International Niemann–Pick Disease Alliance

Orphazyme: Update #12 on the AIDNPC Clinical Programme


Update #12 on the AIDNPC clinical programme

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

 (Available for download in English, French, German, Italian, Polish, Spanish)

Conference call with patient organizations (22 DECEMBER ’16)


  • 002 Study: Recruitment into the 002 Interventional Study
  • 002 Study: US sites — contact details
  • 002 Study: Clinical design overview





The AIDNPC clinical trial programme consists of two studies:

  • The ‘-001’ Observational Study, where patients can join the programme early and participate in a natural history study. This study is running in several European countries. US patients are not able to join the 001 study.
  • The ‘-002’ Interventional Study, is currently opening up in countries where approval has been obtained. Here, patients will receive three-times daily oral treatment with the study drug in a placebo-controlled manner. Not all sites are yet able to enroll.

Recruitment into the 002 Interventional Study

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.


US Sites — Contact Details

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:

  • Mayo Clinic Children’s Center (Rochester, Minnesota)
    • Principal investigator: Marc C. Patterson, MD
    • Contact: Jaime Sorum
    • +1 507 583 5418,
  • Children’s Hospital Pittsburgh (Pittsburgh, Pennsylvania)
    • Principal investigator: Maria Escolar, MD
    • Contact: Michele D. Poe
    • +1 412 692 9952,
  • UCSF Benioff Children’s Hospital Oakland (Oakland, California)
    • Principal investigator: Paul Harmatz, MD
    • Contact: Jacqueline Madden
    • +1 510 428 3885 ext5745,


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


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 January 26th 2017 at 15h EDT.

Visit the AIDNPC Clinical Programme website:

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