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Deferred Pediatric Study Telecon, February, 23, 2012
Participants: Sponsor and FDA (Agnes Lim, Ilan Irony, John Scott)
Re: BLA 125400 – Deferred Pediatric Study
Background: On 2/16/2012, the sponsor had a meeting with FDA on a matter unrelated to this BLA. Following the meeting, the sponsor spoke to me about the deferred pediatric study for BLA 125400. After looking further into the matter, they now believed that they would not be able to enroll enough subjects for they study they had committed to on 1/27/2012. If that was their position, I asked them to send in an official submission stating their position (submission#31).
Teleconference minutes and FDA’s action to submission#31:
On internal review of this submission, with FDA CDRH consultation with Dr. Robert Betz, and SGE periodontal consultants Dr. Mark Reynolds, FDA believes that the study is feasible, and should be conducted. These views are consistent with those stated at the CTGT AC on 11/17/2011 for this BLA. Furthermore, FDA identified the following deficiencies with the pediatric protocol that was submitted on 1/27/2012 (submission#25), and requested the following protocol revisions:
1) Conduct the study in subjects between 12-18 years old
2) Change the primary efficacy endpoint to AG instead of KT; change the success rate from 50% to 65%; sample size will be ~75 evaluable subjects
3) Either use a two-sided test at 0.5 significance, or a one-sided test at 0.025
4) Submit the final protocol by 12/31/2012 instead of March 31, 2013.
The sponsor agreed to conduct the deferred pediatric study, and revise the study protocol. This agreement and protocol revisions were submitted in BLA 125400 submission#35, dated 2/28/2012. The study will be completed by September 30, 2016 and the final study report will be submitted by March 31, 2017.