International Niemann–Pick Disease Alliance

Update #15 on the AIDNPC Clinical Programme


Update #15 on the AIDNPC clinical programme

(arimoclomol in treatment of Niemann-Pick disease type C)

Conference call with patient organizations (30 MAR ’17)

Available for download in English, French, German, Italian, Polish, and Spanish



  • 002 Study: Recruitment completed for the 002 Interventional Study
  • New TC meeting frequency (now quarterly)
  • 002 Study: Clinical design overview

The AIDNPC clinical trial programme consists of two studies:

  • The ‘-001’ Observational Study, which is no longer recruiting.
  • The ‘-002’ Interventional Study, which is no longer recruiting.


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:


Copenhagen, Denmark

Birmingham, UK

Great Ormond St, UK

Mainz, Germany

Münich, Germany

Paris, France

Montpellier, France

Barcelona, Spain

Warsaw, Poland

Udine, Italy

Rome, Italy

Rochester, MN-USA

Oakland, CA-USA


To obtain detailed contact information for individual clinical sites in the AIDNPC programme, visit and use identifier NCT02612129.


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.


Next call:

The next AIDNPC call is schedule for Thursday June 29th 2017 at 15h EDT.

Visit the AIDNPC Clinical Programme website:

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