Completed trials
Gene therapy for Duchenne muscular dystrophy
|
Investigator |
Prof Fardeau |
|
Sponsor |
Transgene |
|
CCPPRB |
Yes |
|
Patients numbers
Total
A the institut of
Myology |
9
9 |
|
Projected dates
Begining
Ending |
2000
2002 |
|
Financing |
AFM |
Summary
- Aims
To evaluate the feasibility and tolerance (phase I) of the intramuscular injection of a naked DNA carrying the full-length human dystrophin gene in 9 patients affected with Duchenne or Becker muscular dystrophy. The DNA was administered in increasing doses: 200 mg, 600 mg and 2 x 600 mg.
Results
Tolerance to the administration was good, with no adverse general or immune reactions, even at maximum dose. Dystrophin expression was found in 6 out of the 9 patients treated, following a muscle biopsy carried out 3 weeks after injection.
- Publication
Romero NB, Braun S, Benviste O, Leturq F, Hogrel JY, Morris GM, Barois A, Eymard B, Payan C, Ortega V, Boch AL, Lejean M, Thioudellet C, Mourot B, Escot C, Choquel A, Recan D, Kaplan J-C, Dickson G, Klatzmann D, Molinier-Frenckel V, Guillet J-G, Squiban P, Herson S, Fardeau M: Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy.
Human Gene Therapy, 2004, 15: 1065-1076,O.
