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

NCT05588401 | Unknown Phase 1 DMC

• 1 other identifier: bASKet
• Study Type: interventional
• Target: 6
• Locations: 0 countries
• Sites: N/A

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.

Conditions

LGMD

Outcome Measures

Primary Outcomes (1)

• 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).

  Through study completion, an average of 1 year

Secondary Outcomes (1)

• Rate of muscle biopsy post-injection structure

  3 month post injection

Other Outcomes (2)

• Rate of Muscle force

  Until 6 month post injection

• Rate of Blood CK

  Until 6 month post injection

Study Arms (1)

GenPHSat safety injection and GenPHSat efficacy injection

EXPERIMENTAL

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

Biological: GenPHSat injection (Safety); Biological: GenPHSat injection (Efficacy); Other: Muscle Biopsy (Safety)

Interventions

GenPHSat injection (Safety) BIOLOGICAL

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

GenPHSat safety injection and GenPHSat efficacy injection

GenPHSat injection (Efficacy) BIOLOGICAL

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

GenPHSat safety injection and GenPHSat efficacy injection

Muscle Biopsy (Safety) OTHER

Participants undergo muscle biopsy 3 month after safety GenPHSat injection.

GenPHSat safety injection and GenPHSat efficacy injection

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Simone Spuler, MD: lead

MeSH Terms

Interventions

Safety

Intervention Hierarchy (Ancestors)

Accident Prevention; Accidents; Public Health; Environment and Public Health

Study Officials

• Simone Spuler, Prof Dr med

  Charite Universitätsmedizin Berlin, Germany

  STUDY CHAIR

Central Study Contacts

Simone Spuler, Prof Dr med

CONTACT

004930450540501; simone.spuler@charite.de

Christian Witzel, Dr med

CONTACT

+49 30 553016; christian.witzel@charite.de

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: October 14, 2022
• First Posted: October 20, 2022
• Study Start: July 1, 2024
• Primary Completion: July 1, 2025
• Study Completion: July 1, 2025
• Last Updated: August 3, 2023

Record last verified: 2023-08