International Niemann–Pick Disease Alliance

Orphazyme: Update #9 on the AIDNPC Clinical Programme




Update #9 on the AIDNPC clinical programme

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


Conference call with patient organizations (26 AUGUST ’16)


  • 002 Study: Recruitment into the 002 Interventional Study
  • 002 Study: Patient considerations
  • Orphazyme at Meeting and Events
  • 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 ‘-001’ and ‘-002’ Studies was presented. In the ‘-001’ Study, a total of 33 patients have been enrolled to date at 12 European sites. In the ‘-002’ Study, so far a total of 3 patients have been enrolled to date at the following site:

Copenhagen, Denmark


Significant progress is being made on the regulatory front to ensure the opening of more sites in the’-002’ Study. In Spain, Italy and the UK, the competent authorities have approved the study, while local ethics committees here are still reviewing and contracts with hospitals are being negotiated. Meanwhile, in Germany, Poland and Switzerland, competent authorities are expected to approve the study shortly. Overall, the regulatory process is advancing well and within the expected timeline. In the US, negotiations with site and discussions with IRBs are progressing well.

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

  • For the ‘-001’ Observational Study, use identifier NCT02435030
  • For the ‘-002’ Interventional Study, use identifier NCT02612129

Based on the success in recruiting patients into the ‘-001’ Observational Study, Orphazyme remains confident that it comfortably will fulfil its goal of enrolling 46 NPC patients into the ‘-002’ Interventional Study.


Patient Considerations:

Asked about how dosing for the individual patient is determined, Orphazyme explained that the dosing is dependent on the patient’s weight. Throughout the study, the patient’s weight is continuously monitored, and a change in weight causing the patient to enter into different weight bracket will caused a change in the dosing of this patient.

Asked about potential side effects, Orphazyme explained that — based on significant experience in healthy volunteers and patients (with other diseases) — side effects are expected to be only minor and transient, with some patients potentially experiencing mainly headache, tiredness, sleepiness, mild gastrointestinal effects, constipation, loose stools, nausea, dry mouth, bowel movement problems, pain and vomiting.

With patients being dosed orally at their homes, monthly telephone calls from the clinical site in addition to the quarterly site visits will ensure that side effects and any complication will be routinely monitored and evaluated. This is a normal process in clinical trials, living up to international guidelines and regulations.


Meeting and Events:

Orphazyme participated in the NNPDF’s 24th annual Family Support Medical Conference in Boston. Orphazyme was very pleased to engage actively and productively with the US NPC patient and clinical community.

Orphazyme will be attending the events at the 7th Interactive Workshop on Niemann-Pick Diseases and 23rd Family Conference in Bedfordshire, UK held September 16-18, 2016.


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.

Study Design CT-ORZY-NPC-002

We encourage the sharing of above information with the patient community.


Next call:

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


Visit the AIDNPC Clinical Programme website:

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