An observational study to evaluate the long-term effects of evinacumab treatment in patients with homozygous familial hypercholesterolemia (HoFH)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/50238
• EU PAS number:
  EUPAS47088
• Study ID:
  50238
• Official title and acronym:
  An observational study to evaluate the long-term effects of evinacumab treatment in patients with homozygous familial hypercholesterolemia (HoFH)
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Brazil

  Canada

  France

  Germany

  Italy

  Japan

  Mexico

  Netherlands

  Portugal

  Spain
• Study description:
  This study is a 5-year observational retrospective cohort study using data collected by the existing European Atherosclerosis Society (EAS) Familial Hypercholesterolemia Studies Collaboration (FHSC) Global Familial Hypercholesterolemia (FH) Registry.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Ultragenyx Germany

Study timelines

Date when funding contract was signed

Planned: 01/09/2022

Actual: 17/07/2023

Study start date

Planned: 01/07/2023

Actual: 01/07/2023

Data analysis start date

Planned: 01/01/2025

Date of interim report, if expected

Planned: 01/06/2025

Date of final study report

Planned: 30/06/2029

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Ultragenyx

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/005449/0005