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

NCT ID: NCT01707290

Last Updated: 2017-05-12

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

125 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-02-28

Study Completion Date

2016-04-30

Brief Summary

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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).

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Detailed Description

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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

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Cystic Fibrosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Ivacaftor

Participants 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.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

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

Observational

Participants 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.

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Ivacaftor

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

Intervention Type DRUG

Other Intervention Names

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Kalydeco VX-770

Eligibility Criteria

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Inclusion Criteria

* 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 of childbearing potential entering the ivacaftor arm must not be pregnant.
* Participants entering the ivacaftor arm must be willing to comply with contraception requirements.

Exclusion Criteria

* 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.
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Cystic Fibrosis Foundation

OTHER

Sponsor Role collaborator

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Joseph Pilewski, MD

Role: PRINCIPAL_INVESTIGATOR

University of Pittsburgh

Locations

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Birmingham, Alabama, United States

Site Status

Palo Alto, California, United States

Site Status

Denver, Colorado, United States

Site Status

Hartford, Connecticut, United States

Site Status

Tampa, Florida, United States

Site Status

Atlanta, Georgia, United States

Site Status

Chicago, Illinois, United States

Site Status

Iowa City, Iowa, United States

Site Status

Lexington, Kentucky, United States

Site Status

Baltimore, Maryland, United States

Site Status

Boston, Massachusetts, United States

Site Status

Ann Arbor, Michigan, United States

Site Status

Detroit, Michigan, United States

Site Status

Grand Rapids, Michigan, United States

Site Status

Minneapolis, Minnesota, United States

Site Status

St Louis, Missouri, United States

Site Status

Omaha, Nebraska, United States

Site Status

New York, New York, United States

Site Status

Syracuse, New York, United States

Site Status

Valhalla, New York, United States

Site Status

Chapel Hill, North Carolina, United States

Site Status

Philadelphia, Pennsylvania, United States

Site Status

Pittsburgh, Pennsylvania, United States

Site Status

Charleston, South Carolina, United States

Site Status

Nashville, Tennessee, United States

Site Status

Dallas, Texas, United States

Site Status

Fort Worth, Texas, United States

Site Status

Houston, Texas, United States

Site Status

Salt Lake City, Utah, United States

Site Status

Richmond, Virginia, United States

Site Status

Seattle, Washington, United States

Site Status

Morgantown, West Virginia, United States

Site Status

Madison, Wisconsin, United States

Site Status

Leuven, , Belgium

Site Status

Montpellier, , France

Site Status

Paris, , France

Site Status

Belfast, , United Kingdom

Site Status

Edinburgh, , United Kingdom

Site Status

Countries

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United States Belgium France United Kingdom

References

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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.

Reference Type DERIVED
PMID: 32965659 (View on PubMed)

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.

Reference Type DERIVED
PMID: 26070913 (View on PubMed)

Other Identifiers

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2012-000389-39

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX12-770-112

Identifier Type: -

Identifier Source: org_study_id

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