International Niemann–Pick Disease Alliance
Update #12 on the AIDNPC clinical programme
(arimoclomol in treatment of Niemann-Pick disease type C)
Conference call with patient organizations (22 DECEMBER ’16)
The AIDNPC clinical trial programme consists of two studies:
At the latest end-of-the-month AIDNPC telephone conference hosted by the sponsor, Orphazyme ApS, the following update on the recruitment into the ‘-002’ Study was presented. Over the last month, 10 more patients have enrolled in ‘-002’ Study, for a the total of 13 patients now enrolled to date at the 5 European sites that so far have opened for ‘-002’ enrolment. Of these, 10 patients have rolled over from the ‘-001’ Study, while 3 are enrolled directly into ‘-002’. To date at the following site have been activated for the ‘-002’ Study:
– Copenhagen, Denmark
– Birmingham, UK
– Mainz, Germany
– Münich, Germany
– Paris, France
Orphazyme was pleased that approvals national competent authorities, local and national ethics committees and research councils, as well as hospitals and clinics are being obtained such that individual sites are now getting activated for the ‘-002’ Study.
Orphazyme informs that it continues to work to getting all sites (including in the US) ready and fully recruited as soon as possible.
To reiterate, Orphazyme announced that the three US sites that are being established can now be announced. The three US sites and their contact details are as follows:
The graphic below illustrates the design of the ‘-002’ Study, including number and timing of patients visits to the sites. An escape route is provided for patients that experience an unacceptable rate of progression of the disease.
We encourage the sharing of above information with the patient community.
The next AIDNPC call is schedule for Thursday January 26th 2017 at 15h EDT.
Visit the AIDNPC Clinical Programme website: www.AIDNPC.com