drugset / Trial / NCT02710500
rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies
Non-randomizedSingle-groupQuadruple-blindTreatment
Summary
The proposed clinical trial is a double-blind, randomized controlled study with direct intramuscular injection of rAAVrh.74.MHCK7.DYSF.DV gene vector to the extensor digitorum brevis muscle (EDB). Two cohorts of subjects with dysferlin deficiency, each with proven mutations will undergo gene transfer. A minimum of three subjects will be enrolled into each cohort.
Timeline
- Start
- 2016-03
- Primary completion
- 2019-07
- Completion
- 2019-07
Publications
- Background Sondergaard PC, Griffin DA, Pozsgai ER, Johnson RW, Grose WE, Heller KN, Shontz KM, Montgomery CL, Liu J, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR. AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models. Ann Clin Transl Neurol. 2015 Mar;2(3):256-70. doi: 10.1002/acn3.172. Epub 2015 Jan 20.
- Background Grose WE, Clark KR, Griffin D, Malik V, Shontz KM, Montgomery CL, Lewis S, Brown RH Jr, Janssen PM, Mendell JR, Rodino-Klapac LR. Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer. PLoS One. 2012;7(6):e39233. doi: 10.1371/journal.pone.0039233. Epub 2012 Jun 15.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | rAAVrh74.MHCK7.DYSF.DV | Gene therapy (AAV / viral vector) | 2e+12 vg | Intramuscular |
| Subject | rAAVrh74.MHCK7.DYSF.DV | Gene therapy (AAV / viral vector) | 6e+12 vg | Intramuscular |