Wednesday, January 23, 2013

Press Release from NIH on Cyclodextrin Trial

Dear Families and Friends,

The NNPDF central office received a press release from the National Institute of Health (NIH) NPC Clinic from Dr. Forbes "Denny" Porter with an update on the Cyclodextrin Trial.

Visit the NNPDF Cyclodextrin page to view it.

Friday, January 11, 2013

UPDATE: FDA Approves NIH TRND Team for NPC Cyclodextrin Clinical Trial!

Dear Families and Friends,

The NNPDF central office received the following update from the National Institute of Health (NIH) NPC Clinic from Dr. Forbes "Denny" Porter.

"We were informed today that the FDA has removed the clinical hold on the hydroxypropyl-β-cyclodextrin trial. We are planning to enroll the first patient in two weeks. This trial is a major step in trying to determine if this is a safe and biochemically effective drug for NPC. Our goal is to use data from this trial to optimize the design of a larger second trial focused on clinical efficacy. Thank you for your help and support!
The TRND Team"

To follow updates and breaking news visit the Cyclodextrin page on the NNPDF web site

Friday, January 4, 2013

NIH's Clinical Trial of Cyclodextrin Update

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