WAYLIVRA® Post-Authorisation Safety Study (PASS) and Product Registry (WAY4001)

Study identification

• EU PAS number:
  EUPAS36702
• Study ID:
  47815
• Official title and acronym:
  WAYLIVRA® Post-Authorisation Safety Study (PASS) and Product Registry (WAY4001)
• DARWIN EU® study:
  No
• Study countries:
  Austria

  France

  Germany

  Greece

  Italy

  Netherlands

  Spain

  Sweden

  United Kingdom
• Study description:
  The aim of this study (PASS phase and WAYLIVRA product registry phase) is to further characterise the safety and effectiveness of WAYLIVRA in patients with Familial Chylomicronaemia Syndrome (FCS) under real-world conditions. This study will be conducted in two phases. The first phase of the study is the PASS phase and will be concluded after a study term of 7 years (or earlier) if data collected amounts to 247 person-years of exposure. The exposure population includes patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. Following the PASS phase, the study will continue as the WAYLIVRA Product Registry, or the Registry phase, which will be conducted throughout the commercial life of the drug, to obtain long-term data on safety and efficacy of WAYLIVRA. In both phases of the study, real world data will be collected on FCS patients who are prescribed WAYLIVRA. This study is designed as a non-interventional observational study. All patients will receive care according to normal clinical practice and clinical care will not be mandated by the protocol. As such, the decision to prescribe WAYLIVRA is separate from the decision to include the patient in the study and patients are not required to undergo any additional diagnostic or monitoring procedures.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Akcea Therapeutics, Inc.

Study timelines

Date when funding contract was signed

Planned:27/08/2020

Actual:27/11/2020

Study start date

Planned:04/12/2020

Actual:04/12/2020

Date of final study report

Planned:31/12/2028

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Akcea Therapeutics, Inc.

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)
• Regulatory procedure number:
  EMEA/H/C/4538

Other study registration identification numbers and links

WAY4001