International Niemann–Pick Disease Alliance

AIDNPC Clinical Programme change: Earlier initiation of Interventional study

The INPDA and its member groups have received the following update from Orphazyme, ApS:

AIDNPC Clinical Programme change: Earlier initiation of Interventional study (CT-ORZY-NPC-002; Arimoclomol treatment).

After valuable dialogue with regulators and patient organizations on the design of the Observational and Interventional studies under the AIDNPC programme, the protocol for the latter has now been finalised and we have made this synopsis for your information and further dissemination.

  • The observational study is being initiated as planned.
  • Meanwhile, the interventional study will now be able to start enrolment as soon as study approvals are in place
  • •As a consequence, patients may now spend less than six months in the observational study before entering the interventional study

The goal of the AIDNPC programme is to provide robust evidence of a beneficial effect of arimoclomol in the shortest possible time. The AIDNPC programme has two parts: an “observational” study followed by an “interventional” study that investigates the efficacy of arimoclomol compared to placebo.

Every day counts for the NP-C patients, and after a constructive dialogue with the regulatory authorities and patient organizations, treatment with arimoclomol will be initiated at the earliest possible time. Therefore, patients enrolled in the “observational” study will enter directly into the “interventional” study as soon as the competent authorities and ethical committees approve the interventional study protocol, irrespective of how long the patients have been in the “observational” study. This means that the duration of the “observational” trial is now going to be up to 6 months, but may likely be shorter.

Participation in the “observational” study requires only one clinical visit ahead of the initiation of the interventional trial, and the visit ensures recording of extensive and uniform information about all the individual patients’ disease status ahead of the initiation of the “interventional” trial. Together with the efficacy-of-treatment measures and biomarkers, the AIDNPC programme will provide new insight into to the biological changes underlying the relentless progress of the disease.

When the “interventional” study starts, patients who have not had an opportunity to enrol in the “observational” study will have the opportunity to enter into the “interventional” study according to the specific inclusion and exclusion criteria as well as the availability of open slots for patients. A key criterion is that these patients, when in treatment with miglustat, must have been on a stable dose for at least 6 months — otherwise, the criteria are consistent with the “observational” study.

We are grateful for the continued support of all the NP-C patient organizations and we look forward to initiating the “investigational” part of the AIDNPC programme even faster.

Yours sincerely

Christian Quaade 

Strategic Alliances Director

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