This is a "Proof of concept" trial, Phase 2, and t
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Posted On: 03/08/2017 10:54:44 AM
Re: Loose Lips #32009
This is a "Proof of concept" trial, Phase 2, and there is NO control group because it would be utterly unethical to have sick people undergo an invasive procedure like a retention enema for 6 weeks, with just water and no drug (thus, NO chance of benefiting from the trial). Instead, they compare results to an untreated population, to see if it's significant.
I don't have the figures for spontaneous remission of ulcerative proctitis. But, I find it highly unlikely that in a 6-week period, every person with UP would improve significantly, and that half of them would have the disease go away.
However, according to the PR:
So it certainly appears that this is NOT a disease in which spontaneous remission is expected from half the patients in 6 weeks; nor would measurable improvement from all other patients be expected in that short time period. Rather, over time, 30-50 percent of patients get worse.
My GUESS is that after the remaining cohort of this Phase 2 proof-of-concept trial, the decision will be made whether to go ahead with a much larger-scale Phase 3 trial. With results like this at these lower doses (with Cohort 3 doubling the dose from this cohort), I'd be surprised if they didn't want to go ahead with Phase 3, and then to FDA approval.
I don't have the figures for spontaneous remission of ulcerative proctitis. But, I find it highly unlikely that in a 6-week period, every person with UP would improve significantly, and that half of them would have the disease go away.
However, according to the PR:
Quote:
...approximately 30-50 percent of patients developing more extensive UC. There is currently no cure. According to estimates provided by GlobalData, the worldwide UC market, which includes products for UP/UPS, is expected to increase at a compound annual growth rate of 4.7 percent, from $4.2 billion in 2012 to approximately $6.6 billion by 2022.
So it certainly appears that this is NOT a disease in which spontaneous remission is expected from half the patients in 6 weeks; nor would measurable improvement from all other patients be expected in that short time period. Rather, over time, 30-50 percent of patients get worse.
My GUESS is that after the remaining cohort of this Phase 2 proof-of-concept trial, the decision will be made whether to go ahead with a much larger-scale Phase 3 trial. With results like this at these lower doses (with Cohort 3 doubling the dose from this cohort), I'd be surprised if they didn't want to go ahead with Phase 3, and then to FDA approval.
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