European Post-Authorization Registry for RAVICTI® (glycerol phenylbutyrate) Oral Liquid in Partnership with the European Registry and Network for Intoxication Type Metabolic Diseases (E-IMD) (HZNP-RAV-401)

Study identification

• EU PAS number:
  EUPAS17267
• Study ID:
  30377
• Official title and acronym:
  European Post-Authorization Registry for RAVICTI® (glycerol phenylbutyrate) Oral Liquid in Partnership with the European Registry and Network for Intoxication Type Metabolic Diseases (E-IMD) (HZNP-RAV-401)
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Denmark

  France

  Germany

  Poland
• Study description:
  This is a multi-center, prospective, non-interventional registry conducted by E-IMD in collaboration with Immedica Pharma designed to collect data on safety and outcomes in patients with urea cycle disorders (UCDs) on treatment with RAVICTI or patients with UCDs treated with alternative nitrogen scavenging medication other than RAVICTI.
• Study status:
  Finalised

Research institutions and networks

Institutions

Multiple centres: 5 centres are involved in the study

Networks

E-IMD

Study timelines

Date when funding contract was signed

Actual:10/10/2016

Study start date

Actual:07/11/2017

Data analysis start date

Planned:31/01/2030

Actual:27/06/2022

Date of final study report

Planned:31/07/2030

Actual:31/10/2022

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Immedica Pharma AB

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)