Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet)
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|ClinicalTrials.gov Identifier: NCT05588401|
Recruitment Status : Not yet recruiting
First Posted : October 20, 2022
Last Update Posted : October 24, 2022
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|Condition or disease||Intervention/treatment||Phase|
|LGMD||Biological: GenPHSat injection (Safety) Biological: GenPHSat injection (Efficacy) Other: Muscle Biopsy (Safety)||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||6 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase 1/2a First-in-human Trial Evaluating Autologous Gene-edited Muscle Stem Cells in Limb Girdle Muscular Dystrophies (GenPHSats-bASKet)|
|Estimated Study Start Date :||July 1, 2023|
|Estimated Primary Completion Date :||July 1, 2025|
|Estimated Study Completion Date :||July 1, 2025|
Experimental: 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.
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.
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Through study completion, an average of 1 year ]Characterization of type, incidence, severity, duration, reversibility, treatability of adverse events recorded at least at visit 1, 2, 3 and 4 (end of trial).
- Rate of muscle biopsy post-injection structure [ Time Frame: 3 month post injection ]The muscle biopsy taken and stained to visualize muscle biopsy structure after 3 month post injection.
- Rate of Muscle force [ Time Frame: Until 6 month post injection ]Muscle force measurement data taken at screening (baseline), visit 1, 2, 3 and 4 (end of trial).
- Rate of Blood CK [ Time Frame: Until 6 month post injection ]CK blood determination at screening (baseline), visit 1, 2, 3 and 4 (end of trial).
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|Ages Eligible for Study:||14 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05588401
|Contact: Simone Spuler, Prof Dr firstname.lastname@example.org|
|Contact: Christian Witzel, Dr med||+49 30 email@example.com|
|Study Chair:||Simone Spuler, Prof Dr med||Charite Universitätsmedizin Berlin, Germany|
|Responsible Party:||Simone Spuler, MD, Clinical Professor, Charite University, Berlin, Germany|
|Other Study ID Numbers:||
|First Posted:||October 20, 2022 Key Record Dates|
|Last Update Posted:||October 24, 2022|
|Last Verified:||October 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Undecided|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|