A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)
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
2 other identifiers
interventional
69
7 countries
41
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jan 2021
41 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 21, 2020
CompletedFirst Posted
Study publicly available on registry
October 22, 2020
CompletedStudy Start
First participant enrolled
January 15, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 14, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
July 14, 2022
CompletedResults Posted
Study results publicly available
August 3, 2023
CompletedAugust 3, 2023
July 1, 2023
1.5 years
October 21, 2020
July 13, 2023
July 13, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 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 Outcomes (2)
Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48
Baseline, Week 48
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 52
Study Arms (1)
ELX/TEZ/IVA
EXPERIMENTALParticipants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening.
Interventions
Fixed dose combination (FDC) tablets for oral administration.
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (41)
Royal Adelaide Hospital
Adelaide, Australia
The Prince Charles Hospital
Chermside, Australia
Alfred Hospital
Melbourne, Australia
Telethon Kids Institute
Nedlands, Australia
The Royal Children's Hospital
Parkville, VIC, Australia
Sydney Children's Hospital
Randwick, Australia
Mater Adult Hospital
South Brisbane, Australia
Queensland Children's Hospital
South Brisbane, Australia
Westmead Hospital
Westmead, Australia
Cliniques Universitaires de Bruxelles Hopital Erasme
Brussels, Belgium
Universitair Ziekenhuis Gent
Ghent, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
Klinika Nemoci Plicnich a Tuberkulozy
Brno, Czechia
Fakultni nemocnice v Motole
Prague, Czechia
Centre Hospitalier Intercommunal Creteil
Créteil, France
CHRU de Lille - Hopital Albert Calmette
Lille, France
CHU Marseille - Hopital Nord
Marseille, France
Hopital Arnaud de Villeneuve
Montpellier, France
Centre Hospitalier Universitaire De Nantes - G. R. Laennec
Nantes, France
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
Nice, France
Centre Hospitalier Lyon Sud
Pierre-Bénite, France
CHU de Rouen - Hopital Charles Nicolle
Rouen Cedex, Seine Maritime, France
Hopitaux Universitaires de Strasbourg
Strasbourg, France
Hopital Foch (Suresnes), Hopital Foch, Adultes
Suresnes, France
Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
Ancona, Italy
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
Genova, Italy
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
Azienda Ospedaliero Universitaria Federico II Napoli
Naples, Italy
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
Verona, Italy
Academisch Medisch Centrum (Academic Medical Centre)
Amsterdam, Netherlands
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
Heidelberglaan, Netherlands
Erasmus Medical Center
Rotterdam, Netherlands
HagaZiekenhuis van den Haag
The Hague, Netherlands
Hospital Saint Joan de Deu
Barcelona, Spain
Hospital Universitari Vall d Hebron
Barcelona, Spain
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona, Spain
Hospital Universitario 12 de Octubre
Madrid, Spain
Hospital Universitario Ramon y Cajal
Madrid, Spain
Hospital Virgen de la Arrixaca
Murcia, Spain
Hospital Universitario Virgen del Rocio
Seville, Spain
Related Publications (1)
Durieu I, Clements B, Fabrizzi B, Mall MA, McKone E, Ramsey B, Tullis E, Taylor-Cousar JL, van der Meer R, Bachman E, Chin A, Conner S, Jennings M, Weinstock T, Colombo C, Robinson P. Impact of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance and Abnormal Glucose Metabolism: A Phase 3b, Open-Label Clinical Trial. Am J Respir Crit Care Med. 2025 Oct;211(10):1926-1934. doi: 10.1164/rccm.202411-2312OC.
PMID: 40788823DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 21, 2020
First Posted
October 22, 2020
Study Start
January 15, 2021
Primary Completion
July 14, 2022
Study Completion
July 14, 2022
Last Updated
August 3, 2023
Results First Posted
August 3, 2023
Record last verified: 2023-07
Data Sharing
- IPD Sharing
- Will not share
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