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Cystic FibrosisPhase 3KONTINUENCT01707290

Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation

A Phase 3 study testing Ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 38 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801). 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
125 participants
Where
38 sitesin 4 countries
Study dates
Feb 2013to Apr 2016
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 from Study 110 or Study 111 entering the ivacaftor arm must have completed the assigned study drug treatment duration in the previous study.
  • Participants from Study 113 entering the ivacaftor arm must have completed all study related treatments through the Follow-up Visit and met the Study 113 responder criteria during the previous study.
  • Participants entering the observational arm must have completed at least 4 weeks of study drug treatment in their previous study (Study 110 or Study 111), must have completed the previous study but do not wish to enroll in the ivacaftor arm, or must have completed the previous study but do not meet the inclusion criteria of the ivacaftor arm.
  • Participants of childbearing potential entering the ivacaftor arm must not be pregnant.
  • Participants entering the ivacaftor arm must be willing to comply with contraception requirements.

Who cannot join

  • History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the Participant.
  • Use of moderate or strong inhibitors or inducers of cytochrome P450 (CYP) 3A.
  • Evidence of cataract or lens opacity at or before the Day 1 Visit.

Trial locations and contacts

38 sites in 4 countries.

38 sites in 4 countries

United States33 sites

  • Study site

    Ann Arbor, Michigan

  • Study site

    Atlanta, Georgia

  • Study site

    Baltimore, Maryland

  • Study site

    Birmingham, Alabama

  • Study site

    Boston, Massachusetts

  • Study site

    Chapel Hill, North Carolina

  • Study site

    Charleston, South Carolina

  • Study site

    Chicago, Illinois

  • Study site

    Dallas, Texas

  • Study site

    Denver, Colorado

  • Study site

    Detroit, Michigan

  • Study site

    Fort Worth, Texas

  • Study site

    Grand Rapids, Michigan

  • Study site

    Hartford, Connecticut

  • Study site

    Houston, Texas

  • Study site

    Iowa City, Iowa

  • Study site

    Lexington, Kentucky

  • Study site

    Madison, Wisconsin

  • Study site

    Minneapolis, Minnesota

  • Study site

    Morgantown, West Virginia

  • Study site

    Nashville, Tennessee

  • Study site

    New York, New York

  • Study site

    Omaha, Nebraska

  • Study site

    Palo Alto, California

  • Study site

    Philadelphia, Pennsylvania

  • Study site

    Pittsburgh, Pennsylvania

  • 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

    Tampa, Florida

  • Study site

    Valhalla, New York

Other trials in United States

France2 sites

  • Study site

    Montpellier

  • Study site

    Paris

Other trials in France

United Kingdom2 sites

  • Study site

    Belfast

  • Study site

    Edinburgh

Other trials in United Kingdom

Belgium1 site

  • Study site

    Leuven

Other trials in Belgium

About this study

Official title: A Phase 3, Two-Arm, Rollover Study to Evaluate the Safety of Long Term Ivacaftor Treatment in Subjects 6 Years of Age and Older With Cystic Fibrosis and a Non-G551D CFTR Mutation

The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).

Read the detailed description

Ivacaftor is the first Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator to show an improvement in CFTR function and clinical benefit in participants with CF. Results from Phase 3 studies (NCT00909532 [Study 102] and NCT00909727 [Study 103]) showed that ivacaftor is effective in the treatment of participants with CF who have the G551D-CFTR mutation, as evidenced by sustained improvements in CFTR channel function (measured by reduction in sweat chloride concentration) and corresponding substantial, durable improvements in lung function, pulmonary exacerbations, respiratory symptoms, and weight gain. Ivacaftor was also well tolerated, as evidenced by the rates and reasons for premature discontinuation and results of safety assessments.

Ivacaftor (Trade Name Kalydeco; 150 mg tablets) was initially approved in the United States for the treatment of CF in participants 6 years of age and older who have a G551D mutation in the CFTR gene.

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 Kalydeco, VX-770

    150 mg tablet, oral use, every 12 hours (q12h)

Study groups in NCT01707290
Study groupRoleWhat they receive
IvacaftorExperimentalParticipants who received Ivacaftor 150 milligram (mg) tablet and/or Placebo matched to Ivacaftor tablet orally, every 12 hours (q12h) in the previous study VX11-770-110 (Study 110; NCT01614457), VX12-770-111 (Study 111; NCT01614470) or VX12-770-113 (Study 113; NCT01685801); received Ivacaftor 150 mg tablet q12h in this VX12-770-112 (Study 112; NCT01707290) up to 104 weeks.
Receives: Drug: Ivacaftor
ObservationalNo interventionParticipants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet, orally, q12h in the previous Study 110 (NCT01614457) or Study 111 (NCT01614470), were observed (did not receive study drug) in this Study 112 (NCT01707290) for up to 2 years.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm

    AE: any untoward medical occurrence in a participants during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs were defined as adverse events with start date or increased severity on and after the first dose of study drug through Week 108.

    Measured: Day 1 up to Week 108 (Study 112)

Secondary outcomes (6)

  1. Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 6 to 17 years and for female participants aged 6 to 15 years. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.

    Measured: Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)

  2. Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104

    BMI was defined as weight in kg divided by height in m^2. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.

    Measured: Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)

  3. Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104

    Sweat samples were collected using an approved collection device. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.

    Measured: Baseline, Week 2, 24, 48 and 104 (Study 112)

  4. Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104

    The CFQ-R is a validated participant reported outcome measuring health related quality of life for participants with CF. 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. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.

    Measured: Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)

  5. Number of Pulmonary Exacerbations Events

    Pulmonary exacerbation events include those events which require treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. The number of events were reported. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.

    Measured: Through Week 104 (Study 112)

  6. Number of Participants With Serious Adverse Events (SAEs) in Observational Arm

    SAE was defined as a medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event.

    Measured: up to 2 years (Study 112)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Oct 2012 (estimated)
  2. Study startFeb 2013
  3. Primary completionApr 2016
  4. Study completionApr 2016

Resources

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
125 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01707290
Sponsor's protocol ID
VX12-770-112
Other IDs
2012-000389-39
Acronym
KONTINUE
Status last verified
Apr 2017
First submitted
9 Oct 2012
Last updated
12 May 2017
Data monitoring committee
No

Publications

  • Pilewski JM, De Boeck K, Nick JA, Tian S, DeSouza C, Higgins M, Moss RB. Long-Term Ivacaftor in People Aged 6 Years and Older with Cystic Fibrosis with Ivacaftor-Responsive Mutations. Pulm Ther. 2020 Dec;6(2):303-313. doi: 10.1007/s41030-020-00129-2. Epub 2020 Sep 23.

    PubMed 32965659

  • Moss RB, Flume PA, Elborn JS, Cooke J, Rowe SM, McColley SA, Rubenstein RC, Higgins M; VX11-770-110 (KONDUCT) Study Group. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. Lancet Respir Med. 2015 Jul;3(7):524-33. doi: 10.1016/S2213-2600(15)00201-5. Epub 2015 Jun 9.

    PubMed 26070913

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 38 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 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 12 May 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 38 sites in United States, France, United Kingdom, Belgium. 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 12 May 2017). 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 Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm (time frame: Day 1 up to Week 108 (Study 112)). 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 NCT01707290, 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 12 May 2017.

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