Cystic FibrosisPhase 3NCT04599465
A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)
A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 41 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- ELX/TEZ/IVA and IVA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 69 participants
- Where
- 41 sitesin 7 countries
- Study dates
- 15 Jan 2021to 14 Jul 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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.
Trial locations and contacts
41 sites in 7 countries.
41 sites in 7 countries
France10 sites
Centre Hospitalier Intercommunal Creteil
Créteil
CHRU de Lille - Hopital Albert Calmette
Lille
CHU Marseille - Hopital Nord
Marseille
Hopital Arnaud de Villeneuve
Montpellier
Centre Hospitalier Universitaire De Nantes - G. R. Laennec
Nantes
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
Nice
Centre Hospitalier Lyon Sud
Pierre-Bénite
CHU de Rouen - Hopital Charles Nicolle
Rouen Cedex, Seine Maritime
Hopitaux Universitaires de Strasbourg
Strasbourg
Hopital Foch (Suresnes), Hopital Foch, Adultes
Suresnes
Australia9 sites
Royal Adelaide Hospital
Adelaide
The Prince Charles Hospital
Chermside
Alfred Hospital
Melbourne
Telethon Kids Institute
Nedlands
The Royal Children's Hospital
Parkville, VIC
Sydney Children's Hospital
Randwick
Mater Adult Hospital
South Brisbane
Queensland Children's Hospital
South Brisbane
Westmead Hospital
Westmead
Spain7 sites
Hospital Saint Joan de Deu
Barcelona
Hospital Universitari Vall d Hebron
Barcelona
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona
Hospital Universitario 12 de Octubre
Madrid
Hospital Universitario Ramon y Cajal
Madrid
Hospital Virgen de la Arrixaca
Murcia
Hospital Universitario Virgen del Rocio
Seville
Italy6 sites
Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
Ancona
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
Genova
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan
Azienda Ospedaliero Universitaria Federico II Napoli
Naples
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
Verona
Netherlands4 sites
Academisch Medisch Centrum (Academic Medical Centre)
Amsterdam
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
Heidelberglaan
Erasmus Medical Center
Rotterdam
HagaZiekenhuis van den Haag
The Hague
Belgium3 sites
Cliniques Universitaires de Bruxelles Hopital Erasme
Brussels
Universitair Ziekenhuis Gent
Ghent
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven
Czechia2 sites
Klinika Nemoci Plicnich a Tuberkulozy
Brno
Fakultni nemocnice v Motole
Prague
About this study
Official title: 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.
- Conditions
- Cystic Fibrosis
- Being tested
- ELX/TEZ/IVA, IVA
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ELX/TEZ/IVADrug
Also called VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Fixed dose combination (FDC) tablets for oral administration.
IVADrug
Also called VX-770, ivacaftor
Tablets for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| ELX/TEZ/IVA | Experimental | Participants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening. Receives: Drug: ELX/TEZ/IVA; Drug: IVA |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, Week 36 and 48
Secondary outcomes (2)
Percentage of Participants With Improvement in Dysglycemia Categorization at 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.
Measured: Baseline, Week 48
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: Day 1 up to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2020
- Study start15 Jan 2021
- Primary completion14 Jul 2022
- Study completion14 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04599465
- Sponsor's protocol ID
- VX19-445-117
- Other IDs
- 2020-003170-44
- Status last verified
- Jul 2023
- First submitted
- 21 Oct 2020
- Last updated
- 3 Aug 2023
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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
Publications
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.
PubMed 40788823
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04599465
- Acronym / sponsor ID
- VX19-445-117
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 69 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 15 Jan 2021 (actual)
- Primary completion
- 14 Jul 2022 (actual)
- Study completion
- 14 Jul 2022 (actual)
- First posted
- 22 Oct 2020 (actual)
- Results
- Posted 3 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ELX/TEZ/IVA | Experimental | Drug: ELX/TEZ/IVA, Drug: IVAParticipants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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:
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ELX/TEZ/IVAAlso: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftorFixed dose combination (FDC) tablets for oral administration. | Drug | ELX/TEZ/IVA |
| IVAAlso: VX-770, ivacaftorTablets for oral administration. | Drug | ELX/TEZ/IVA |
Primary outcome
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.
2 secondary outcomes
Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48
Time frame: 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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40788823Durieu 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.
- Individual participant dataSharing planned: No. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
41 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 41 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in France, Australia, Spain, Italy, Netherlands and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Aug 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04599465, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Aug 2023.