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

14. januar 2013 opdateret af: Vertex Pharmaceuticals Incorporated

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects With Cystic Fibrosis and the G551D Mutation

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.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

167

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Australien
    • New South Wales
      • Westmead, New South Wales, Australien, 2145
        • The Children's Hospital Westmead
    • Queensland
      • Chermside, Queensland, Australien, 4032
        • The Prince Charles Hospital
      • Herston, Queensland, Australien, 4026
        • Royal Children's Hospital Brisbane
      • South Brisbane, Queensland, Australien, 4101
        • Mater Adult Hospital
    • Victoria
      • Parkville, Victoria, Australien, 3052
        • Royal Children's Hospital Melbourne
    • Western Australia
      • Nedlands, Western Australia, Australien, 6009
        • Lung Institute of Western Australia
      • Subiaco, Western Australia, Australien, 6008
        • Princess Margaret Hospital for Children
  • Canada
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 3A7
        • Queen Elizabeth Ii Health Sciences Centre
    • Ontario
      • Toronto, Ontario, Canada, M5B 1W8
        • St. Michael's Hospital
      • Toronto, Ontario, Canada, M5G 1X8
        • CF Center, Hospital for Sick Children
    • Quebec
      • Montreal, Quebec, Canada, H3H 1P3
        • Montreal Children's Hospital - MUHC
  • Det Forenede Kongerige
      • London, Det Forenede Kongerige, SW3 6LR
        • Imperial College London
    • Northern Ireland
      • Belfast, Northern Ireland, Det Forenede Kongerige, BT9 7AB
        • Belfast City Hospital
  • Forenede Stater
    • Alabama
      • Birmingham, Alabama, Forenede Stater, 35233-1711
        • University of Alabama
    • California
      • Oakland, California, Forenede Stater, 94611
        • Kaiser Permanente Medical Care Program
      • Palo Alto, California, Forenede Stater, 94304
        • Cystic Fibrosis Research Office, Stanford University
      • San Diego, California, Forenede Stater, 92123-5070
        • Rady Children's Hospital
    • Colorado
      • Denver, Colorado, Forenede Stater, 80206
        • National Jewish Medical and Research Center
    • Georgia
      • Atlanta, Georgia, Forenede Stater, 30322
        • Emory Cystic Fibrosis Center
    • Idaho
      • Boise, Idaho, Forenede Stater, 83712
        • St. Luke's CF Clinic
    • Illinois
      • Chicago, Illinois, Forenede Stater, 60614
        • Children's Memorial Hospital
    • Indiana
      • Indianapolis, Indiana, Forenede Stater, 46202
        • Indiana University
    • Iowa
      • Iowa City, Iowa, Forenede Stater, 52242
        • University of Iowa
    • Maryland
      • Baltimore, Maryland, Forenede Stater, 21205
        • Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, Forenede Stater, 02114
        • Massachusetts General Hospital
      • Boston, Massachusetts, Forenede Stater, 02115
        • Children's Hospital Boston
    • Michigan
      • Ann Arbor, Michigan, Forenede Stater, 48109
        • University of Michigan
    • Minnesota
      • Minneapolis, Minnesota, Forenede Stater, 55455
        • Pulmonary, Allergy & Critical Care Medicine, University of Minnesota
    • Missouri
      • Kansas City, Missouri, Forenede Stater, 64108
        • The Children's Mercy Hospital
      • St. Louis, Missouri, Forenede Stater, 63110
        • Washington University
    • Nebraska
      • Omaha, Nebraska, Forenede Stater, 68198-5300
        • Adult Pulmonary/ CF, University of Nebraska Medical Center
    • New Jersey
      • Long Branch, New Jersey, Forenede Stater, 07740
        • Monmouth Medical Center
    • New York
      • Buffalo, New York, Forenede Stater, 14222
        • Women and Children's Hospital of Buffalo
      • New Hyde Park, New York, Forenede Stater, 11042
        • Long Island Jewish Medical Center
      • Syracuse, New York, Forenede Stater, 13210
        • Suny Upstate Medical University
    • North Carolina
      • Chapel Hill, North Carolina, Forenede Stater, 27599
        • University of North Carolina At Chapel Hill
    • Ohio
      • Cincinnati, Ohio, Forenede Stater, 45229
        • Cincinnati Children's Hospital Medical Center
      • Cleveland, Ohio, Forenede Stater, 44106
        • Pediatric & Pulmonary Division, Rainbow Babies/Case Western
      • Columbus, Ohio, Forenede Stater, 43205
        • Nationwide Children's Hospital
      • Toledo, Ohio, Forenede Stater, 43606
        • Toledo Children's Hospital
    • Oregon
      • Portland, Oregon, Forenede Stater, 97239-3098
        • Oregon Health & Sciences University
    • Pennsylvania
      • Hershey, Pennsylvania, Forenede Stater, 17033
        • Hershey Medical Center
      • Philadelphia, Pennsylvania, Forenede Stater, 19104
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, Forenede Stater, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • Tennessee
      • Knoxville, Tennessee, Forenede Stater, 37916
        • East Tennessee Children's Hospital
      • Nashville, Tennessee, Forenede Stater, 37232-5735
        • Vanderbilt University Medical Center
    • Utah
      • Salt Lake City, Utah, Forenede Stater, 84132
        • University of Utah
    • Virginia
      • Charlottesville, Virginia, Forenede Stater, 22908
        • University of Virginia
    • Washington
      • Seattle, Washington, Forenede Stater, 98105
        • Seattle Children's Hospital
      • Seattle, Washington, Forenede Stater, 98195-6522
        • Division of Pulmonary and CCM, University of Washington
    • West Virginia
      • Morgantown, West Virginia, Forenede Stater, 26506
        • West Virginia University
    • Wisconsin
      • Milwaukee, Wisconsin, Forenede Stater, 53226
        • Medical College of Wisconsin
  • Frankrig
      • Paris, Frankrig, 75014
        • Hôpital Cochin
      • Paris, Frankrig, 75015
        • Hôpital Necker
      • Roscoff, Frankrig, 29684
        • Centre de Perharidy
  • Irland
      • Cork, Irland
        • Cork University Hospital
      • Dublin, Irland, 9
        • Beaumont Hospital
      • Dublin, Irland, 4
        • St. Vincent's University Hospital
      • Dublin, Irland, 12
        • Our Lady's Children's Hospital
      • Dublin, Irland, 24
        • The National Children's Hospital
  • Tjekkiet
      • Prague, Tjekkiet, 15006
        • FN Motol
  • Tyskland
      • Erlangen, Tyskland, 91054
        • Kinder- und Jugendklinik Universitätsklinikum Erlangen
      • Jena, Tyskland, 07740
        • Mukoviszidose-Zentrum am Klinikum der Friedrich-Schiller-Universität Jena, Klinik für Kinder- und Jugendmedizin
      • Munich, Tyskland, 80337
        • Klinikum der LMU München, Dr. von Haunersches Kinderspital (CHA)
      • Wurzburg, Tyskland, 97080
        • Universitäts-Kinderklinik Würzburg

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

12 år og ældre (Barn, Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

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)

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Firedobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Placebo komparator: Placebo
Forsøgspersoner, der fik placebo hver 12. time (q12h) i op til 48 uger.
Medicin: Placebo
Tablet givet oralt hver 12. time i op til 48 uger
Eksperimentel: 150 mg Ivacaftor q12h
Forsøgspersoner, der fik 150 mg ivacaftor hver 12. time i op til 48 uger.
Medicin: Ivacaftor
150-mg tablets given orally q12h for up to 48 weeks
Andre navne:
  • VX-770

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24
Tidsramme: baseline through 24 weeks
Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
baseline through 24 weeks

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Absolut ændring fra baseline i svedkloridkoncentration gennem uge 24 og uge 48
Tidsramme: baseline gennem 24 uger og 48 uger
Svedklorid-testen (kvantitativ pilocarpiniontoforese) er et standard diagnostisk værktøj til cystisk fibrose (CF), der tjener som en indikator for cystisk fibrose transmembrankonduktansregulator (CFTR) aktivitet.
baseline gennem 24 uger og 48 uger
Absolut ændring fra baseline i vægt i uge 24 og uge 48
Tidsramme: baseline til 24 uger og 48 uger
Da underernæring er almindelig hos patienter med cystisk fibrose (CF) på grund af øget energiforbrug på grund af lungesygdom og fedtmalabsorption, er kropsvægt et vigtigt klinisk mål for ernæringsstatus.
baseline til 24 uger og 48 uger
Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48
Tidsramme: baseline through 48 weeks
Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
baseline through 48 weeks
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)
Tidsramme: baseline through 24 weeks and 48 weeks
The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID).
baseline through 24 weeks and 48 weeks
Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48
Tidsramme: baseline through 24 weeks and 48 weeks
Pulmonary exacerbation was defined as a change in antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of signs/symptoms such as change in sputum; new or increased hemoptysis; increased cough or dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees C; anorexia or weight loss; sinus pain/tenderness and discharge; change in physical examination of the chest; decreased pulmonary function by 10%; and radiographic changes indicative of pulmonary infection.
baseline through 24 weeks and 48 weeks

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Vertex Pharmaceuticals Incorporated

Samarbejdspartnere

Cystic Fibrosis Foundation

Efterforskere

  • Ledende efterforsker: Bonnie W. Ramsey, MD, Children's Hospital and Regional Medical Center, Seattle, Washington, USA
  • Ledende efterforsker: Stuart Elborn, MD, Respiratory Medicine Group, Queen's University of Belfast, Belfast, Northern Ireland, UK

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Generelle publikationer

Hjælpsomme links

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart

1. juni 2009

Primær færdiggørelse (Faktiske)

1. juli 2010

Studieafslutning (Faktiske)

1. november 2012

Datoer for studieregistrering

Først indsendt

26. maj 2009

Først indsendt, der opfyldte QC-kriterier

26. maj 2009

Først opslået (Skøn)

28. maj 2009

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Skøn)

18. januar 2013

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

14. januar 2013

Sidst verificeret

1. januar 2013

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • VX08-770-102

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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