Dear NNPDF Families and Friends,
The National Niemann-Pick Disease Foundation has been advised by Orphazyme that an “Observational” clinical study for NP-C, tied to their clinical development program of arimoclomol in NPC, has been registered and made available on www.clinicaltrials.gov. The link to the information pertaining to just this trial is at: https://clinicaltrials.gov/ct2/show/NCT02435030?term=02435030&rank=1.
This information was updated as of April 30, 2015.
At this time, no site in the United States has been announced and currently the study is not open for participant recruitment. Orphazyme has indicated that they are targeting a clinical trial to start date in the United States toward the end of first quarter 2016. As we receive further announcements and updates, it will be posted to our NPD family community!
You can read either the full text view or the tabular view depending upon your preference. The text allows you to see the inclusion and exclusion information along with the primary outcome measures they will be looking at.
Nadine M. Hill
National Niemann-Pick Disease Foundation