A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)

July 13, 2023 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3b Open-label Study to Assess the Effect of Elexacaftor (ELX)/Tezacaftor (TEZ)/Ivacaftor (IVA) on Glucose Tolerance in Cystic Fibrosis Subjects With Abnormal Glucose Metabolism

This study was evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in CF participants, 12 years of age and older who are heterozygous for the F508del mutation and a minimal function mutation (F/MF genotypes), with abnormal glucose metabolism.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

69

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Adelaide, Australia
        • Royal Adelaide Hospital
      • Chermside, Australia
        • The Prince Charles Hospital
      • Melbourne, Australia
        • Alfred Hospital
      • Nedlands, Australia
        • Telethon Kids Institute
      • Parkville, VIC, Australia
        • The Royal Children's Hospital
      • Randwick, Australia
        • Sydney Children's Hospital
      • South Brisbane, Australia
        • Queensland Children's Hospital
      • South Brisbane, Australia
        • Mater Adult Hospital
      • Westmead, Australia
        • Westmead Hospital
  • Belgium
      • Brussels, Belgium
        • Cliniques Universitaires de Bruxelles Hopital Erasme
      • Gent, Belgium
        • Universitair Ziekenhuis Gent
      • Leuven, Belgium
        • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
  • Czechia
      • Brno, Czechia
        • Klinika Nemoci Plicnich a Tuberkulozy
      • Praha 5, Czechia
        • Fakultni nemocnice v Motole
  • France
      • Créteil, France
        • Centre Hospitalier Intercommunal Creteil
      • Lille, France
        • CHRU de Lille - Hopital Albert Calmette
      • Marseille, France
        • CHU Marseille - Hopital Nord
      • Montpellier Cedex 5, France
        • Hopital Arnaud de Villeneuve
      • Nantes, France
        • Centre Hospitalier Universitaire De Nantes - G. R. Laennec
      • Nice, France
        • Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
      • Pierre-Bénite, France
        • Centre Hospitalier Lyon Sud
      • Rouen Cedex, Seine Maritime, France
        • CHU de Rouen - Hôpital Charles Nicolle
      • Strasbourg, France
        • Hôpitaux Universitaires de Strasbourg
      • Suresnes, France
        • Hopital Foch (Suresnes), Hopital Foch, Adultes
  • Italy
      • Ancona, Italy
        • Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
      • Genova, Italy
        • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
      • Messina, Italy
        • Azienda Ospedaliera Universitaria Policlinico G. Martino
      • Milano, Italy
        • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
      • Naples, Italy
        • Azienda Ospedaliero Universitaria Federico II Napoli
      • Verona, Italy
        • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
  • Netherlands
      • Amsterdam, Netherlands
        • Academisch Medisch Centrum (Academic Medical Centre)
      • Den Haag, Netherlands
        • HagaZiekenhuis van Den Haag
      • Heidelberglaan, Netherlands
        • University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
      • Rotterdam, Netherlands
        • Erasmus Medical Center
  • Spain
      • Barcelona, Spain
        • Hospital Universitari Vall d Hebron
      • Barcelona, Spain
        • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
      • Barcelona, Spain
        • Hospital Saint Joan de Deu
      • Madrid, Spain
        • Hospital Universitario 12 de Octubre
      • Madrid, Spain
        • Hospital Universitario Ramón y Cajal
      • Murcia, Spain
        • Hospital Virgen de la Arrixaca
      • Sevilla, Spain
        • Hospital Universitario Virgen del Rocio

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Heterozygous for F508del and an MF mutation (F/MF genotypes)
  • Forced expiratory volume in 1 second (FEV1) value ≥ 30% of predicted mean for age, sex, and height

  • Abnormal glucose tolerance determined by an OGTT as either:

    • Impaired glucose tolerance (IGT) defined as 2 hour post OGTT blood glucose level ≥140 to <200 mg/dL (≥7.77 to <11.10 mmol/L) and fasting blood glucose level <126 mg/dL (<7.00 mmol/L)
    • CF-related diabetes (CFRD) defined as either fasting hyperglycemia (blood glucose level ≥126 mg/dL [≥7.00 mmol/L] after an 8-hour fast) or 2-hour post OGTT blood glucose level ≥200 mg/dL (≥11.10 mmol/L)

Key Exclusion Criteria:

  • Clinically significant liver cirrhosis with or without portal hypertension
  • Solid organ or hematological transplantation
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Type 1 or Type 2 diabetes
  • Duration of CFRD ≥5 years

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ELX/TEZ/IVA
Participants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening.
Drug: IVA
Tablets for oral administration.
Other Names:
  • VX-770
  • ivacaftor
Drug: ELX/TEZ/IVA
Fixed dose combination (FDC) tablets for oral administration.
Other Names:
  • VX-445/VX-661/VX-770
  • elexacaftor/tezacaftor/ivacaftor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48
Time Frame: Baseline, Week 36 and 48
Baseline 2-hour post-OGTT blood glucose level was defined as the average of valid pre-dose measurements at screening and Day 1. OGTT results were considered valid only when the participant was fasting for at least 8 hours.
Baseline, Week 36 and 48

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48
Time Frame: Baseline, Week 48
Baseline dysglycemia category was defined as the most recent non-missing measurement before the first dose of study drug in the treatment period. Improvement in dysglycemia is a change from cystic fibrosis-related diabetes (CFRD) at baseline to impaired glucose tolerance (IGT)/normal glucose tolerance (NGT) at Week 48 OR change from IGT at baseline to NGT at Week 48. CFRD: 2-hour post-OGTT blood glucose level ≥200 mg/dL or fasting blood glucose level ≥126 mg/dL; IGT: 2-hour post-OGTT blood glucose level ≥140 to <200 mg/dL and fasting blood glucose level <126 mg/dL; NGT: 2 hour post-OGTT blood glucose level <140 mg/dL and fasting blood glucose level <126 mg/dL.
Baseline, Week 48
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to Week 52
Day 1 up to Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 15, 2021

Primary Completion (Actual)

July 14, 2022

Study Completion (Actual)

July 14, 2022

Study Registration Dates

First Submitted

October 21, 2020

First Submitted That Met QC Criteria

October 21, 2020

First Posted (Actual)

October 22, 2020

Study Record Updates

Last Update Posted (Actual)

August 3, 2023

Last Update Submitted That Met QC Criteria

July 13, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX19-445-117
  • 2020-003170-44 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Cystic Fibrosis

Clinical Trials on IVA

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Bangladesh

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe