The NIH 2-hydroxypropyl-β-cyclodextrin trial has been put on hold by the FDA. The FDA had specific issues regarding the trial, the majority of which we were able to resolve during a teleconference on December 10, 2012. However, there were questions concerning the compatibility of the drug and the Ommaya reservoir. Both Johnson and Johnson and Integra (the manufacturer of the Ommaya reservoir) provided significant assistance in helping to answer these questions.
The FDA was provided with our complete written response to their questions on December 13th and we provided an updated protocol on December 31st. We are currently waiting for the review of our response from the device division, and we believe that we have adequately addressed their concerns. The FDA has 30 days (plus Federal holidays) from December 13th to provide a formal response. In order to allow for this, we have moved back our targeted start date by two weeks to the end of January 2013. This will allow us time to accommodate a positive response from the FDA and to reconcile any changes with what the NICHD IRB has approved.
We fully understand the disappointment that this message entails; however, we are committed to resolve any further questions that the FDA may raise. Despite this delay, we are hopeful that we are very close to the start of the trial. We will plan to update the NPC1 community as more information becomes available.
The TRND Team