible for this clinical trial? The trial is open to non-ambulant patients with Duchenne Muscular Dystrophy aged 12 to 18 years who have confirmed exon 51 skippable mutations in the dystrophin gene. Participants must have less than 50% reduction in muscle mass and meet other specific health criteria.
  • How is DMD06-Mab administered? DMD06-Mab is administered through a single intramuscular injection into the foot medial Extensor Digitorum Brevis (EDB) muscle. This targeted approach allows researchers to assess the therapy's effects on a specific muscle group.
  • What are the main objectives of this clinical trial? The primary objectives are to assess the safety of DMD06-Mab by monitoring adverse events and to evaluate its efficacy in restoring dystrophin production. The goal is to achieve at least 10% of normal dystrophin levels in the treated muscle.
  • How long will participants be monitored after receiving DMD06-Mab? Participants will be monitored for adverse events for one year following the injection of DMD06-Mab. Additionally, a muscle biopsy will be performed three months after the injection to assess dystrophin production.

Ongoing Clinical Trials on Dmd06-Mab

  • Study on DMD06-MAB Injection for Duchenne Muscular Dystrophy in Non-Ambulant Patients

    Not recruiting

    2 1 1
    Investigated drugs:
    Italy

Glossary

  • Duchenne Muscular Dystrophy (DMD): A genetic disorder characterized by progressive muscle degeneration and weakness, caused by mutations in the dystrophin gene.
  • Mesoangioblasts (MABs): A type of stem cell that can differentiate into various cell types, including muscle cells. In this trial, they are used as a vehicle for gene therapy.
  • Exon skipping: A therapeutic approach that aims to 'skip' over faulty sections of genetic code, potentially allowing the production of a partially functional protein.
  • Dystrophin: A protein crucial for maintaining muscle fiber strength and stability. Its absence or dysfunction leads to DMD.
  • Autologous: Derived from the same individual. In this context, it refers to using the patient's own cells for treatment.
  • Non-ambulant: Unable to walk independently, typically referring to patients who require a wheelchair for mobility.
  • Intramuscular injection: Administration of a substance directly into a muscle.
  • Extensor Digitorum Brevis (EDB): A small muscle in the foot used in this trial for the injection of DMD06-Mab.
  • Phase I/IIa study: An early-stage clinical trial that assesses both the safety and initial effectiveness of a new treatment.
  • Adverse events: Any unfavorable or unintended sign, symptom, or disease associated with the use of a medical treatment.

References

  1. http://clinicaltrials.eu/trial/study-on-dmd06-mab-injection-for-duchenne-muscular-dystrophy-in-non-ambulant-patients/