ATA-001-FKRP -A Phase 1-2 GMO Study of GNT0006
Research type
Research Study
Full title
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 patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I)
IRAS ID
306837
Contact name
Sophie Olivier
Contact email
Sponsor organisation
Atamyo Therapeutics
Eudract number
2021-004276-33
Clinicaltrials.gov Identifier
Duration of Study in the UK
7 years, 0 months, 1 days
Research summary
FKRP-related limb-girdle muscular dystrophy (LGMD R9) is a genetic disorder caused by mutations in the FuKutin-Related Protein (FKRP) gene responsible for producing the protein called FKRP, which participates in the stability and resistance of the muscles, in particular during effort. The deficiency in FKRP results in a progressive loss of strength of the muscles of the hip, thighs (pelvic girdle) and shoulder areas (scapular girdle). Respiratory and cardiac symptoms can also develop. This trial aims to test a gene therapy in patients suffering from a moderate form of LGMD R9, to find out whether it is safe to deliver the studied drug into the bloodstream via intravenous infusion and if this treatment can improve or correct disease symptoms.
This trial is divided into two stages: Some patients will participate in the first part (called Stage 1) where the purpose is to determine the optimal study drug dose to administer. Other patients will participate in the second part (called Stage 2) where the purpose is to assess the effectiveness of the selected dose of study drug. In any case, all participants in this trial will receive a potentially effective dose of the experimental study drug.
This study is investigating a gene therapy called GNT0006. It consists in administering in the bloodstream a normal copy of the faulty (mutated) FKRP gene, packaged in a harmless virus (adeno-associated virus AAV9), called a vector. This is the first time it will be administered in humans. In the second part of this trial, GNT0006 will be compared to a placebo (an inactive copy of the study drug).All subjects will be followed for up to 5-6 years after active GNT0006 administration.
REC name
North East - York Research Ethics Committee
REC reference
22/NE/0003
Date of REC Opinion
18 Feb 2022
REC opinion
Further Information Favourable Opinion