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

NCT ID: NCT05588401

Last Updated: 2023-08-03

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-07-01
• Study Completion Date: 2025-07-01

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: OTHER
• Blinding Strategy: NONE

Study Groups

GenPHSat safety injection and GenPHSat efficacy injection

Initial intervention with six injections into the left biceps. A second intervention with 36 injections into the right biceps.

Group Type: EXPERIMENTAL

GenPHSat injection (Safety)

Intervention Type: BIOLOGICAL

Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the left biceps muscle.

GenPHSat injection (Efficacy)

Intervention Type: BIOLOGICAL

Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the right biceps muscle.

Muscle Biopsy (Safety)

Intervention Type: OTHER

Participants undergo muscle biopsy 3 month after safety GenPHSat injection.

Interventions

GenPHSat injection (Safety)

Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the left biceps muscle.

Intervention Type: BIOLOGICAL

GenPHSat injection (Efficacy)

Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the right biceps muscle.

Intervention Type: BIOLOGICAL

Muscle Biopsy (Safety)

Participants undergo muscle biopsy 3 month after safety GenPHSat injection.

Intervention Type: OTHER

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

• Minimum Eligible Age: 14 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Simone Spuler, MD

OTHER

Sponsor Role: lead

Responsible Party

Simone Spuler, MD

Clinical Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Simone Spuler, Prof Dr med

Role: STUDY_CHAIR

Charite Universitätsmedizin Berlin, Germany

Central Contacts

Simone Spuler, Prof Dr med

Role: CONTACT

simone.spuler@charite.de

004930450540501

Christian Witzel, Dr med

Role: CONTACT

christian.witzel@charite.de

+49 30 553016

Other Identifiers

bASKet

Identifier Type: -

Identifier Source: org_study_id