DISEASE CLASSIFICATION(S):
Duchenne Muscular Dystrophy(DMD)
NAME OF CLINICAL TRIAL/STUDY:
Dystrophin gene transfer by intramuscular injection -- phase 1 safety trial in DMD
TRIAL UPDATES:
April 2010
In April 2010, the investigators presented results from this trial at the annual meeting of the American Academy of Neurology. In the six trial participants, the dystrophin gene therapy was safe, but four of the participants showed immune responses to the newly created dystrophin. The extent to which these immune responses will affect gene therapy development for DMD are not yet clear.
See Caution: Immune Response Seen in DMD Gene Therapy.
May 2009
The injection of three additional participants has not yet taken place. There is some uncertainty about the next step in this gene transfer study.
July 2008
This trial is now recruiting three additional participants, who will receive a higher dosage level of transferred dystrophin genes.
January 2008
Trial clinician Jerry Mendell, M.D., of Columbus (Ohio) Children’s Research Institute, Columbus Children’s Hospital and Ohio State University College of Medicine, announced that the dystrophin (Biostrophin) injections were safe and well tolerated in six participants at two dosage levels. He said that the investigators will continue the trial with three additional participants at a higher dosage level. The trial is not recruiting new participants at this time.
PURPOSE AND RATIONALE:
The gene for dystrophin, a muscle protein, is flawed in DMD. The safety and feasibility of injecting miniaturized but probably functional dystrophin genes inside adeno-associated viral shells will be tested. The investigators will also see whether the transferred genes produce dystrophin after being injected into muscle tissue.
STUDY DETAILS:
In this MDA-supported trial, six to nine boys with DMD will undergo injections of dystrophin genes inside viral particles (Biostrophin, developed by Asklepios Biopharmaceutical in Chapel Hill, N.C.) in one biceps muscle and placebo (sham, look-alike) injections into the other biceps. Neither they nor the investigators will know which arm got which type of injection until after results are analyzed. Muscle biopsies, blood tests and other clinical evaluations will be part of this study.
Each group of three boys will be given a higher dosage of the dystrophin genes.
OPENING DATES:
March 2006
CLOSING DATES:
Uncertain
TARGET NUMBER OF PARTICIPANTS:
6-9
RECRUITMENT STATUS:
Open
