Table 4 Design of tgAAC94 trials
| Â | Â | Number of patients | Â | ||
|---|---|---|---|---|---|
Cohort | Dose (DNase-resistant particles) | Drug | Placebo | Repeat dose (drug only) | Endpoints |
Phase I (RAC 2003) | Â | Â | Â | Primary | |
1 | 1010 | 6 | 2 |  |    Safety |
2 | 1011 | 6 | 2 | Â | Secondary |
3 | 1012 | 6 | 2 |  |    Changes in tenderness and swelling; injected and noninjected joints |
4 | TBD | 6 | 2 |  |    American College of Rheumatology and Disease Activity Score scoring |
|  |  |  |  |  |    Joint fluid cell counts |
|  |  |  |  |  |    TNFR:Fc levels in joint fluid and serum |
|  |  |  |  |  |    Serum neutralizing antibodies to adeno-associated virus serotype 2 |
|  |  |  |  |  |    Presence of tgAAC94 in peripheral blood mononuclear cells |
Phase I/II (RAC 2007) | Â | Â | Â | Â | |
1 | 1011 | 15 | 5 | 20 | Primary |
2 | 1012 | 15 | 5 | 20 |    Safety |
3 | 1013 | 15 | 5 | 20 | Secondary |
|  |  |  |  |  |    Tenderness and swelling of injected joint |
4 | 1011 | 15 | 5 | 20 |    Time to second injection of study drug |
5 | 1012 | 15 | 5 | 20 |    Overall disease activity |
6 | 1013 | 15 | 5 | 20 |    TNFR:Fc protein levels in serum and synovial fluid |
|  |  |  |  |  |    Serum anti-adeno-associated virus capsid neutralizing titers |
| Â | Â | Â | Â | Â | Explore new outcome measures for single joints |
|  |  |  |  |  |    Patient assessment |
|  |  |  |  |  |    Functional assessment |
|  |  |  |  |  |    Joint inflammation and damage on magnetic resonance imaging (select subjects) |