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NCT05588401
Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet)
Phase 1, PHASE2 trial testing GenPHSat injection (Safety) in LGMD in 6 participants. Status unknown.
1 July 2025
Quick facts
| Lead sponsor | Simone Spuler, MD |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Status unknown |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | sequential |
| Masking | none |
| Primary purpose | other |
| Enrollment | 6 |
| Start date | 1 July 2024 |
| Primary completion | 1 July 2025 |
| Estimated completion | 1 July 2025 |
Drugs / interventions tested
- GenPHSat injection (Safety) — full drug profile →
- GenPHSat injection (Efficacy) — full drug profile →
- Muscle Biopsy (Safety)
Conditions studied
- LGMD — all drugs for LGMD →
Sponsor
Simone Spuler, MD — full company profile →
Who can join
14 and older, any sex, with LGMD. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
7 peer-reviewed publications reference this trial (live from Europe PMC):
-
Gene therapy for genetic diseases: challenges and future directions.
Qie B, Tuo J, Chen F, Ding H, et al · · 2025 · cited 14× · PMID 39949979 · DOI 10.1002/mco2.70091 -
Advancing gene editing therapeutics: Clinical trials and innovative delivery systems across diverse diseases.
Raigani M, Eftekhari Z, Adeli A, Kazemi-Lomedasht F. · · 2025 · cited 5× · PMID 40896588 · DOI 10.1016/j.omtn.2025.102666 -
Cell therapy for Duchenne muscular dystrophy: promises, challenges, and controversies.
Łoboda A, Dulak J. · · 2025 · cited 1× · PMID 41074937 · DOI 10.1007/s00018-025-05904-5 -
Clinical Trial Landscape of Gene-Edited Autologous Hematopoietic Stem Cells for Hemoglobinopathies and Immunodeficiencies.
O'Hanlon Cohrt K, O'Dea S. · · 2026 · PMID 42074029 · DOI 10.3390/ijms27083384 -
Progress on cell therapy for skeletal muscle disorders.
Azzag K, Perlingeiro RCR. · · 2026 · PMID 41871765 · DOI 10.1016/j.addr.2026.115859 -
Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group
Cavazza A, Molina-Estévez F, Reyes Á, Ronco V, et al · · 2025 -
Undetected Neuromuscular Disease in Patients after Heart Transplantation.
Bekele BM, Gazzerro E, Schoenrath F, Falk V, et al · · 2024 · PMID 39063061 · DOI 10.3390/ijms25147819
Verify or expand the search:
- PubMed search for NCT05588401
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for LGMD
Currently open trials in the same condition.
- NCT06363357 — The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients Wi · NA · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT05588401 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Simone Spuler, MD
- Last refreshed: 3 August 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05588401.
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