Cystic FibrosisPhase 3NCT05153317
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older
A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 22 sites in 5 countries.
Trial summary
The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older. Read more about this study
- Who is it for?
- Ages 2 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
- 71 participants
- Where
- 22 sitesin 5 countries
- Study dates
- 17 Jan 2022to 29 Jan 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completed study drug treatment in the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study
Who cannot join
- History of study drug intolerance in the parent study
- History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study
- Other protocol defined Inclusion/Exclusion criteria may apply
Trial locations and contacts
22 sites in 5 countries.
22 sites in 5 countries
United States13 sites
Children's Hospital of Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202
The Children's Mercy Hospital
Kansas City, Missouri, 64108
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
Stanford University
Palo Alto, California, 94304
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health & Science University
Portland, Oregon, 97239
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, 63110
Banner University of Arizona Medical Center
Tucson, Arizona, 85724
Australia3 sites
Telethon Kids Institute
Nedlands
The Royal Children's Hospital
Parkville
Queensland Children's Hospital
South Brisbane
Canada2 sites
The Hospital for Sick Children
Toronto
British Columbia Children's Hospital
Vancouver
Germany2 sites
Charite Paediatric Pulmonology Department
Berlin
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
Essen
United Kingdom2 sites
Alder Hey Children's NHS Foundation Trust
Liverpool
Royal Brompton Hospital
London
About this study
Official title: A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older
The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.
- 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 granules or tablets for oral administration.
IVADrug
Also called VX-770, ivacaftor
Granules or tablets for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| ELX/TEZ/IVA | Experimental | Participants will receive ELX/TEZ/IVA in the morning and IVA 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.
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From Day 1 up to Week 196
Secondary outcomes (2)
Absolute Change in Sweat Chloride (SwCl)
Measured: From Baseline up to Week 192
Absolute Change in Lung Clearance Index (LCI) 2.5
Measured: From Baseline up to Week 192
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2021
- Study start17 Jan 2022
- Primary completion29 Jan 2026
- Study completion29 Jan 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 71 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
- NCT05153317
- Sponsor's protocol ID
- VX20-445-112
- Other IDs
- 2020-002239-31
- Status last verified
- Feb 2026
- First submitted
- 29 Nov 2021
- Last updated
- 3 Mar 2026
- Results posted
- No
- 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/our-science/clinical-trials-data-sharing/
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older
- ClinicalTrials.gov ID
- NCT05153317
- Acronym / sponsor ID
- VX20-445-112
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 71 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 17 Jan 2022 (actual)
- Primary completion
- 29 Jan 2026 (actual)
- Study completion
- 29 Jan 2026 (actual)
- First posted
- 10 Dec 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.
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 will receive ELX/TEZ/IVA in the morning and IVA in the evening. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Completed study drug treatment in the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study
Key exclusion criteria (3 listed)
- History of study drug intolerance in the parent study
- History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study
- Other protocol defined Inclusion/Exclusion criteria may apply
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 granules or tablets for oral administration. | Drug | ELX/TEZ/IVA |
| IVAAlso: VX-770, ivacaftorGranules or tablets for oral administration. | Drug | ELX/TEZ/IVA |
Primary outcome
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 up to Week 196
2 secondary outcomes
Absolute Change in Sweat Chloride (SwCl)
Time frame: From Baseline up to Week 192
Absolute Change in Lung Clearance Index (LCI) 2.5
Time frame: From Baseline up to Week 192
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2026.
- Individual participant dataSharing planned: No. Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-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.
22 sites in 5 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 22 sites in 5 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 1 inclusion and 3 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 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, Australia, Canada, Germany, United Kingdom. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: From Day 1 up to Week 196). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 Mar 2026.