Disease registry for patients with Niemann-Pick Type C disease (NPC Registry)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/28331
• EU PAS number:
  EUPAS4622
• Study ID:
  28331
• Official title and acronym:
  Disease registry for patients with Niemann-Pick Type C disease (NPC Registry)
• DARWIN EU® study:
  No
• Study countries:
  Australia

  Austria

  Brazil

  Bulgaria

  Canada

  China

  Czechia

  France

  Germany

  Greece

  Italy

  Netherlands

  Norway

  Poland

  Portugal

  Russian Federation

  Slovakia

  Slovenia

  Spain

  Sweden

  Switzerland

  United Kingdom
• Study description:
  International, prospective, observational, long-term, disease-specific registry for patients with Niemann-Pick Type C disease.Inclusion criteria: - Patients with NP-C disease- Patients and/or legal guardian must be willing to give written informed consent- Where necessary in order to comply with national regulatory requirements, children will be asked for written informed consent.Exclusion criteria: - NP-C patients who died before the Registry was launched
• Study status:
  Finalised

Research institutions and networks

Institutions

Actelion Pharmaceuticals

Multiple centres: 112 centres are involved in the study

Study timelines

Date when funding contract was signed

Actual: 10/02/2009

Study start date

Actual: 09/09/2009

Date of final study report

Actual: 06/12/2017

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Actelion

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)