Dear Families and Friends,
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
