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.
This trial is directed towards a first-in-human application of GenPHSats; gene edited primary human satellite cell derived muscle stem cells as a new Advanced Therapy Medicinal Product (ATMP) in a phase 1/2a clinical trial with Gene edited PHSats (GenPHSats) initiating healthy muscle development in patients with LGDM. The trial is set up to verify if GenPHSats can provide an therapy option for LGDM patients as there is currently no therapy available. The GenPHSats are an autologous product comprised of primary human satellite cell derived muscle stem cells obtained from the patient's own muscle tissue and gene edited in vitro prior to transplantation.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
6
Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the left biceps muscle.
Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the right biceps muscle.
Participants undergo muscle biopsy 3 month after safety GenPHSat injection.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Characterization of type, incidence, severity, duration, reversibility, treatability of adverse events recorded at least at visit 1, 2, 3 and 4 (end of trial).
Time frame: Through study completion, an average of 1 year
Rate of muscle biopsy post-injection structure
The muscle biopsy taken and stained to visualize muscle biopsy structure after 3 month post injection.
Time frame: 3 month post injection
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