Dose escalation* and revision
| Group/no. patients . | Dose/site, vg† . | Dose/kg, vg‡ . | Total dose, vg1-153 . | Legs injected . |
|---|---|---|---|---|
| Original outline for dose escalation | ||||
| 1/3 | 2.3 × 1012 | 2.0 × 1011 | 1.4 × 1013 | One or both |
| 2/3 | 7.0 × 1012 | 2.0 × 1012 | 1.4 × 1014 | Both |
| 3/3 | 3.5 × 1013 | 1.0 × 1013 | 7.0 × 1014 | Both |
| Revised plan used in clinical study | ||||
| 1/3 | 1.5 × 1012 | 2.0 × 1011 | 1.4 × 1013 | One or both |
| 2/3 | 1.5 × 1012 | 6.0 × 1011 | 4.2 × 1014 | Both |
| 2/2 | 1.5 × 1012 | 1.8 × 1012 | 1.3 × 1014 | Both1-155 |
| Group/no. patients . | Dose/site, vg† . | Dose/kg, vg‡ . | Total dose, vg1-153 . | Legs injected . |
|---|---|---|---|---|
| Original outline for dose escalation | ||||
| 1/3 | 2.3 × 1012 | 2.0 × 1011 | 1.4 × 1013 | One or both |
| 2/3 | 7.0 × 1012 | 2.0 × 1012 | 1.4 × 1014 | Both |
| 3/3 | 3.5 × 1013 | 1.0 × 1013 | 7.0 × 1014 | Both |
| Revised plan used in clinical study | ||||
| 1/3 | 1.5 × 1012 | 2.0 × 1011 | 1.4 × 1013 | One or both |
| 2/3 | 1.5 × 1012 | 6.0 × 1011 | 4.2 × 1014 | Both |
| 2/2 | 1.5 × 1012 | 1.8 × 1012 | 1.3 × 1014 | Both1-155 |
After the death of a patient in a gene transfer trial, the sponsors and investigators in this trial voluntarily slowed the pace of dose escalation from a one-log increase between the low and mid-dose cohorts to 1/2 log between each dose cohort.
Vector genomes.
Dosing performed according to the patient weight obtained at the time of injection
Assuming a 70-kg adult
Plus auxiliary muscles (deltoid, soleus).