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Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation

NCT ID: NCT00909532

Last Updated: 2013-01-18

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

167 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-06-30

Study Completion Date

2012-11-30

Brief Summary

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The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.

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Detailed Description

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This was a phase 3 study in subjects with cystic fibrosis (CF) age 12 years and older who have a G551D-CFTR mutation and percent predicted forced expiratory volumn in 1 second (FEV1) between 40% and 90%.

Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating function of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation.

This study was designed to further evaluate the efficacy of ivacaftor in subjects with CF who have a G551D-CFTR gene mutation and to evaluate safety in this population over a longer period than previously studied.

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Placebo

Subjects who received placebo every 12 hours (q12h) for up to 48 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Tablet given orally q12h for up to 48 weeks

150 mg Ivacaftor q12h

Subjects who received 150 mg of ivacaftor q12h for up to 48 weeks.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

150-mg tablets given orally q12h for up to 48 weeks

Interventions

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Ivacaftor

150-mg tablets given orally q12h for up to 48 weeks

Intervention Type DRUG

Placebo

Tablet given orally q12h for up to 48 weeks

Intervention Type DRUG

Other Intervention Names

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VX-770

Eligibility Criteria

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Inclusion Criteria

* Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
* Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.
* No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
* Willing to use highly effective birth control methods during the study

Exclusion Criteria

* History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
* Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study
* History of alcohol, medication or illicit drug abuse within one year prior to Day 1
* Abnormal liver function ≥ 3x the upper limit of normal
* Abnormal renal function at Screening
* History of solid organ or hematological transplantation
* Pregnant, planning a pregnancy, breast-feeding, or unwilling to follow contraception requirements
* Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening
* Use of inhaled hypertonic saline treatment
* Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)
Minimum Eligible Age

12 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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Bonnie W. Ramsey, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital and Regional Medical Center, Seattle, Washington, USA

Stuart Elborn, MD

Role: PRINCIPAL_INVESTIGATOR

Respiratory Medicine Group, Queen's University of Belfast, Belfast, Northern Ireland, UK

Locations

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University of Alabama

Birmingham, Alabama, United States

Site Status

Kaiser Permanente Medical Care Program

Oakland, California, United States

Site Status

Cystic Fibrosis Research Office, Stanford University

Palo Alto, California, United States

Site Status

Rady Children's Hospital

San Diego, California, United States

Site Status

National Jewish Medical and Research Center

Denver, Colorado, United States

Site Status

Emory Cystic Fibrosis Center

Atlanta, Georgia, United States

Site Status

St. Luke's CF Clinic

Boise, Idaho, United States

Site Status

Children's Memorial Hospital

Chicago, Illinois, United States

Site Status

Indiana University

Indianapolis, Indiana, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Children's Hospital Boston

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Pulmonary, Allergy & Critical Care Medicine, University of Minnesota

Minneapolis, Minnesota, United States

Site Status

The Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

Adult Pulmonary/ CF, University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

Monmouth Medical Center

Long Branch, New Jersey, United States

Site Status

Women and Children's Hospital of Buffalo

Buffalo, New York, United States

Site Status

Long Island Jewish Medical Center

New Hyde Park, New York, United States

Site Status

SUNY Upstate Medical University

Syracuse, New York, United States

Site Status

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Pediatric & Pulmonary Division, Rainbow Babies/Case Western

Cleveland, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Toledo Children's Hospital

Toledo, Ohio, United States

Site Status

Oregon Health & Sciences University

Portland, Oregon, United States

Site Status

Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

East Tennessee Children's Hospital

Knoxville, Tennessee, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

University of Virginia

Charlottesville, Virginia, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Division of Pulmonary and CCM, University of Washington

Seattle, Washington, United States

Site Status

West Virginia University

Morgantown, West Virginia, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

The Children's Hospital Westmead

Westmead, New South Wales, Australia

Site Status

The Prince Charles Hospital

Chermside, Queensland, Australia

Site Status

Royal Children's Hospital Brisbane

Herston, Queensland, Australia

Site Status

Mater Adult Hospital

South Brisbane, Queensland, Australia

Site Status

Royal Children's Hospital Melbourne

Parkville, Victoria, Australia

Site Status

Lung Institute of Western Australia

Nedlands, Western Australia, Australia

Site Status

Princess Margaret Hospital for Children

Subiaco, Western Australia, Australia

Site Status

Queen Elizabeth II Health Sciences Centre

Halifax, Nova Scotia, Canada

Site Status

St. Michael's Hospital

Toronto, Ontario, Canada

Site Status

CF Center, Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Montreal Children's Hospital - MUHC

Montreal, Quebec, Canada

Site Status

FN Motol

Prague, , Czechia

Site Status

Hopital Cochin

Paris, , France

Site Status

Hopital Necker

Paris, , France

Site Status

Centre de Perharidy

Roscoff, , France

Site Status

Kinder- und Jugendklinik Universitätsklinikum Erlangen

Erlangen, , Germany

Site Status

Mukoviszidose-Zentrum am Klinikum der Friedrich-Schiller-Universität Jena, Klinik für Kinder- und Jugendmedizin

Jena, , Germany

Site Status

Klinikum der LMU München, Dr. von Haunersches Kinderspital (CHA)

Munich, , Germany

Site Status

Universitäts-Kinderklinik Würzburg

Würzburg, , Germany

Site Status

Cork University Hospital

Cork, , Ireland

Site Status

Our Lady's Children's Hospital

Dublin, , Ireland

Site Status

The National Children's Hospital

Dublin, , Ireland

Site Status

St. Vincent's University Hospital

Dublin, , Ireland

Site Status

Beaumont Hospital

Dublin, , Ireland

Site Status

Belfast City Hospital

Belfast, Northern Ireland, United Kingdom

Site Status

Imperial College London

London, , United Kingdom

Site Status

Countries

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United States Australia Canada Czechia France Germany Ireland United Kingdom

References

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Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.

Reference Type BACKGROUND
PMID: 22047557 (View on PubMed)

Flume PA, Wainwright CE, Elizabeth Tullis D, Rodriguez S, Niknian M, Higgins M, Davies JC, Wagener JS. Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor. J Cyst Fibros. 2018 Jan;17(1):83-88. doi: 10.1016/j.jcf.2017.06.002. Epub 2017 Jun 24.

Reference Type DERIVED
PMID: 28651844 (View on PubMed)

Solem CT, Vera-Llonch M, Liu S, Botteman M, Castiglione B. Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis. Health Qual Life Outcomes. 2016 Apr 21;14:63. doi: 10.1186/s12955-016-0465-z.

Reference Type DERIVED
PMID: 27097977 (View on PubMed)

Quittner A, Suthoff E, Rendas-Baum R, Bayliss MS, Sermet-Gaudelus I, Castiglione B, Vera-Llonch M. Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial. Health Qual Life Outcomes. 2015 Jul 2;13:93. doi: 10.1186/s12955-015-0293-6.

Reference Type DERIVED
PMID: 26135562 (View on PubMed)

Related Links

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http://ghr.nlm.nih.gov/

Genetics Home Reference

http://www.nlm.nih.gov/medlineplus/

Medline Plus

http://www.clinicaltrials.gov/ct2/info/fdalinks

U.S. FDA Resources

Other Identifiers

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VX08-770-102

Identifier Type: -

Identifier Source: org_study_id

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