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Table 4 Design of tgAAC94 trials

From: Arthritis gene therapy's first death

   Number of patients  
Cohort Dose (DNase-resistant particles) Drug Placebo Repeat dose (drug only) Endpoints
Phase I (RAC 2003)     Primary
1 1010 6 2      Safety
2 1011 6 2   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)     
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
      Explore new outcome measures for single joints
         Patient assessment
         Functional assessment
         Joint inflammation and damage on magnetic resonance imaging (select subjects)
  1. RAC = Recombinant DNA Advisory Committee; TBD, to be determined – the highest safe dose, as determined from cohorts 1 to 3; TNFR:Fc = tumor necrosis factor receptor:Fc domain of immunoglobulin fusion protein.