Cystic FibrosisPhase 3NCT01117012
Rollover Study of VX-770 in Cystic Fibrosis Subjects
A Phase 3 study testing Ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 58 sites in 8 countries.
Trial summary
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF. 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?
- Ivacaftor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 192 participants
- Where
- 58 sitesin 8 countries
- Study dates
- Jul 2010to May 2014
- 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
- Participants who have completed the assigned study treatment in Study 102 or Study 103
- Participants who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770)
- Participants who are able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator
- Participants of child bearing potential and who are sexually active must meet the contraception requirements
- Participants must sign the informed consent form (ICF), and where appropriate, assent must be obtained
Who cannot join
- Participants with a history of any illness or condition 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
- Participants with a history of study treatment intolerance as observed in their previous VX-770 study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
- Participants who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements
- Participants taking any inhibitors or inducers of Cytochrome P450 3A4 (CYP3A4), including certain herbal medications (for example, St. John's Wort) and grapefruit/grapefruit juice
Trial locations and contacts
58 sites in 8 countries.
58 sites in 8 countries
United States36 sites
Study site
Ann Arbor, Michigan
Study site
Atlanta, Georgia
Study site
Baltimore, Maryland
Study site
Birmingham, Alabama
Study site
Boise, Idaho
Study site
Boston, Massachusetts
Study site
Buffalo, New York
Study site
Chicago, Illinois
Study site
Chrlottesville, Virginia
Study site
Cincinnati, Ohio
Study site
Cleveland, Ohio
Study site
Columbus, Ohio
Study site
Denver, Colorado
Study site
Detroit, Michigan
Study site
Hershey, Pennsylvania
Study site
Indianapolis, Indiana
Study site
Iowa City, Iowa
Study site
Kansas City, Missouri
Study site
Knoxville, Tennessee
Study site
Long Branch, New Jersey
Study site
Minneapolis, Minnesota
Study site
Morgantown, West Virginia
Study site
Nashville, Tennessee
Study site
New Hyde Park, New York
Study site
Oakland, California
Study site
Omaha, Nebraska
Study site
Palo Alto, California
Study site
Philadelphia, Pennsylvania
Study site
Pittsburgh, Pennsylvania
Study site
Portland, Oregon
Study site
Salt Lake City, Utah
Study site
San Diego, California
Study site
Seattle, Washington
Study site
St Louis, Missouri
Study site
Syracuse, New York
Study site
Toledo, Ohio
Australia7 sites
Study site
Chermside, Queensland
Study site
Herston, Queensland
Study site
Nedlands, Western Australia
Study site
Parkville, Victoria
Study site
South Brisbane, Queensland
Study site
Subiaco, Western Australia
Study site
Westmead, New South Wales
Canada4 sites
Study site
Halifax, Nova Scotia
Study site
Montreal, Quebec
Study site
Toronto, Ontario
Study site
Vancouver, British Columbia
Germany4 sites
Study site
Erlangen
Study site
Jena
Study site
Munich
Study site
Würzburg
United Kingdom2 sites
Study site
Belfast, Northern Ireland
Study site
London
About this study
Official title: An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF.
Read the detailed description
This open-label, rollover study of orally administered VX-770 was conducted in participants with CF to evaluate the safety and efficacy of long-term VX-770 treatment. Participants who were previously enrolled in Study 102 (VX08-770-102/NCT00909532) and Study 103 (VX08-770-103/NCT00909727), and met certain criteria were eligible to enroll in this study (Study 105/VX08-770-105/NCT01117012).
- Conditions
- Cystic Fibrosis
- Being tested
- Ivacaftor
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IvacaftorDrug
Also called VX-770
| Study group | Role | What they receive |
|---|---|---|
| VX-770 | Experimental | VX-770 (ivacaftor) 150 milligram (mg) tablet orally twice daily (q12h). Receives: Drug: Ivacaftor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
Adverse Event: any untoward medical occurrence in a participant during the study, including any unfavorable and unintended sign, symptom, or disease whether or not it was considered to be study drug related. This included any newly occurring event or previous condition that increased in severity or frequency after obtaining informed consent and assent (where applicable). SAE: medical event or condition, which resulted in any of following, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Non-Serious AEs included all AEs except SAEs.
Measured: Study 105: Day 1 up to Week 168
Secondary outcomes (6)
Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as Study 105 Day 15.
Measured: Study 105: Baseline through Week 96
Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in Study 105. Absolute Change at Week 48 and Week 96 are reported.
Measured: Study 105: Baseline through Week 96
Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Absolute Change at Week 48 and Week 96 are reported.
Measured: Study 105: Baseline through Week 96
Annualized Pulmonary Exacerbation Event Rate
Annualized event rate was calculated by regression with negative binomial distribution.
Measured: Study 105: Day 1 through Week 96
Annualized Duration of Pulmonary Exacerbation Events
Measured: Study 105: Day 1 through Week 96
Absolute Change From Study 105 Baseline in Weight Through Week 96
Weight is a measurement of nutritional status. Absolute change in weight, measured in kilograms (kg), at Week 48 and Week 96 are reported.
Measured: Study 105: Baseline through Week 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2010 (estimated)
- Study startJul 2010
- Primary completionMay 2014
- Study completionMay 2014
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
- 192 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Edward McKone, MD, Principal investigator, St Vincent's University Hospital, Ireland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01117012
- Sponsor's protocol ID
- VX08-770-105
- Other IDs
- PERSIST
- Status last verified
- Jun 2015
- First submitted
- 3 May 2010
- Last updated
- 7 Jul 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
McKone EF, Borowitz D, Drevinek P, Griese M, Konstan MW, Wainwright C, Ratjen F, Sermet-Gaudelus I, Plant B, Munck A, Jiang Y, Gilmartin G, Davies JC; VX08-770-105 (PERSIST) Study Group. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). Lancet Respir Med. 2014 Nov;2(11):902-910. doi: 10.1016/S2213-2600(14)70218-8. Epub 2014 Oct 9.
PubMed 25311995
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT01117012
- Acronym / sponsor ID
- VX08-770-105
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 192 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Jul 2010
- Primary completion
- May 2014 (actual)
- Study completion
- May 2014 (actual)
- First posted
- 5 May 2010 (estimated)
- Results
- Posted 7 Jul 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF.
Detailed description
This open-label, rollover study of orally administered VX-770 was conducted in participants with CF to evaluate the safety and efficacy of long-term VX-770 treatment. Participants who were previously enrolled in Study 102 (VX08-770-102/NCT00909532) and Study 103 (VX08-770-103/NCT00909727), and met certain criteria were eligible to enroll in this study (Study 105/VX08-770-105/NCT01117012).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VX-770 | Experimental | Drug: IvacaftorVX-770 (ivacaftor) 150 milligram (mg) tablet orally twice daily (q12h). |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Participants who have completed the assigned study treatment in Study 102 or Study 103
- Participants who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770)
- Participants who are able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator
- Participants of child bearing potential and who are sexually active must meet the contraception requirements
- Participants must sign the informed consent form (ICF), and where appropriate, assent must be obtained
Key exclusion criteria (4 listed)
- Participants with a history of any illness or condition 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
- Participants with a history of study treatment intolerance as observed in their previous VX-770 study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
- Participants who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements
- Participants taking any inhibitors or inducers of Cytochrome P450 3A4 (CYP3A4), including certain herbal medications (for example, St. John's Wort) and grapefruit/grapefruit juice
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 |
|---|---|---|
| IvacaftorAlso: VX-770 | Drug | VX-770 |
Primary outcome
Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Study 105: Day 1 up to Week 168
Adverse Event: any untoward medical occurrence in a participant during the study, including any unfavorable and unintended sign, symptom, or disease whether or not it was considered to be study drug related. This included any newly occurring event or previous condition that increased in severity or frequency after obtaining informed consent and assent (where applicable). SAE: medical event or condition, which resulted in any of following, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Non-Serious AEs included all AEs except SAEs.
6 secondary outcomes
Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96
Time frame: Study 105: Baseline through Week 96
Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96
Time frame: Study 105: Baseline through Week 96
Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96
Time frame: Study 105: Baseline through Week 96
Annualized Pulmonary Exacerbation Event Rate
Time frame: Study 105: Day 1 through Week 96
Annualized Duration of Pulmonary Exacerbation Events
Time frame: Study 105: Day 1 through Week 96
Absolute Change From Study 105 Baseline in Weight Through Week 96
Time frame: Study 105: Baseline through Week 96
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 25311995McKone EF, Borowitz D, Drevinek P, Griese M, Konstan MW, Wainwright C, Ratjen F, Sermet-Gaudelus I, Plant B, Munck A, Jiang Y, Gilmartin G, Davies JC; VX08-770-105 (PERSIST) Study Group. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). Lancet Respir Med. 2014 Nov;2(11):902-910. doi: 10.1016/S2213-2600(14)70218-8. Epub 2014 Oct 9.
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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.
Edward McKone, MD principal investigator
St Vincent's University Hospital, Ireland
58 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 58 sites in 8 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 4 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 7 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 58 sites in United States, Australia, Canada, Germany, France and 3 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 7 Jul 2015). 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?
Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Study 105: Day 1 up to Week 168). 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 NCT01117012, 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 7 Jul 2015.