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Posted On: 04/04/2019 10:48:37 PM
Post# of 72446
That's a good question. Some here could probably answer this better than I can.
My understanding is the type of study suggested by Dr Bertilino could be a stage 0 trial which is described below. I think these studies could also be done quickly in research centers both private and university level with strong institutional control committees. The stage 2 Brilacidin trial for ABSSSI established the safety of Brilacidin and the Stage 2 Brilacidin trial for Ulcerative Proctitis and Oral Mucositis were well tolerated which would make achieving approval for early IBD pharmokinetic studies for oral Brilacidin much easier.
https://journals.lww.com/oncology-times/Fullt...ls_.6.aspx
https://www.profil.com/knowledge-center/trial-stages#
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2435428/
Phase 0 trials are designed primarily to evaluate the pharmacodynamic and/or pharmacokinetic properties of selected investigational agents prior to initiating more traditional phase 1 testing. One of the major objectives of phase 0 trials is to interrogate and refine a target or biomarker assay for drug effect in human samples implementing procedures developed and validated in preclinical models. Thus, close collaboration between laboratory scientists and clinical investigators is essential to the design and conduct of phase 0 trials. Given the relatively small number of patients and tissue samples, demonstrating a significant drug effect in phase 0 trials requires precise and reproducible assay procedures and innovative statistical methodology. Furthermore, phase 0 trials involving limited exposure of study agent administered at low doses and/or for a short period allows them to be initiated under the FDA Exploratory IND Guidance with less preclinical toxicity data than usually required for traditional first-in-human studies. Because of the very limited drug exposure, phase 0 trials offer no chance of therapeutic benefit, which can impede patient enrollment, particularly if invasive tumor biopsies are required. However, the challenges to accrual are not insurmountable, and well-designed and executed phase 0 trials are feasible and have great potential for improving the efficiency and success of subsequent trials, particularly those evaluating molecularly targeted agents.
My understanding is the type of study suggested by Dr Bertilino could be a stage 0 trial which is described below. I think these studies could also be done quickly in research centers both private and university level with strong institutional control committees. The stage 2 Brilacidin trial for ABSSSI established the safety of Brilacidin and the Stage 2 Brilacidin trial for Ulcerative Proctitis and Oral Mucositis were well tolerated which would make achieving approval for early IBD pharmokinetic studies for oral Brilacidin much easier.
https://journals.lww.com/oncology-times/Fullt...ls_.6.aspx
https://www.profil.com/knowledge-center/trial-stages#
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2435428/
Phase 0 trials are designed primarily to evaluate the pharmacodynamic and/or pharmacokinetic properties of selected investigational agents prior to initiating more traditional phase 1 testing. One of the major objectives of phase 0 trials is to interrogate and refine a target or biomarker assay for drug effect in human samples implementing procedures developed and validated in preclinical models. Thus, close collaboration between laboratory scientists and clinical investigators is essential to the design and conduct of phase 0 trials. Given the relatively small number of patients and tissue samples, demonstrating a significant drug effect in phase 0 trials requires precise and reproducible assay procedures and innovative statistical methodology. Furthermore, phase 0 trials involving limited exposure of study agent administered at low doses and/or for a short period allows them to be initiated under the FDA Exploratory IND Guidance with less preclinical toxicity data than usually required for traditional first-in-human studies. Because of the very limited drug exposure, phase 0 trials offer no chance of therapeutic benefit, which can impede patient enrollment, particularly if invasive tumor biopsies are required. However, the challenges to accrual are not insurmountable, and well-designed and executed phase 0 trials are feasible and have great potential for improving the efficiency and success of subsequent trials, particularly those evaluating molecularly targeted agents.
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