Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation

Plain-language summary

This study evaluates the efficacy and safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA), compared with placebo, in cystic fibrosis patients who do not have an F508del mutation. It enrolled patients with a qualifying, treatment-responsive CFTR mutation whose lung function values fell within a defined range. The study was conducted in Phase 3 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 3

Status

Completed

Sponsor

Vertex Pharmaceuticals Incorporated

Intervention

ELX/TEZ/IVA, IVA, Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA)

Start date

5/9/2022

Estimated completion

7/5/2023

Countries

Austria, Belgium, Canada, Czechia, France, Germany, Hungary, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, Switzerland

Related genes

Locations

• Innsbruck, Austria
• Brussels, Belgium
• Brussels, Belgium
• Edegem, Belgium
• Ghent, Belgium
• Leuven, Belgium
• Woluwe-Saint-Lambert, Belgium
• Edmonton, Canada
• Montreal, Canada
• Montreal, Canada
• Toronto, Canada
• Vancouver, Canada
• Vancouver, Canada
• Brno, Czechia
• Prague, Czechia
• Bron, France
• Créteil, France
• Lille, France
• Marseille, France
• Montpellier, France
• Nantes, France
• Nice, France
• Paris, France
• Paris, France
• Paris, France
• Pessac, France
• Pierre-Bénite, France
• Reims, France
• Rennes, France
• Roscoff, France
• Toulouse, France
• Tours, France
• Berlin, Germany
• Cologne, Germany
• Erlangen, Germany
• Essen, Germany
• Frankfurt, Germany
• Giessen, Germany
• Halle, Germany
• Hanover, Germany

+50

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Key Inclusion Criteria:

* Participant has a qualifying ELX/TEZ/IVA-responsive CFTR mutation and does not have an exclusionary CFTR mutation
* Forced expiratory volume in 1 second (FEV1) value \>=40% and \<=100% of predicted mean for age, sex, and height

Key Exclusion Criteria:

* History of solid organ or hematological transplantation
* Clinically significant cirrhosis with or without portal hypertension
* Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply.