Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet)

Launched by SIMONE SPULER, MD · Oct 18, 2022

Keywords

ClinConnect Summary

This clinical trial, called GenPHSats-bASKet, is exploring a new treatment using gene-edited muscle stem cells for patients with Limb-Girdle Muscular Dystrophy (LGMD), a rare disease that affects muscle strength. It’s a first-of-its-kind study in humans designed to see if this therapy is safe and effective. The trial is currently not recruiting participants, but it will focus on individuals aged 14 and older who have been diagnosed with LGMD and have a specific gene defect that can be targeted for editing.

To participate, candidates must be receiving treatment at the Charité, Universitätsmedizin Berlin, and must be able to provide signed consent. However, certain health conditions may exclude individuals from the trial, such as chronic diseases or specific heart and airway issues. Those who qualify will have the chance to contribute to important research that could lead to new options for treating LGMD. Participants can expect to receive close medical supervision throughout the study to ensure their safety and well-being.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • LGDM diagnosed,
• • Identified gene defect location and gene editing proved feasible,
• • Age ≥14 years,
• • Patient in treatment in the department at Charité, Universitätsmedizin Berlin, Muscle Research Unit and Outpatient Clinic for Muscle Disorders,
• • Signed informed consent
• Exclusion Criteria:
• • Acute or chronic inflammatory local or systemic disease
• • Coagulation disorder
• • Known complications due to local anesthesia,
• • Congenital heart defect, cardiac arrhythmia,
• • Pathology of the airways such as micrognathia
• • Pierre Robin Sequence
• • Central hypoventilation syndrome/Ondine syndrome
• • Significant other medical or psychiatric illness
• • Positive serology for HIV and/or hepatitis A, B, C
• • Pregnant or lactating women
• • Known allergic reaction to constituents of the cryopreservation medium