Phase1/2-Cohort2a vs. Phase2a-Cohort2
The amended Phase1/2 safety protocol with legally blind DAMD participants ‘no better than 20/400 vision acuity’ for the treated eye and legally blind SMD participants ‘no better than hand motion visual acuity’ for the treated eye that allows an efficacy trial Cohort2a no better than 20/100 visual acuity patients is a spectacular acceleration, allowed by the regulatory authorities, towards potential approval IMO of a Commercial BLA. Normally such a jump from safety international Clinical Trials Phase1/2 to Efficacy Clinical Trials Phase2a requires a new trial with new FDA/EMA agreed protocol and typically an elapsed time of anywhere between 3 months to 1 year after the Phase1/2 safety is completed. This new efficacy Cohort 2a 100K trial is an unprecedented acceleration introduced by the regulatory authorities based on Phase1/2 observed safety and unexpected efficacy results for Cohorts1(50K) & 2 (100K)in both the DAMD and SMD clinical trials.
The modified safety Clinical Trial Phase1/2 continues as follows.
“This study is a Phase1/2, open-label, non randomized, sequential, multi-center clinical trial. There will be 5 cohorts, the 4 low vision cohorts will contain 3 patients, the better vision cohort will contain 4 patients.”
4 new patients will then be recruited for the new better vision cohort for 100K in both the DAMD and SMD Phase1/2
Hopefully with safety established in the soon 3 patients for Cohort 3(150K) and 3 patients for Cohort 4(200K) along with efficacy established in the soon 4 patients for Cohort 2a(100K), the regulatory authorities DSMB, IRB, FDA, and EMA will allow the new efficacy trials to be expanded for Cohort 3a (150K) and Cohort 4a(200K) for better vision patients with 20/100 visual acuity.
