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Typically in Stage 1 trials, they test safety effectiveness by giving doses of varying size, often times below the level of clinical effectiveness. They also gi
by mikeyouse 10y ago
Typically in Stage 1 trials, they test safety effectiveness by giving doses of varying size, often times below the level of clinical effectiveness. They also give these to much smaller populations, usually fewer than 25 people. The main goal of these trials is to figure out "safety and dosage" -- so they measure side effects at each dosage and try to discern interactions with the body.
They'll be especially careful with the CRISPR drugs due to the disasters that Gene Therapy trials have previously undergone. Most notably, TGN1412 was given at a dosage about 500x smaller than the safe dosage in animals but caused almost immediate cytokine storms that nearly killed all 6 participants in the trial.
- jessriedel 10y ago> they test safety effectiveness by giving doses of varying size, often times below the level of clinical effectiveness. OK, good point. But even then there needs to be a reasonable belief that either (1) the likelihood of clinical effectiveness is nonzero, and is still worth trying compared to the smaller chance of bad effects, or (2) the same patient will be able to receive a clinically effective dose in the future based on the safety information gained testing the smaller, clinically ineffective dose on them.