Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02710500 | rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies | PHASE1 | COMPLETED | 2 | — | — | Mar 1, 2016 | Jul 1, 2019 | May 13, 2021 | 1 | United States |
Defined as the occurrence of any one Grade III or higher, unanticipated, treatment-related toxicity
| Arm | Type | Description |
|---|---|---|
| Cohort 1 (Low Dose) | EXPERIMENTAL | Three (n=3) dysferlin deficiency subjects will receive bilateral injections with one extensor digitorum brevis muscle (EDB) receiving the rAAVrh74.MHCK7.DYSF.DV and the other side receiving saline alone.Subjects will receive a total dose of 2 x 10\^12 in one muscle. Intervention Drug: rAAVrh.MHCK7.DYSF.DV |
| Cohort 2 (High Dose) | EXPERIMENTAL | Three (n=3) dysferlin deficiency subjects will receive bilateral injections with one extensor digitorum brevis muscle (EDB) receiving the rAAVrh74.MHCK7.DYSF.DV and the other side receiving saline alone.Subjects will receive a total dose of 6 x 10\^12 vg in one muscle. Intervention Drug: rAAVrh74.MHCK7.DYSF.DV |
| Name | Type | Description |
|---|---|---|
| rAAVrh74.MHCK7.DYSF.DV | DRUG | Biological/Vaccine: rAAVrh74.MHCK7.DYSF.DV Recombinant adeno-associated virus carrying a dysferlin transgene under control of a muscle specific MHCK7 promoter. |
Inclusion Criteria: * Must be Non-ambulant (cannot walk 10 meters in ≤ 30 sec) and age 18 years or older * Established mutations of the dysferlin gene on both alleles * Impaired muscle function but with sufficient muscle preservation to ensure muscle transfection based on magnetic resonance image o...