Cystic FibrosisPhase 3NCT04043806
A Study Evaluating the Long-term Safety of VX-445 Combination Therapy
A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 97 sites in 11 countries.
Trial summary
This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF). 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
- 458 participants
- Where
- 97 sitesin 11 countries
- Study dates
- 9 Aug 2019to 14 Dec 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
- Currently participating in study VX17-659-105 (NCT03447262)
Who cannot join
- History of drug intolerance in study VX17-659-105 that would pose an additional risk to the participant in the opinion of the investigator
- Current participation in an investigational drug trial (other than study VX17-659-105)
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
97 sites in 11 countries.
97 sites in 11 countries
United States46 sites
Albany Medical College, Pulmonary and Critical Care Medicine
Albany, New York, 12208
Michigan Medicine
Ann Arbor, Michigan, 48109-5212
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins Hospital
Baltimore, Maryland, 21287
University of Alabama at Birmingham
Birmingham, Alabama, 35233
St. Luke's CF Center of Idaho
Boise, Idaho, 83702
Boston Children's Hospital
Boston, Massachusetts, 02115
Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo
Buffalo, New York, 14222
Clinical Research of Charlotte
Charlotte, North Carolina, 28277
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Duke University Medical Center
Durham, North Carolina, 27710
Cook Children's Medical Center
Fort Worth, Texas, 76104
Cystic Fibrosis Institute
Glenview, Illinois, 60025
Hartford Hospital
Hartford, Connecticut, 06102
Texas Children's Hospital
Houston, Texas, 77030
Indiana University
Indianapolis, Indiana, 46202
The University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
University of Mississippi Medical Center
Jackson, Mississippi, 39216
The Children's Mercy Hospital
Kansas City, Missouri, 64108
University of Tennessee Medical Center
Knoxville, Tennessee, 37920
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
University of Kentucky
Lexington, Kentucky, 40536
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202
Children's Foundation Research Center / Le Bonheur Children's Hospital
Memphis, Tennessee, 38103
University of Miami Miller School of Medicine
Miami, Florida, 33136
Nicklaus Children's Hospital
Miami, Florida, 33155
University of Minnesota
Minneapolis, Minnesota, 55455
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Rutgers Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
Yale New Haven Hospital
New Haven, Connecticut, 06510
Northwell Health- Long Island Jewish Medical Center
New Hyde Park, New York, 11040
Columbia University Medical Center
New York, New York, 10032
Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
Niles, Illinois, 60714
Santiago Reyes, M.D.
Oklahoma City, Oklahoma, 73112
Arnold Palmer Hospital Pulmonary and Sleep Medicine
Orlando, Florida, 32806
Stanford University
Palo Alto, California, 94304
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Oregon Health & Science University
Portland, Oregon, 97239
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, 84132
Seattle Children's Hospital
Seattle, Washington, 98105
Sanford Children's Speciality Clinic
Sioux Falls, South Dakota, 57105
Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
Spokane, Washington, 99204
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, 63110
Johns Hopkins All Children's Hospital Outpatient Care Center
St. Petersburg, Florida, 33701
SUNY Upstate Medical University
Syracuse, New York, 13210
UMass Memorial Medical Center
Worcester, Massachusetts, 01655
Germany9 sites
Charite Paediatric Pulmonology Department
Berlin
Universitaetsklinkum Koeln, CF-Studienzentrum
Cologne
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen
Johann Wolfgang Goethe University
Frankfurt
Hannover Medical School
Hanover
Universitaetsklinikum Jena, Mukoviszidose-Zentrum
Jena
Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin
Lübeck
Klinikum Innenstadt, University of Munich
München
Pneumologisches Studienzentrum Muenchen-West
München
United Kingdom9 sites
Papworth Hospital NHS Foundation Trust, Papworth Everard
Cambridge
NHS Greater Glasgow and Clyde, Gartnavel General Hospital
Glasgow
St. James University Hospital
Leeds
Liverpool Heart and Chest Hospital
Liverpool
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London
Wythenshawe Hospital
Manchester
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
Newcastle upon Tyne
Nottingham University Hospitals NHS Trust, Queens Medical Center
Nottingham
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth
Australia7 sites
Royal Adelaide Hospital
Adelaide
The Prince Charles Hospital
Chermside
Alfred Hospital
Melbourne, VIC
Institute for Respiratory Health
Nedlands
Perth Children's Hospital
Nedlands
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
New Lambton
Queensland Children's Hospital
South Brisbane
Ireland7 sites
Cork University Hospital
Cork
Beaumont Hospital
Dublin
Children's Health Ireland at Crumlin
Dublin
Children's Health Ireland at Temple Street
Dublin
St. Vincent's University Hospital
Dublin
University Hospital Galway
Galway
University Hospital Limerick (Adults)
Limerick
Spain7 sites
Hospital Universitari Vall d Hebron
Barcelona
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona
Hospital Universitario 12 de Octubre
Madrid
Hospital Universitario Infantil La Paz
Madrid
Parc Tauli Sabadell Hospital Universitari
Sabadell
Hospital Universitario Virgen del Rocio
Seville
Hospital Universitario y Politecnico La Fe
Valencia
Israel5 sites
Lady Davis Carmel Medical Center
Haifa
Pediatric Pulmonary Unit Rambam Medical Center
Haifa
Hadassah Medical Organization
Jerusalem
Schneider Children's Medical Center
Petah Tikva
Sheba Medical Center
Tel Litwinsky
Canada3 sites
Stollery Children's Hospital
Edmonton
Queen Elizabeth II Health Sciences Center
Halifax
St. Michael's Hospital
Toronto
Switzerland2 sites
Lindenhofspital - Quartier Bleu
Bern
Kinderspital Zuerich
Zurich
Poland1 site
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
Łomianki
About this study
Official title: A Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis
This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).
- 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) tablet for oral administration.
IVADrug
Also called VX-770, ivacaftor
Tablet for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| ELX/TEZ/IVA | Experimental | Part A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks.
Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. Receives: Drug: ELX/TEZ/IVA; Drug: IVA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: From Baseline through Week 100
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2019
- Study start9 Aug 2019
- Primary completion14 Dec 2022
- Study completion14 Dec 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 (2)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
- 458 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
- NCT04043806
- Sponsor's protocol ID
- VX18-445-113
- Other IDs
- 2018-004652-38, 2018
- Status last verified
- Jun 2023
- First submitted
- 31 Jul 2019
- Last updated
- 6 Jul 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
- Expanded access
- Available
Publications
Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PubMed 37983082Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
PubMed 33331662
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2023. 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 of VX-445 Combination Therapy in Subjects With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT04043806
- Acronym / sponsor ID
- VX18-445-113
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 458 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 9 Aug 2019 (actual)
- Primary completion
- 14 Dec 2022 (actual)
- Study completion
- 14 Dec 2022 (actual)
- First posted
- 2 Aug 2019 (actual)
- Results
- Posted 6 Jul 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 evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).
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: IVAPart A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Currently participating in study VX17-659-105 (NCT03447262)
Key exclusion criteria (3 listed)
- History of drug intolerance in study VX17-659-105 that would pose an additional risk to the participant in the opinion of the investigator
- Current participation in an investigational drug trial (other than study VX17-659-105)
- 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 (FDC) tablet for oral administration. | Drug | ELX/TEZ/IVA |
| IVAAlso: VX-770, ivacaftorTablet for oral administration. | Drug | ELX/TEZ/IVA |
Primary outcome
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: From Baseline through Week 100
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37983082Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
- Derived from this study: PubMed 33331662Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
- 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
Related trials for Cystic Fibrosis
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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.
97 sites in 11 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 97 sites in 11 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 6 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 97 sites in United States, Germany, United Kingdom, Australia, Ireland and 6 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 6 Jul 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?
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (time frame: From Baseline through Week 100). 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 NCT04043806, which also lists 2 linked publications.
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 6 Jul 2023; AzTrials last refreshed its copy on 31 Oct 2026.