Ann: Phase I Clinical Trial of R327 I.V. - 6000mg Complete, page-18

@andrewk65 The phase 1 is about toxicity of the drug not whether it works. in the phase I study it has been shown to not be toxic at a dosage rate that is expected (from the invitro and animal studies) to be high enough to be effective against pathogens. This study will suffice for any indication with an IV route of administration.

Phase II will be a safety and efficacy trial, designed to demonstrate that it is safe in a human study group that is sick and it will give an indication of the optimal dosage that will be subsequently tested in a blinded placebo controlled trial with greater numbers to prove with an acceptable confidence level that it works. I suspect that it likely to be in a group that is suffering from septicemia, as giving sepsis patients an unproven treatment may be a death sentence and wouldn't be ethical. Conversely, I suppose it is possible that the treatment might be given in addition to standard of care, but this is probably unlikely as the toxicity of the polypharmacy wouldn't be known.

Phase II is likely to occur in multiple hospitals. Septicemia / Sepsis doesn't happen with a high frequency in any one hospital so to get numbers quickly enough it would likely be a multi-centre trial. Septicemia / Sepsis are serious life threatening conditions and patients will almost certainly be in critical care environments in hospital not in an outpatient environment.

It is also quite possible that it will be trialed against escape pathogens on less time critical infections. Once proven there it could be moved into the sepsis population with knowledge that it works against the array of pathogens that are targeted in sepsis.
Nice situation to be in to be thinking of the next trial design. Come on Recce the world needs you to succeed.