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02609 THE HEC PLACEBO: DESIGNED FOR NO EFFECT Moench, Thomas R.* Vehicle controls are commonly used as placebos for two reasons: the vehicle (usually) provides a good physical match to the active product, and a vehicle placebo provides a comparison arm in which the absence of the active ingredient is the only difference between arms. Despite these common reasons for using the vehicle as the placebo, another consideration is arguably more important: the placebo must not cause unintended protection, and it certainly must not cause toxicity that increases susceptibility. Here we describe a placebo designed to minimize both protective and toxic effects. The design considerations included using: 1) a non-ionic gelling agent, HEC, (hydroxyethylcellulose) with no intrinsic microbicide properties (avoiding potentially active polyanionic gelling agents); 2) a preservative that is non-virucidal, non-inflammatory, and metabolizable (sorbic acid); 3) a formulation that has negligible buffering capacity (to avoid acid-mediated microbicidal effects even when formulated at pH ~4 to match the pH of the healthy vagina); and 4) a formulation that is isotonic to avoid causing an osmotic stress to the epithelium (and also to avoid the hypertonicity of many existing vehicle formulations, since hypertonic formulations cause osmosis that dilutes the gel and increases leakage from the vagina). In vitro studies document that the HEC Placebo has minimal cytocidal activity (by sperm motility and vaginal epithelial cell MTT assays), minimal stimulation of a proinflammatory response (IL-1 alpha release by vaginal epithelial cells), and minimal HIV inhibitory activity (2h virus-compound-cell incubation). In all four assays, the HEC Placebo had even less effect than K-Y® Jelly, a product that is generally assumed to be inert, and is commonly used as a control gel. In vivo experiments show that the HEC Placebo was not protective in an HSV-2 mouse vaginal challenge model. Moreover, unlike detergent spermicide/microbicides, when given 12 hours prior to HSV-2 challenge, it did not induce an increase in susceptibility to HSV-2. The minimal effects of the HEC Placebo should make it useful as a control gel for microbicide safety and efficacy trials Dr. Thomas R. Moench |
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