Actually is the update. 9/18 - 9/20
please find some interesting notes....i copy them from the pdf.
there are some graphs also but i don't know how to apload them here
2015 –Orphan Drug Designations transferred to CTD via its partner MNE from Sue French (for UK & Europe) and Caroline Hastings (US)
•Type II Drug Master File accepted by U.S. FDA
•August 2015: CTD announces plans for Intravenous Trappsol® Cyclo™ clinical trial and upcoming meeting with European health authority to discuss initiation of clinical program. 5
Goal: Gaining market approval for use of Trappsol® Cyclo® in the treatment of NPC
•Initial steps: 2013 –2015
•Established an expert team to develop/conduct the clinical program
•Dr. Sharon Hrynkow, first as consultant, now as Senior VP for Medical Affairs
•Accenture Accelerate R & D Services for Life Sciences
•Boyd Consultants --Alan Boyd
•AptusClinical
•KJC Statistics
•Prism Ideas
Why an Intravenous trial?
•NPC is a systemic disease
•Cyclodextrinsprovided to mice in a systemic manner lead to reduced total cholesterol, decrease in liver size, increase in longevity
•Following Slides courtesy of B. Liu, published data in A. Lopez et al, Clin. Exp. Pharm. Phys. 2014
Why an Intravenous trial?
•Anecdotal clinical data available for IV administration for up to five years
•Published reports on IV administration leading to stabilization of symptomology or improvements, including in CNS (“wakefulness”)
•IV less invasive than some other routes of administration
•IV easier for families and physicians to manage
•IV does not require sedation 13
When will the trial start?
Expected enrollment first half of 2016.
CTD’s cyclodextrin(Trappsol® Cyclo™) in use in 20 patients right now, and it has been in use in some for more than 5 years
•Trappsol® Cyclo™ has a consistent manufacturing process, a DMF as an Active Pharmaceutical Ingredient and has proven stability
•Anecdotal data suggest that an IV trial will be important for
NPC Patients and their Families
We are working to launch our IV trial in the first part of 2016
•Compassionate use programs will continue in parallel with clinical programs
•We look forward to working with the whole NPC community to improve the lives of NPC patients globally.
enjoy
