International Niemann–Pick Disease Alliance
Update #13 on the AIDNPC clinical programme
(arimoclomol in treatment of Niemann-Pick disease type C)
Conference call with patient organizations (26 JANUARY ’17)
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, 9 more patients have enrolled in ‘-002’ Study, for a the total of 22 patients now enrolled to date at the 8 European sites that so far have opened for ‘-002’ enrolment. Of these, 18 patients have rolled over from the ‘-001’ Study, while 4 are enrolled directly into ‘-002’. To date at the following site have been activated for the ‘-002’ Study:
– Birmingham, UK
– Great Ormond St, UK
– Mainz, Germany
– Münich, Germany
– Paris, France
– Barcelona, Spain
– Warsaw, Poland
The total number of patients planned to be enrolled is 46. Orphazyme was pleased with pick-up in the rate of enrolment that has been experienced over the last 2 months, and informed that the ‘-001’ Observational Study is no longer recruiting patients.
Orphazyme informs that it continues to work to getting all sites (including in the US) ready and fully recruited as soon as possible, and the company expects all patients to have been recruited by early Spring.
Orphazyme was also pleased to announce that two US sites are now all very close to opening up for recruitment. The US sites to participate and their contact details are hereby updated:
Dr. Thomas Kirkegaard Jensen, Orphazyme’s CSO, will be talking at the 13th Annual WORLD Symposium in San Diego US (13/2–17/2 2017), presenting the company’s approach to Heat Shock Protein-based therapies.
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 February 23th 2017 at 15h EDT.
Visit the AIDNPC Clinical Programme website: www.AIDNPC.com