Cerliponase alfa Observational Study

Study identification

• EU PAS number:
  EUPAS29031
• Study ID:
  46783
• Official title and acronym:
  Cerliponase alfa Observational Study
• DARWIN EU® study:
  No
• Study countries:
  Denmark

  France

  Germany

  Italy

  Netherlands

  Romania

  Sweden

  United Kingdom
• Study description:
  This is a voluntary, multicenter, multinational, cerliponase alfa observational study for patients with a confirmed diagnosis of neuronal ceriod lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are currently being treated with cerliponase alfa.
• Study status:
  Ongoing

Research institutions and networks

Institutions

ICON Commercialisation & Outcomes

Germany

Ireland

Study timelines

Date when funding contract was signed

Planned:14/03/2019

Actual:19/03/2019

Study start date

Planned:28/10/2019

Actual:10/10/2019

Date of final study report

Planned:27/04/2030

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

BioMarin Pharmaceutical

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 2 (specific obligation of marketing authorisation)