International Niemann–Pick Disease Alliance
Update #15 on the AIDNPC clinical programme
(arimoclomol in treatment of Niemann-Pick disease type C)
Conference call with patient organizations (30 MAR ’17)
The AIDNPC clinical trial programme consists of two studies:
Recruitment completed for the 002 Interventional Study:
At the latest end-of-the-month AIDNPC telephone conference hosted by the sponsor, Orphazyme ApS could announce that it had met its recruitment target of 46 patients. The following sites are participating in the ‘-002’ Study:
Great Ormond St, UK
New TC meeting frequency (now quarterly):
With the AIDNPC study now fully recruited, Orphazyme will change the frequency for the now-monthly teleconference calls providing updates. With the last patients enrolled, the TC will now be on a quarterly basis. Beside updates from the TC’s. the company will provide brief updates and other relevant information to the patient organisations on a regular basis.
Clinical Design Overview:
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 June 29th 2017 at 15h EDT.
Visit the AIDNPC Clinical Programme website: www.AIDNPC.com