Status: Ongoing First registered on: 15/09/2021
Last updated on: 27/01/2022
1. Study identification
EU PAS Register NumberEUPAS43022
Official titleReal-World Effects and Utilisation Patterns of Elexacaftor, Tezacaftor, and Ivacaftor Combination Therapy (ELX/TEZ/IVA) in Patients with Cystic Fibrosis (CF)
Study title acronym
Study typeObservational study
Brief description of the studyCystic fibrosis (CF) is an autosomal recessive disease with serious, chronically debilitating morbidities, and high premature mortality. ELX/TEZ/IVA is currently indicated for treatment of CF in patients 12 years and older in the EU who have specified CFTR mutations. This 5-year observational post-authorisation safety study (PASS) will evaluate safety, effectiveness / CF disease progression, and pregnancy outcomes in patients with CF who are treated with ELX/TEZ/IVA, as well as its drug utilisation patterns using observational cohorts of patients receiving therapy in a real-world setting. Existing CF registries provide an established source to obtain data on long term effects in real world use for analysis. In the US Cystic Fibrosis Foundation Patient Registry (CFFPR) and German CF Registry, within-cohort evaluation of outcomes in the 5-year periods before and after treatment initiation will be performed. Evaluation of the outcome patterns and trends in the 5-year pre-treatment period will place into context the outcome patterns and trends observed in the post-treatment period. In addition, the European Cystic Fibrosis Society Patient Registry (ECFSPR) will be used to provide additional information for the evaluation of drug utilisation patterns in the European region. Information regarding the safety profile of the therapy under the real-world conditions of use will be informative to patients, caregivers, and prescribers. Existing CF registries provide an established source from which to obtain these data.
Was this study requested by a regulator?Yes: EMA
Is the study required by a Risk Management Plan (RMP)? EU RMP category 3 (required)
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 nameVertex Pharmaceuticals Incorporated
Centre locationBoston, MA, USA
Details of (Primary) lead investigator
Title Dr
Last name Bower
First name Julie
Is this study being carried out with the collaboration of a research network?
No
Other centres where this study is being conducted
Multiple centres

In total how many centres are involved in this Study?3
German CF Register, Germany
ECFSPR, European region (multiple countries)
US CFF Patient Registry, United States
Countries in which this study is being conducted
International study

Germany
United States
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed31/08/202131/08/2021
Start date of data collection31/08/202131/08/2021
Start date of data analysis
Date of interim report, if expected31/12/2021
Date of final study report31/12/2025
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesVertex Pharmaceuticals Incorporated100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Dr
Last name Global Medical Information
First name Vertex Pharmaceuticals
Address line 12 Kingdom Street
Address line 2 
Address line 3 
CityLondon 
PostcodeW2 6BD 
CountryUnited Kingdom
Phone number (incl. country code)0035317617299 
Alternative phone number 
Fax number (incl. country code) 
Public Enquiries
Title Dr 
Last name Global Medical Information 
First name Vertex Pharmaceuticals 
Address line 12 Kingdom Street 
Address line 2 
Address line 3 
CityLondon 
PostcodeW2 6BD 
CountryUnited Kingdom 
Phone number (incl. country code)0035317617299 
Alternative phone number 
Fax number (incl. country code) 
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