Phase 1/2a First-in-human Trial Evaluating Autologous Gene-edited Muscle Stem Cells in Limb Girdle Muscular Dystrophies (GenPHSats-bASKet)

Trial ID: NCT05588401

Interventional - Phase 1/2 - Unknown status

This study is an investigator initiated first-in-human interventional open label phase 1/2a clinical trial investigating an ATMP in the orphan disease LGMD to evaluate safety and efficacy.

Conditions

• LGMD

Interventions

• GenPHSat injection (Safety)
• GenPHSat injection (Efficacy)
• Muscle Biopsy (Safety)

Eligibility

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

Sponsor

Simone Spuler, MD

Source: Clinicaltrials.gov

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