welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!

Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy

key information

study id #: NCT02354781

condition: Duchenne Muscular Dystrophy

status: completed


The proposed clinical trial is an outgrowth of the safety record and functional improvement seen in the BMD follistatin gene therapy trial. In this study the investigators propose to inject AAV1.CMV.huFS344 at a total dose of 2.4E12 vg/kg to six DMD patients. This dose will be divided between gluteal muscles, quadriceps and tibialis anterior. This is a wider distribution of vector than given to BMD patients, who overall improved the distance walked on the 6MWT without adverse events related to viral transduction into a single muscle.

intervention: rAAV1.CMV.huFollistin344

mechanism of action: Vectored gene therapy to introduce a version of dystrophin

results: https://clinicaltrials.gov/ct2/show/results/NCT02354781

last updated: April 19, 2020