Combined CTIMP and companion diagnostic trial: Use of Elecsys Anti-AAVrh74 assay in ENVOL study
Research type
Research Study
Full title
Measuring of anti-AAVrh74 antibody levels using Elecsys® Anti-AAVrh74 assay in patients serum samples for the BN43881 Phase II clinical study
IRAS ID
331338
Contact name
Laurent Servais
Contact email
Sponsor organisation
Roche Diagnostics GmbH
Clinicaltrials.gov Identifier
1006080, IRAS project ID for ENVOL (BN43881) application; CIV-23-05-043014, Eudamed CIV for RD006698 study; 2022-000691-19, EudraCT for ENVOL study
Duration of Study in the UK
4 years, 0 months, 30 days
Research summary
The Pharma Partner (PP) is developing a gene therapy treatment, called delandistrogene moxeparvovec which is based on recombinant adeno-associated viral vector (AAVrh74). Delandistrogene moxeparvovec is a gene therapy designed to treat patients with Duchenne Muscular Dystrophy. DMD is a rare neuromuscular disorder that occurs due to mutations in the Dystrophin gene which prohibit proper production of the dystrophin protein. This protein is required to stabilize muscle fibers and it’s absence results in progressive loss of motility and, in most cases, death in the late teens/early 20’s due to cardiac arrest. Elevated neutralizing antibody levels against recombinant AAVrh74 vector are an exclusion criterion for enrollment in the clinical trial subsequent treatment with delandistrogene moxeparvovec. Therefore, following the collaboration with PP, RD006698 study will support patient screening for the ENVOL Phase II clinical study (BN43881) by investigating the delandistrogene moxeparvovec treatment using Elecsys® Anti-AAVrh74 assay. All clinical samples will be collected under the ENVOL clinical protocol and measured prospectively with the Elecsys® Anti-AAVrh74 assay under RD006698 clinical performance evaluation study protocol.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
23/NW/0228
Date of REC Opinion
21 Aug 2023
REC opinion
Favourable Opinion