USA – UPDATE: NIH/TRND NPC Cyclodextrin Trial

Hello Friends of the NNPDF, We have received the following update from Dr. Denny Porter and Nicole Yanjanin of the NIH/TRND NPC Research Team with regards to the status on the NPC Cyclodextrin Clinical Trials. Dear NPD Community, The NIH cyclodextrin trial has started and the first patient will receive the drug on Monday 2/4. We are very excited to get started and to get data that will direct future studies of cyclodextrin in NPC. We have been in contact with many families and would like to provide an update about the trial screening process. The patients in this trial are grouped into “cohorts” of three patients each. Because we did not know exactly when we would be able to start the trial, the first three patients were selected because they live relatively close to the NIH or were able to come to the NIH with very little notice (less than 2 weeks in one case). Only the first 3 patients have been scheduled. We have not yet filled the remaining slots for the trial. The FDA asked us to start at a lower dose than we initially proposed. The initial data from these first three patients will determine what exactly we will do next. We decided that we will need to re-screen patients when we are ready to schedule the next cohort (patients 4, 5 and 6), in order to make sure they are still eligible. This will also need to happen again with the last 3 patients. We have a screening log of all individuals who we have screened and will be in touch with families when we are prepared to schedule the remaining slots in the trial. We would like to thank you for your continued patience and we apologize for any delays in returning phone calls or emails. We are doing our best to get this trial going as quickly and safely as possible so we can answer questions about cyclodextrin in NPC. Please do not hesitate to contact us with additional questions. The NIH NPC Research Team The NNPDF will continue to update our WebSite and Facebook Awareness pages with these periodic releases for future reference. Please follow the following web pages for more detailed and background information: ~ NNPDF Cyclodextrin Web page: ~ NNPDF Newsline Updates Web page: ~ NNPDF Facebook Awareness page: Nadine M. Hill Executive Director National Niemann-Pick Disease Foundation, Inc. 401 Madison Avenue, Suite B; PO Box 49 Fort Atkinson, WI 53538-0049 Phone: 920-563-0930 Fax: 920-563-0931 E-mail: Web site:

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