Status: Ongoing First registered on: 16/01/2017
Last updated on: 03/07/2019
1. Study identification
EU PAS Register NumberEUPAS17267
Official titleEuropean Post-Authorization Registry for RAVICTI® (glycerol phenylbutyrate) Oral Liquid in Partnership with the European Registry and Network for Intoxication Type Metabolic Diseases (E-IMD)
Study title acronym
Study typeObservational study
Brief description of the studyThis is a multi-center, prospective, non-interventional registry conducted by E-IMD in collaboration with Horizon 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.
Was this study requested by a regulator?Yes: EMA
Is the study required by a Risk Management Plan (RMP)? EU RMP category 1 (imposed as condition of marketing authorisation)
Regulatory procedure number (RMP Category 1 and 2 studies only)
Other study registration identification numbers and URLs as applicable
2. Research centres and Investigator details
Coordinating study entity
Centre nameImmedica Pharma AB
Centre locationStockholm, Sweden
Details of (Primary) lead investigator
Title Ms
Last name Jansson
First name Åsa
Is this study being carried out with the collaboration of a research network?
Yes
E-IMD
Other centres where this study is being conducted
Multiple centres

In total how many centres are involved in this Study?20
Countries in which this study is being conducted
International study

Austria
Belgium
Croatia
Denmark
France
Germany
Italy
Poland
Portugal
Spain
Switzerland
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed10/10/2016
Start date of data collection07/11/2017
Start date of data analysis31/01/2030
Date of interim report, if expected
Date of final study report31/07/2030
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesImmedica Pharma AB100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Ms
Last name Jansson
First name Åsa
Address line 1Norrtullsgatan 15
Address line 2 
Address line 3 
CityStockholm 
Postcode11329 
CountrySweden
Phone number (incl. country code)46-766337071 
Alternative phone number 
Fax number (incl. country code) 
Email address asa.jansson@immedica.com
Public Enquiries
Title Ms 
Last name Jansson 
First name Åsa 
Address line 1Norrtullsgatan 15 
Address line 2 
Address line 3 
CityStockholm 
Postcode11329 
CountrySweden 
Phone number (incl. country code)46-766337071 
Alternative phone number 
Fax number (incl. country code) 
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