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Grant: Operating Grant

Université Laval

$462,858.00

Phase I/II clinical trial of myoblast transplantation to Duchenne Muscular Dystrophy patients.

EF 2012-13

Résumé
The present submission for a Phase I/II of the clinical trial is to permit us to investigate whether the transplantation of normal myoblasts throughout one muscle (the extensor carpi radialis longus) of the patients will permit to increase the strength of that muscle. During this Phase I/II, the patients will be transplanted with myoblasts grown from the muscle biopsy of the donor and kept frozen in liquid nitrogen. Thirty million myoblasts will be injected per cm3 in a progressively higher surface of the radialis (i.e., 3, 7 and 10 cm2). The contralateral muscle will be injected with saline to serve as a negative control. The strength of both muscles will be measured at 3 months to verify whether the myoblast transplantation increase the strength of the muscle. If there is no significant strength increase the protocol will be terminated immediately for that patient. If there is a significant strength increase, the patient will be maintained under immunosuppression until 6 months and his strength will be re-evaluated.
Programme
Operating Grant
Bénéficiaire
Emplacement
Quebec, Canada
Type de bénéficiaire
Academia
Type
Grant
Source
CIHR Funding Decisions Database

Tel que publié

Id
279698
Name
Tremblay Jacques P
Region
Québec
Country
Canada
Orgtype
Institution Paid; Research Institution
Instname2
Musculoskeletal Health and Arthritis
Theme2
Clinical
Prcname2
Movement & Exercise
Projecttitle
Phase I/II clinical trial of myoblast transplantation to Duchenne Muscular Dystrophy patients.
Programtype2
Operating Grants
Orgnameinp2
Université Laval
Cihramount2
$462,858
Cihrequipment2
$0
Programname2
Operating Grant
Keyworddelim
Cell Therapy; Cell Transplantation; Clinical Trial; Duchenne Muscular Dystrophy
Abstract
The present submission for a Phase I/II of the clinical trial is to permit us to investigate whether the transplantation of normal myoblasts throughout one muscle (the extensor carpi radialis longus) of the patients will permit to increase the strength of that muscle. During this Phase I/II, the patients will be transplanted with myoblasts grown from the muscle biopsy of the donor and kept frozen in liquid nitrogen. Thirty million myoblasts will be injected per cm3 in a progressively higher surface of the radialis (i.e., 3, 7 and 10 cm2). The contralateral muscle will be injected with saline to serve as a negative control. The strength of both muscles will be measured at 3 months to verify whether the myoblast transplantation increase the strength of the muscle. If there is no significant strength increase the protocol will be terminated immediately for that patient. If there is a significant strength increase, the patient will be maintained under immunosuppression until 6 months and his strength will be re-evaluated.
Competitiondate
201303
Approvedterm2
3 yrs 0 mth
Pinamesdelim
Tremblay, Jacques P
Approvedterm
3 ans 0 mois
Cihrcontribution2
~ $462,858~ $0~