LGMDPhase 1/2NCT05588401
Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet)
A Phase 1/2 study testing GenPHSat injection (Safety), GenPHSat injection (Efficacy) and Muscle Biopsy (Safety) in people with LGMD, run by Simone Spuler, MD.
Trial 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. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- LGMD
- What is being tested?
- GenPHSat injection (Safety), GenPHSat injection (Efficacy) and Muscle Biopsy (Safety)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- 1 Jul 2024to 1 Jul 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Phase 1/2a First-in-human Trial Evaluating Autologous Gene-edited Muscle Stem Cells in Limb Girdle Muscular Dystrophies (GenPHSats-bASKet)
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.
Read the 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
- Being tested
- GenPHSat injection (Safety), GenPHSat injection (Efficacy), Muscle Biopsy (Safety)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GenPHSat injection (Safety)Biological
Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the left biceps muscle.
GenPHSat injection (Efficacy)Biological
Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the right biceps muscle.
Muscle Biopsy (Safety)Other
Participants undergo muscle biopsy 3 month after safety GenPHSat injection.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: GenPHSat injection (Safety); Biological: GenPHSat injection (Efficacy); Other: Muscle Biopsy (Safety) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Through study completion, an average of 1 year
Secondary outcomes (1)
Rate of muscle biopsy post-injection structure
The muscle biopsy taken and stained to visualize muscle biopsy structure after 3 month post injection.
Measured: 3 month post injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2022
- Study start1 Jul 2024 (estimated)
- Primary completion1 Jul 2025 (estimated)
- Study completion1 Jul 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LGMDStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Simone Spuler, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Simone Spuler, MD, Charite University, Berlin, Germany
- Investigators
- Simone Spuler, Prof Dr med, Study chair, Charite Universitätsmedizin Berlin, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05588401
- Sponsor's protocol ID
- bASKet
- Status last verified
- Aug 2023
- First submitted
- 14 Oct 2022
- Last updated
- 3 Aug 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2a First-in-human Trial Evaluating Autologous Gene-edited Muscle Stem Cells in Limb Girdle Muscular Dystrophies (GenPHSats-bASKet)
- ClinicalTrials.gov ID
- NCT05588401
- Acronym / sponsor ID
- bASKet
- Conditions
- LGMD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Simone Spuler, MDUniversity, hospital or other
- Study start
- 1 Jul 2024 (estimated)
- Primary completion
- 1 Jul 2025 (estimated)
- Study completion
- 1 Jul 2025 (estimated)
- First posted
- 20 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GenPHSat safety injection and GenPHSat efficacy injection | Experimental | Biological: GenPHSat injection (Safety), Biological: GenPHSat injection (Efficacy), Other: Muscle Biopsy (Safety)Initial intervention with six injections into the left biceps. A second intervention with 36 injections into the right biceps. |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GenPHSat injection (Safety)Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the left biceps muscle. | Biological | GenPHSat safety injection and GenPHSat efficacy injection |
| GenPHSat injection (Efficacy)Participants receive gene edited primary human muscle stem cells (GenPHsat) injection in the right biceps muscle. | Biological | GenPHSat safety injection and GenPHSat efficacy injection |
| Muscle Biopsy (Safety)Participants undergo muscle biopsy 3 month after safety GenPHSat injection. | Other | GenPHSat safety injection and GenPHSat efficacy injection |
Primary outcome
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).
1 secondary outcome
Rate of muscle biopsy post-injection structure
Time frame: 3 month post injection
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2023.
- Individual participant dataSharing planned: Undecided
Related trials for LGMD
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Study of BBP-418 in Patients With LGMD2I
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A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
Limb Girdle Muscular Dystrophy
RecruitingObservational study
Long-Term Development of Muscular Dystrophy Outcome Assessments
LGMD1B, LGMD1C
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Simone Spuler, Prof Dr med study chair
Charite Universitätsmedizin Berlin, Germany
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing GenPHSat injection (Safety), GenPHSat injection (Efficacy) and Muscle Biopsy (Safety) in people with LGMD, run by Simone Spuler, MD.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Simone Spuler, Prof Dr med (004930450540501, simone.spuler@charite.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: Through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Aug 2023.