International Niemann–Pick Disease Alliance

Orphazyme: Update #13 on the AIDNPC Clinical Programme


Update #13 on the AIDNPC clinical programme

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

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

Conference call with patient organizations (26 JANUARY ’17)



  • 002 Study: Recruitment into the 002 Interventional Study
  • 002 Study: US sites to open soon — contact details
  • Orphazyme at the WORLD conference
  • 002 Study: Clinical design overview


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, 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:

-Copenhagen, Denmark

– 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.

US sites to soon open — Contact Details

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:

  • Mayo Clinic Children’s Center (Rochester, Minnesota)
    • Principal investigator: Marc C. Patterson, MD
    • Contact: Jaime Sorum
    • +1 507 583 5418,
  • 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.

Orphazyme at the WORLD Conference (San Diego)

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.

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 February 23th 2017 at 15h EDT.

Visit the AIDNPC Clinical Programme website:

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