Limb-girdle muscular dystrophies (LGMD) are a heterogeneous group of genetically determined muscle disorders with a primary or predominant involvement of the pelvic or shoulder girdle musculature. More than 20 genes with autosomal recessive (LGMD2A to LGMD2Q) and autosomal dominant inheritance (LGMD1A to LGMD1H) have been mapped/identified to date.
Clinical trials on LGMD ongoing at the Institute:
- Jain CO2: An International Clinical Outcome Study of Dysferlinopathy
- ATA-001-FKRP: A Phase 1-2 Multicenter Study (2 stages) to Evaluate the Safety and Efficacy of Intravenous GNT0006, Adeno-Associated Viral Vector Carrying the FKRP Gene, in Patiens with FKRP-Related Limb-Girdle Muscular Dystrophy (LGMD R9, formely LGMD2I)
- ATA-003-GSAR (Atamyo): A phase 1-2, open-label, dose escalation study to evaluate the safety of 3 doses of intravenous ATA-200, an adeno-associated viral vector carrying the human gamma-sarcoglycan gene, in patients with gamma-sarcoglycanopathy (LGMD R5, forely LGMD 2C)
Acronym |
Jain CO2 |
ATA-001-FKRP |
ATA-003-GSAR (Atamyo) |
| Intervention | – | GNT0006 | AT 132 |
| Pathology | LGMD R2 | LGMD R9 | LGMD R5 |
| Principal investigator | Tanya Stojkovic | Tanya Stojkovic | Marina Colella |
| Sponsor | AIM | ATAMYO Therapeutics | ATAMYO Therapeutics |
| Study status | Active | Active | In preparation |
| Recruitment status | Completed | Ongoing | Not started |
| Population | Adult | Adult | Child |
| + infos on clinicalmtrial.gov | + infos on clinicaltrial.gov | + infos on clinicaltrials.gov |
Contact: essais-adultes@institut-myologie.org
