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

NCT05588401 · Status: UNKNOWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2023-08-03

No results posted yet for this study

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

Interventions

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.

Sponsors & Collaborators

  • Simone Spuler, MD

    lead OTHER

Principal Investigators

  • Simone Spuler, Prof Dr med · Charite Universitätsmedizin Berlin, Germany

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
14 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-07-01
Primary Completion
2025-07-01
Completion
2025-07-01

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05588401 on ClinicalTrials.gov