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| Safety Symposium on a Serious Adverse Event on OBA Protocol 705 |
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In July 2007 a young woman who was enrolled in a gene transfer trial for individuals with rheumatoid arthritis died of multiorgan failure approximately 3 weeks after receiving the second dose of the study agent. The trial, NIH OBA protocol #0504-705, titled
A Phase I/II Study of Repeat Intra-articular Administration of tgAAC94, A Recombinant Adeno-Associated Vector Containing the TNFR:Fc Fusion Gene, in Inflammatory Arthritis with and without Concurrent TNF-α Antagonist,
was immediately placed on clinical hold by the FDA.
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On September 17, 2007 the RAC convened a panel of outside experts and heard presentations from the Principle Investigator, clinicians and pathologists from the University of Chicago and experts in rheumatoid arthritis, infectious diseases, hematology, hepatology and gene transfer. On December 3, 2007 the RAC continued its discussion of this case, focusing primarily on whether the gene transfer played a role in the subject's death. On January 14, 2008 the RAC convened a special meeting to review its final conclusions.
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The RAC concluded that:
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It is the Committee's opinion that this patient's unfortunate death was primarily a result of an opportunistic infection, disseminated histoplasmosis, as well as bleeding complications and multi-organ failure. Her apparent risk factor for histoplasmosis was her systemic rheumatoid arthritis therapy, chiefly the TNF-antagonist adalimumab. The contribution of an immune response to the AAV vector could not be evaluated.
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