NCT05588401

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

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

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Simone Spuler, MD · Academic / Other
• Sex: All
• Age: 14 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• LGMD

Interventions

Type	Name	Description
BIOLOGICAL	GenPHSat injection (Safety)	Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the left biceps muscle.
BIOLOGICAL	GenPHSat injection (Efficacy)	Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the right biceps muscle.
OTHER	Muscle Biopsy (Safety)	Participants undergo muscle biopsy 3 month after safety GenPHSat injection.

Timeline

• Start date: 2024-07-01
• Primary completion: 2025-07-01
• Completion: 2025-07-01
• First posted: 2022-10-20
• Last updated: 2023-08-03