Cystic FibrosisPhase 2NCT03224351
A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
A Phase 2 study testing VX-659, TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 47 sites in 4 countries.
Trial summary
This is a Phase 2, randomized, double-blind, placebo- and tezacaftor/ivacaftor (TEZ/IVA)-controlled, parallel-group, 3-part, multicenter study designed to evaluate the safety and efficacy of VX-659 in triple combination (TC) with TEZ and IVA in subjects with cystic fibrosis (CF) who are homozygous for the F508del… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- VX-659, TEZ/IVA and IVA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 124 participants
- Where
- 47 sitesin 4 countries
- Study dates
- 8 Aug 2017to 28 Feb 2018
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Body weight ≥35 kg.
- Subjects must have an eligibleCFTR genotype.
- Part 1 and Part 3: Heterozygous for F508del and an MF mutation (F/MF)
- Part 2: Homozygous for F508del (F/F)
- FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height
Who cannot join
- History of clinically significant cirrhosis with or without portal hypertension.
- Glucose-6-phosphate dehydrogenase (G6PD) deficiency
- Lung infection with organisms associated with a more rapid decline in pulmonary status.
- History of solid organ or hematological transplantation.
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
47 sites in 4 countries.
47 sites in 4 countries
United States25 sites
Albany Medical College
Albany, New York, 12208
University of Michigan Health System
Ann Arbor, Michigan, 48109
The Johns Hopkins Hospital/ Johns Hopkins Hospital, David Rubenstein Child Health Building
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02155
Helen DeVos Children's Hospital CF Center
Grand Rapids, Michigan, 49503
Indiana University Health
Indianapolis, Indiana, 46202
The University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Children's Mercy Hospital
Kansas City, Missouri, 64108
University of Tennessee Medical Center-Adult Cystic Fibrosis Clinic
Knoxville, Tennessee, 37920
Children's Foundation Research Center / Le Bonheur Children's Hospital
Memphis, Tennessee, 38103
University of Miami/Miller School of Medicine
Miami, Florida, 33136
Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
Morton Grove, Illinois, 60053
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Rutgers-Robert Wood Johnson Medical School/ Rutgers-Robert Wood Johnson Medical School, Clinical Research Center
New Brunswick, New Jersey, 08902
Yale New Haven Hospital
New Haven, Connecticut, 06520
Northwell Health, Long Island Jewish Medical Center
New Hyde Park, New York, 11040
Columbia University Medical Center
New York, New York, 10032
Respiratory Diseases of Children & Adolescents
Oklahoma City, Oklahoma, 73112
Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, 84014
Seattle Children's Hospital
Seattle, Washington, 98105
Sanford Research / USD
Sioux Falls, South Dakota, 57104
Providence Pediatric Pulmonary & Allergy/Immunology Clinic
Spokane, Washington, 99204
SUNY Upstate Medical University
Syracuse, New York, 13210
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01655
United Kingdom13 sites
Birmingham Heartlands Hospital
Birmingham, B95SS
Papworth Hospital NHS Foundation Trust, Papworth Everard
Cambridge
University Hospital Llandough in Cardiff
Cardiff
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
Devon
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
Fulham
Greater Glasgow and Clyde NHS Board, Glasgow Clinical Research Facility
Glasgow
Southampton University Hospitals NHS Foundation Trust
Hampshire
Regional Respiratory Centre Belfast City Hospital
London
Royal Brompton & Harefied NHS Foundation Trust
London
University Hospital of South Manchester NHS Trust, North West Lung Centre
Manchester
Liverpool Heart and Chest Hospital
Merseyside
Nottingham University Hospitals NHS Trust
Nottingham
Ruth Children's Hospital Rambam Health Care Campus
Nottingham
Israel5 sites
Carmel Medical Center
Haifa
Ruth Children's Hospital Rambam Health Care Campus
Haifa
Hadassah Medical Organization
Jerusalem
The Chaim Sheba medical center
Ramat Gan
Schneider Children's Medical Center
Tikvah
Ireland4 sites
Cork University Hospital
Cork
St. Vincent's University Hosptial
Dublin
Galway University Hospitals
Galway
University Hospital Limerick
Limerick
About this study
Official title: A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-659 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis
This is a Phase 2, randomized, double-blind, placebo- and tezacaftor/ivacaftor (TEZ/IVA)-controlled, parallel-group, 3-part, multicenter study designed to evaluate the safety and efficacy of VX-659 in triple combination (TC) with TEZ and IVA in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F/F genotype), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ, IVA, or TEZ/IVA (F/MF genotypes).
- Conditions
- Cystic Fibrosis
- Being tested
- VX-659, TEZ/IVA, IVA, Placebo (matched to VX-659/TEZ/IVA), TEZ
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-659Drug
Tablet for oral administration.
TEZ/IVADrug
Also called VX-661/VX-770, Tezacaftor/Ivacaftor
TEZ/IVA fixed-dose combination tablet for oral administration.
IVADrug
Also called VX-770, Ivacaftor
Tablet for oral administration.
Placebo (matched to VX-659/TEZ/IVA)Drug
Placebo matched to VX-659 and TEZ/IVA.
TEZDrug
Also called VX-661, Tezacaftor
Tablet for oral administration.
VX-561Drug
Also called CTP-656
Tablet for oral administration.
Placebo (matched to VX-659/TEZ/VX-561)Drug
Placebo matched to VX-659, TEZ and VX-561.
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Placebo | Placebo comparator | Participants received placebo matched to VX-659/TEZ/IVA in TC treatment period for 4 weeks and placebo matched TEZ/IVA in washout period for 4 days. Receives: Drug: Placebo (matched to VX-659/TEZ/IVA) |
| Part 1: VX-659/TEZ/IVA TC - Low Dose | Experimental | Participants received VX-659 80 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days. Receives: Drug: VX-659; Drug: TEZ/IVA; Drug: IVA |
| Part 1: VX-659/TEZ/IVA TC - Medium Dose | Experimental | Participants received VX-659 240 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days. Receives: Drug: VX-659; Drug: TEZ/IVA; Drug: IVA |
| Part 1: VX-659/TEZ/IVA TC - High Dose | Experimental | Participants received VX-659 400 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days. Receives: Drug: VX-659; Drug: TEZ/IVA; Drug: IVA |
| Part 2: TEZ/IVA | Active comparator | Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 weeks. Receives: Drug: TEZ/IVA; Drug: IVA |
| Part 2: VX-659/TEZ/IVA TC | Experimental | Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-659 400 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 weeks. Receives: Drug: VX-659; Drug: TEZ/IVA; Drug: IVA |
| Part 3: Placebo | Placebo comparator | Participants received placebo matched to VX-659/TEZ/VX-561 in TC treatment period for 4 weeks. Receives: Drug: Placebo (matched to VX-659/TEZ/VX-561) |
| Part 3: VX-659/TEZ/VX-561 TC | Experimental | Participants received VX-659 400 mg qd/TEZ 100 mg qd/VX-561 200 mg qd in TC treatment period for 4 weeks. Receives: Drug: VX-659; Drug: TEZ; Drug: VX-561 |
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 Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: Day 1 Through Safety Follow-up (up to Day 61 for Part 1, Day 85 for Part 2 and Day 57 for Part 3)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Measured: From Baseline Through Day 29
Secondary outcomes (4)
Absolute Change in Sweat Chloride Concentrations
Sweat samples were collected using an approved collection device.
Measured: From Baseline Through Day 29
Relative Change in ppFEV1
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Measured: From Baseline Through Day 29
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Measured: From Baseline at Day 29
Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, IVA, M1-IVA, and VX-561
Measured: Pre-dose at Day 15 and Day 29
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2017
- Study start8 Aug 2017
- Primary completion28 Feb 2018
- Study completion28 Feb 2018
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 (3)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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 124 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
- NCT03224351
- Sponsor's protocol ID
- VX16-659-101
- Other IDs
- 2016-003585-11, 2016
- Status last verified
- Feb 2021
- First submitted
- 18 Jul 2017
- Last updated
- 22 Apr 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
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 33331662Davies JC, Moskowitz SM, Brown C, Horsley A, Mall MA, McKone EF, Plant BJ, Prais D, Ramsey BW, Taylor-Cousar JL, Tullis E, Uluer A, McKee CM, Robertson S, Shilling RA, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Rowe SM; VX16-659-101 Study Group. VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1599-1611. doi: 10.1056/NEJMoa1807119. Epub 2018 Oct 18.
PubMed 30334693
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-659 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03224351
- Acronym / sponsor ID
- VX16-659-101
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 124 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 8 Aug 2017 (actual)
- Primary completion
- 28 Feb 2018 (actual)
- Study completion
- 28 Feb 2018 (actual)
- First posted
- 21 Jul 2017 (actual)
- Results
- Posted 22 Apr 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 2, randomized, double-blind, placebo- and tezacaftor/ivacaftor (TEZ/IVA)-controlled, parallel-group, 3-part, multicenter study designed to evaluate the safety and efficacy of VX-659 in triple combination (TC) with TEZ and IVA in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F/F genotype), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ, IVA, or TEZ/IVA (F/MF genotypes).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| Part 1: Placebo | Placebo comparator | Drug: Placebo (matched to VX-659/TEZ/IVA)Participants received placebo matched to VX-659/TEZ/IVA in TC treatment period for 4 weeks and placebo matched TEZ/IVA in washout period for 4 days. |
| Part 1: VX-659/TEZ/IVA TC - Low Dose | Experimental | Drug: VX-659, Drug: TEZ/IVA, Drug: IVAParticipants received VX-659 80 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days. |
| Part 1: VX-659/TEZ/IVA TC - Medium Dose | Experimental | Drug: VX-659, Drug: TEZ/IVA, Drug: IVAParticipants received VX-659 240 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days. |
| Part 1: VX-659/TEZ/IVA TC - High Dose | Experimental | Drug: VX-659, Drug: TEZ/IVA, Drug: IVAParticipants received VX-659 400 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days. |
| Part 2: TEZ/IVA | Active comparator | Drug: TEZ/IVA, Drug: IVAFollowing run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 weeks. |
| Part 2: VX-659/TEZ/IVA TC | Experimental | Drug: VX-659, Drug: TEZ/IVA, Drug: IVAFollowing run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-659 400 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 weeks. |
| Part 3: Placebo | Placebo comparator | Drug: Placebo (matched to VX-659/TEZ/VX-561)Participants received placebo matched to VX-659/TEZ/VX-561 in TC treatment period for 4 weeks. |
| Part 3: VX-659/TEZ/VX-561 TC | Experimental | Drug: VX-659, Drug: TEZ, Drug: VX-561Participants received VX-659 400 mg qd/TEZ 100 mg qd/VX-561 200 mg qd in TC treatment period for 4 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Body weight ≥35 kg.
- Subjects must have an eligibleCFTR genotype.
- FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height
Key exclusion criteria (5 listed)
- History of clinically significant cirrhosis with or without portal hypertension.
- Glucose-6-phosphate dehydrogenase (G6PD) deficiency
- Lung infection with organisms associated with a more rapid decline in pulmonary status.
- History of solid organ or hematological transplantation.
- 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 |
|---|---|---|
| VX-659Tablet for oral administration. | Drug | Part 1: VX-659/TEZ/IVA TC - High Dose, Part 1: VX-659/TEZ/IVA TC - Low Dose, Part 1: VX-659/TEZ/IVA TC - Medium Dose, Part 2: VX-659/TEZ/IVA TC, Part 3: VX-659/TEZ/VX-561 TC |
| TEZ/IVAAlso: VX-661/VX-770, Tezacaftor/IvacaftorTEZ/IVA fixed-dose combination tablet for oral administration. | Drug | Part 1: VX-659/TEZ/IVA TC - High Dose, Part 1: VX-659/TEZ/IVA TC - Low Dose, Part 1: VX-659/TEZ/IVA TC - Medium Dose, Part 2: TEZ/IVA, Part 2: VX-659/TEZ/IVA TC |
| IVAAlso: VX-770, IvacaftorTablet for oral administration. | Drug | Part 1: VX-659/TEZ/IVA TC - High Dose, Part 1: VX-659/TEZ/IVA TC - Low Dose, Part 1: VX-659/TEZ/IVA TC - Medium Dose, Part 2: TEZ/IVA, Part 2: VX-659/TEZ/IVA TC |
| Placebo (matched to VX-659/TEZ/IVA)Placebo matched to VX-659 and TEZ/IVA. | Drug | Part 1: Placebo |
| TEZAlso: VX-661, TezacaftorTablet for oral administration. | Drug | Part 3: VX-659/TEZ/VX-561 TC |
| VX-561Also: CTP-656Tablet for oral administration. | Drug | Part 3: VX-659/TEZ/VX-561 TC |
| Placebo (matched to VX-659/TEZ/VX-561)Placebo matched to VX-659, TEZ and VX-561. | Drug | Part 3: Placebo |
Primary outcome
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 Through Safety Follow-up (up to Day 61 for Part 1, Day 85 for Part 2 and Day 57 for Part 3)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: From Baseline Through Day 29
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
4 secondary outcomes
Absolute Change in Sweat Chloride Concentrations
Time frame: From Baseline Through Day 29
Relative Change in ppFEV1
Time frame: From Baseline Through Day 29
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
Time frame: From Baseline at Day 29
Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, IVA, M1-IVA, and VX-561
Time frame: Pre-dose at Day 15 and Day 29
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2021.
- 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.
- Derived from this study: PubMed 30334693Davies JC, Moskowitz SM, Brown C, Horsley A, Mall MA, McKone EF, Plant BJ, Prais D, Ramsey BW, Taylor-Cousar JL, Tullis E, Uluer A, McKee CM, Robertson S, Shilling RA, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Rowe SM; VX16-659-101 Study Group. VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1599-1611. doi: 10.1056/NEJMoa1807119. Epub 2018 Oct 18.
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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.
47 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VX-659, TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 47 sites in 4 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 5 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 22 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 47 sites in United States, United Kingdom, Israel, Ireland. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 22 Apr 2021). 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?
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (time frame: Day 1 Through Safety Follow-up (up to Day 61 for Part 1, Day 85 for Part 2 and Day 57 for Part 3)). The study lists 2 primary outcomes in all. 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 NCT03224351, which also lists 3 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 22 Apr 2021; AzTrials last refreshed its copy on 6 Nov 2026.