Cystic FibrosisPhase 3NCT02544451
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
A Phase 3 study testing LUM/IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 60 sites in 9 countries.
Trial summary
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- LUM/IVA
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 246 participants
- Where
- 60 sitesin 9 countries
- Study dates
- Aug 2015to Apr 2020
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects entering the Treatment Cohort must meet both of the following criteria:
- Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment
- Willing to remain on a stable CF medication through the Safety Follow-up Visit.
- Subjects entering the Observational Cohort must meet 1 of the following criteria:
- Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B.
- Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.
Who cannot join
- History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject (e.g., cirrhosis with portal hypertension).
- Pregnant and nursing females.
- Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
- History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator
- History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator.
- Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor).
Trial locations and contacts
60 sites in 9 countries.
60 sites in 9 countries
United States37 sites
Study site
Atlanta, Georgia
Study site
Aurora, Colorado
Study site
Austin, Texas
Study site
Birmingham, Alabama
Study site
Boston, Massachusetts
Study site
Buffalo, New York
Study site
Chapel Hill, North Carolina
Study site
Charleston, South Carolina
Study site
Charlottesville, Virginia
Study site
Chicago, Illinois
Study site
Cincinnati, Ohio
Study site
Cleveland, Ohio
Study site
Colchester, Vermont
Study site
Dayton, Ohio
Study site
Houston, Texas
Study site
Indianapolis, Indiana
Study site
Iowa City, Iowa
Study site
Jacksonville, Florida
Study site
Kansas City, Missouri
Study site
Lebanon, New Hampshire
Study site
Long Beach, California
Study site
Madison, Wisconsin
Study site
Milwaukee, Wisconsin
Study site
Minneapolis, Minnesota
Study site
Norfolk, Virginia
Study site
Omaha, Nebraska
Study site
Orlando, Florida
Study site
Palo Alto, California
Study site
Pittsburgh, Pennsylvania
Study site
Portland, Oregon
Study site
Richmond, Virginia
Study site
Salt Lake City, Utah
Study site
Seattle, Washington
Study site
St Louis, Missouri
Study site
Syracuse, New York
Study site
Tucson, Arizona
Study site
Wilmington, Delaware
Australia5 sites
Study site
Herston
Study site
New Lambton Heights
Study site
Parkville, Victoria
Study site
Subiaco
Study site
Westmead
Germany4 sites
Study site
Berlin
Study site
Cologne
Study site
Giessen
Study site
Hanover
United Kingdom4 sites
Study site
Belfast
Study site
Edinburgh
Study site
Leeds, West Yorkshire
Study site
London
Canada3 sites
Study site
Montreal, Quebec
Study site
Toronto, Ontario
Study site
Vancouver, British Columbia
About this study
Official title: A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.
- Conditions
- Cystic Fibrosis
- Being tested
- LUM/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.
LUM/IVADrug
Also called VX-809/VX-770, lumacaftor/ivacaftor
Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).
LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).
LUM/IVADrug
Also called VX-809/VX-770, lumacaftor/ivacaftor
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
| Study group | Role | What they receive |
|---|---|---|
| Treatment Period 1: LUM/IVA to LUM/IVA | Experimental | Not described Receives: Drug: LUM/IVA |
| Treatment Period 1: Placebo (PBO) to LUM/IVA | Experimental | Not described Receives: Drug: LUM/IVA |
| Treatment Period 1: Observational Cohort | No intervention | Not described |
| Treatment Period 2: LUM/IVA | Experimental | Not described Receives: Drug: LUM/IVA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Day 1 up to Week 100
Secondary outcomes (21)
Absolute Change in Lung Clearance Index (LCI) 2.5
LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
Measured: From Parent Study Baseline at Week 96
Absolute Change in Sweat Chloride
Sweat samples were collected using an approved collection device.
Measured: From Parent Study Baseline at Week 96
Absolute Change in Body Mass Index (BMI)
BMI was defined as weight in kilograms divided by height in square meter (m^2).
Measured: From Parent Study Baseline at Week 96
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 Parent Study Baseline at Week 96
Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)
Measured: Day 1 up to Week 100
Absolute Change in LCI 5.0
LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value.
Measured: From Parent Study Baseline at Week 96
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 Parent Study Baseline at Week 96
Relative Change in ppFEV1
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Measured: From Parent Study Baseline at Week 96
Absolute Change in BMI-for-age Z-score
BMI was defined as weight in kilograms divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
Measured: From Parent Study Baseline at Week 96
Absolute Change in Weight
Measured: From Parent Study Baseline at Week 96
Absolute Change in Weight-for-age Z-score
z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
Measured: From Parent Study Baseline at Week 96
Absolute Change in Height
Measured: From Parent Study Baseline at Week 96
Absolute Change in Height-for-age Z-score
z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
Measured: From Parent Study Baseline at Week 96
Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score
The TSQM measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed in the total domain score, which ranges from 0 to 100, where higher scores indicate greater satisfaction.
Measured: From Parent Study Baseline at Week 96
Time-to-first Pulmonary Exacerbation
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Measured: From Parent Study Baseline through Week 96
Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Measured: From Parent Study Baseline through Week 96
Number of Pulmonary Exacerbation Events Per Patient-year
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Measured: From Parent Study Baseline through Week 96
Rate of Change in LCI 2.5
Rate of change analysis evaluates the change in LCI 2.5 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
Measured: Day 15 after first dose of LUM/IVA through Week 96
Rate of Change in LCI 5.0
Rate of change analysis evaluates the change in LCI 5.0 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
Measured: Day 15 after first dose of LUM/IVA through Week 96
Rate of Change in ppFEV1
Rate of change analysis evaluates the change in ppFEV1 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
Measured: Day 15 after first dose of LUM/IVA through Week 96
Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Day 1 up to Week 168
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2015 (estimated)
- Study startAug 2015
- Primary completionAug 2018
- Study completionApr 2020
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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 246 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
- NCT02544451
- Sponsor's protocol ID
- VX15-809-110
- Status last verified
- Feb 2021
- First submitted
- 1 Sep 2015
- Last updated
- 24 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Chilvers MA, Davies JC, Milla C, Tian S, Han Z, Cornell AG, Owen CA, Ratjen F. Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study. Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28.
PubMed 33516285
Study at a glance
From the registry record, last updated by the sponsor on 24 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
- ClinicalTrials.gov ID
- NCT02544451
- Acronym / sponsor ID
- VX15-809-110
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 246 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- Aug 2015 (actual)
- Primary completion
- Aug 2018 (actual)
- Study completion
- Apr 2020 (actual)
- First posted
- 9 Sep 2015 (estimated)
- Results
- Posted 29 Oct 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Treatment Period 1: LUM/IVA to LUM/IVA | Experimental | Drug: LUM/IVA |
| Treatment Period 1: Placebo (PBO) to LUM/IVA | Experimental | Drug: LUM/IVA |
| Treatment Period 1: Observational Cohort | No intervention | – |
| Treatment Period 2: LUM/IVA | Experimental | Drug: LUM/IVA |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment
- Willing to remain on a stable CF medication through the Safety Follow-up Visit.
- Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B.
- Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.
Key exclusion criteria (6 listed)
- History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject (e.g., cirrhosis with portal hypertension).
- Pregnant and nursing females.
- Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
- History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator
- History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator.
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 |
|---|---|---|
| LUM/IVAAlso: VX-809/VX-770, lumacaftor/ivacaftorLumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age). LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older). | Drug | Treatment Period 1: LUM/IVA to LUM/IVA, Treatment Period 1: Placebo (PBO) to LUM/IVA |
| LUM/IVAAlso: VX-809/VX-770, lumacaftor/ivacaftorLUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age). | Drug | Treatment Period 2: LUM/IVA |
Primary outcome
Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 100
21 secondary outcomes
Absolute Change in Lung Clearance Index (LCI) 2.5
Time frame: From Parent Study Baseline at Week 96
Absolute Change in Sweat Chloride
Time frame: From Parent Study Baseline at Week 96
Absolute Change in Body Mass Index (BMI)
Time frame: From Parent Study Baseline at Week 96
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
Time frame: From Parent Study Baseline at Week 96
Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 100
Absolute Change in LCI 5.0
Time frame: From Parent Study Baseline at Week 96
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: From Parent Study Baseline at Week 96
Relative Change in ppFEV1
Time frame: From Parent Study Baseline at Week 96
Absolute Change in BMI-for-age Z-score
Time frame: From Parent Study Baseline at Week 96
Absolute Change in Weight
Time frame: From Parent Study Baseline at Week 96
Absolute Change in Weight-for-age Z-score
Time frame: From Parent Study Baseline at Week 96
Absolute Change in Height
Time frame: From Parent Study Baseline at Week 96
Absolute Change in Height-for-age Z-score
Time frame: From Parent Study Baseline at Week 96
Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score
Time frame: From Parent Study Baseline at Week 96
Time-to-first Pulmonary Exacerbation
Time frame: From Parent Study Baseline through Week 96
Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event
Time frame: From Parent Study Baseline through Week 96
Number of Pulmonary Exacerbation Events Per Patient-year
Time frame: From Parent Study Baseline through Week 96
Rate of Change in LCI 2.5
Time frame: Day 15 after first dose of LUM/IVA through Week 96
Rate of Change in LCI 5.0
Time frame: Day 15 after first dose of LUM/IVA through Week 96
Rate of Change in ppFEV1
Time frame: Day 15 after first dose of LUM/IVA through Week 96
Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 168
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33516285Chilvers MA, Davies JC, Milla C, Tian S, Han Z, Cornell AG, Owen CA, Ratjen F. Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study. Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28.
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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.
60 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing LUM/IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 60 sites in 9 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 6 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 24 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 60 sites in United States, Australia, Germany, United Kingdom, Canada and 4 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 29 Oct 2019). 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?
Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Day 1 up to 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 NCT02544451, 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 24 May 2021.