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Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation (DISCOVER)

27 sierpnia 2015 zaktualizowane przez: Vertex Pharmaceuticals Incorporated

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of VX-770 in Subjects Aged 12 Years and Older With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

The purpose of this study was to evaluate the safety and efficacy of ivacaftor in participants with cystic fibrosis (CF) who were aged 12 years or older and were homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation. 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 adenosine monophosphate (AMP)-dependent protein kinase A (PKA) activation.

Przegląd badań

Status

Zakończony

Warunki

Interwencja / Leczenie

Szczegółowy opis

This study investigated the effects of ivacaftor in participants with cystic fibrosis (CF) >=12 years of age with a forced expiratory volume in 1 second (FEV1) >=40 percent (%) predicted. This study was conducted in 2 parts.

  • Part A of this study was a randomized, double-blind, placebo-controlled, parallel-group evaluation of participants with CF who were aged 12 years or older and were homozygous for the F508del-CFTR mutation.
  • Part B of this study was an open-label extension of Part A, enrolling participants who completed Part A and met pre-specified endpoint criteria, and explored the safety and efficacy of ivacaftor over long-term treatment in participants with CF aged 12 years or older who were homozygous for the F508del-CFTR mutation.

Typ studiów

Interwencyjne

Zapisy (Rzeczywisty)

140

Faza

  • Faza 2

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Lokalizacje studiów

  • Stany Zjednoczone
    • Alabama
      • Birmingham, Alabama, Stany Zjednoczone, 35294
        • University of Alabama
    • Alaska
      • Anchorage, Alaska, Stany Zjednoczone, 99508
        • Providence Medical Center
    • California
      • Oakland, California, Stany Zjednoczone, 94611
        • Kaiser Permanente Medical Care Program
    • Connecticut
      • Hartford, Connecticut, Stany Zjednoczone, 06106
        • Connecticut Children's Medical Center
    • Florida
      • Miami, Florida, Stany Zjednoczone, 33136
        • University of Miami Miller School of Medicine
      • Orlando, Florida, Stany Zjednoczone, 32801
        • Nemours Children's Clinic
    • Idaho
      • Boise, Idaho, Stany Zjednoczone, 83712
        • St. Luke's CF Clinic
    • Illinois
      • Chicago, Illinois, Stany Zjednoczone, 60637
        • University of Chicago
    • Indiana
      • Indianapolis, Indiana, Stany Zjednoczone, 46202
        • Riley Hospital for Children
    • Maine
      • Portland, Maine, Stany Zjednoczone, 04102
        • Maine Medical Center
    • Massachusetts
      • Boston, Massachusetts, Stany Zjednoczone, 02114
        • Massachusetts General Hospital
      • Worcester, Massachusetts, Stany Zjednoczone, 01655
        • University of Massachussetts Medical School
    • Michigan
      • Grand Rapids, Michigan, Stany Zjednoczone, 49503
        • Helen DeVos Children's Hospital; Spectrum Health Hospitals
    • Missouri
      • Kansas City, Missouri, Stany Zjednoczone, 64108
        • The Children's Mercy Hospital
    • New Hampshire
      • Lebanon, New Hampshire, Stany Zjednoczone, 03756
        • Dartmouth-Hitchcock Medical Center
    • New Jersey
      • Long Branch, New Jersey, Stany Zjednoczone, 07740
        • Monmouth Medical Center
      • Morristown, New Jersey, Stany Zjednoczone, 07962
        • Morristown Memorial Hospital
    • New York
      • Albany, New York, Stany Zjednoczone, 12208
        • Albany Medical College
      • Buffalo, New York, Stany Zjednoczone, 14222
        • Women and Children's Hospital of Buffalo
      • Hawthorne, New York, Stany Zjednoczone, 10532
        • New York Medical College
      • New York, New York, Stany Zjednoczone, 10032
        • Columbia University Medical Center
      • New York City, New York, Stany Zjednoczone, 10003
        • The CF Center, Beth Israel Medical Center
    • Ohio
      • Akron, Ohio, Stany Zjednoczone, 44308
        • Akron Children's Hospital
      • Cincinnati, Ohio, Stany Zjednoczone, 45229
        • Cincinnati Children's Hospital
      • Toledo, Ohio, Stany Zjednoczone, 43606
        • Toldedo Children's Hospital
    • Oklahoma
      • Oklahoma City, Oklahoma, Stany Zjednoczone, 73104
        • University of Oklahoma Health Sciences Center
    • Pennsylvania
      • Hershey, Pennsylvania, Stany Zjednoczone, 17033
        • Hershey Medical Center
      • Philadelphia, Pennsylvania, Stany Zjednoczone, 19134
        • St. Christopher's Hospital for Children
    • South Carolina
      • Charleston, South Carolina, Stany Zjednoczone, 29425
        • Medical University of South Carolina
    • Tennessee
      • Memphis, Tennessee, Stany Zjednoczone, 38103
        • University of Tennessee
    • Texas
      • Fort Worth, Texas, Stany Zjednoczone, 76104
        • Cook Children's Medical Center
    • Utah
      • Salt Lake City, Utah, Stany Zjednoczone, 84132
        • Univeristy of Utah
    • Vermont
      • Colchester, Vermont, Stany Zjednoczone, 05446
        • Vermont Lung Center at The University of Vermont
    • Virginia
      • Richmond, Virginia, Stany Zjednoczone, 23298
        • Medical College of Virginia

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

12 lat i starsze (Dziecko, Dorosły, Starszy dorosły)

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Wszystko

Opis

Inclusion Criteria:

  • Confirmed diagnosis of cystic fibrosis (CF) and homozygous for F508del-CFTR mutation
  • Forced expiratory volume in 1 second (FEV1) of at least 40% of predicted normal for age, gender, and height
  • Willing to use at least 2 highly effective birth control methods during the study
  • No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
  • Able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator

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 >=3 x the upper limit of normal
  • Abnormal renal function at Screening
  • History of solid organ or hematological transplantation
  • Pregnant or breast-feeding (for women)
  • Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to screening
  • Previous participation in a VX-809 study
  • Used inhaled hypertonic saline treatment
  • Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP3A4)

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Randomizowane
  • Model interwencyjny: Przydział równoległy
  • Maskowanie: Poczwórny

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Komparator placebo: Placebo
Placebo matched to ivacaftor tablet orally every 12 hours (q12h) for 16 weeks during Part A (double-blind treatment period), followed by ivacaftor 150 mg tablet orally q12h for 96 weeks during Part B (open-label extension period).
Lek: Placebo
Tablet
Lek: Ivacaftor
Tablet
Inne nazwy:
  • VX-770
Eksperymentalny: Ivacaftor
Ivacaftor 150 milligram (mg) tablet orally q12h for 16 weeks during Part A (double-blind treatment period), followed by ivacaftor 150 mg tablet orally q12h for 96 weeks during Part B (open-label extension period).
Lek: Ivacaftor
Tablet
Inne nazwy:
  • VX-770

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Part A : Absolute Change From Part A Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 16
Ramy czasowe: Part A baseline through Week 16
Spirometry (as measured by ppFEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. ppFEV1 (predicted for age, gender, and height) was calculated using the Knudson method.
Part A baseline through Week 16

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Part A : Absolute Change From Part A Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 16
Ramy czasowe: Part A baseline through Week 16
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for participants 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.
Part A baseline through Week 16
Part A : Absolute Change From Part A Baseline in Sweat Chloride Concentration Through Week 16
Ramy czasowe: Part A baseline through Week 16
The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.
Part A baseline through Week 16
Part A : Rate of Change From Baseline in Weight Through Week 16
Ramy czasowe: Part A baseline through Week 16
As malnutrition is common in participants with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status.
Part A baseline through Week 16
Part B : Absolute Change From Part A and Part B Baseline in ppFEV1 Through Week 64
Ramy czasowe: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
ppFEV1 is defined in Outcome Measure 1.
Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
Part B : Rate of Change From Part A Baseline in ppFEV1 Through Week 64
Ramy czasowe: Part A baseline through Week 64
ppFEV1 is defined in Outcome Measure 1.
Part A baseline through Week 64
Part B : Rate of Change From Part B Baseline in ppFEV1 Through Week 64
Ramy czasowe: Part B baseline through Week 64
ppFEV1 is defined in Outcome Measure 1.
Part B baseline through Week 64
Part B : Absolute Change From Part A and Part B Baseline in CFQ-R Respiratory Domain Score Through Week 64
Ramy czasowe: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for participants 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.
Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
Part B : Absolute Change From Part A and Part B Baseline in Sweat Chloride Concentration Through Week 64
Ramy czasowe: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.
Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
Part B : Absolute Change From Part A and Part B Baseline in Weight Through Week 64
Ramy czasowe: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status.
Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
Part B : Number of Participants With Pulmonary Exacerbations
Ramy czasowe: Part B baseline through Week 64
Pulmonary exacerbation was defined as new, or changed, antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of the following signs/symptoms: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees Celsius; anorexia or weight loss; sinus pain or tenderness; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent (%); and radiographic changes indicative of pulmonary infection.
Part B baseline through Week 64
Part B : Number of Pulmonary Exacerbation Events
Ramy czasowe: Part B baseline through Week 64
Pulmonary exacerbation was defined as new, or changed, antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of the following signs/symptoms: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees Celsius; anorexia or weight loss; sinus pain or tenderness; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent (%); and radiographic changes indicative of pulmonary infection.
Part B baseline through Week 64
Part B : Number of Pulmonary Exacerbation Events Per Participant Per Year
Ramy czasowe: Part B baseline through Week 64
Pulmonary exacerbation was defined as new, or changed, antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of the following signs/symptoms: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees Celsius; anorexia or weight loss; sinus pain or tenderness; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent (%); and radiographic changes indicative of pulmonary infection.
Part B baseline through Week 64

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Vertex Pharmaceuticals Incorporated

Współpracownicy

Cystic Fibrosis Foundation

Śledczy

  • Główny śledczy: Patrick A Flume, MD, Medical University of South Carolina

Publikacje i pomocne linki

Osoba odpowiedzialna za wprowadzenie informacji o badaniu dobrowolnie udostępnia te publikacje. Mogą one dotyczyć wszystkiego, co jest związane z badaniem.

Publikacje ogólne

Przydatne linki

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów

1 września 2009

Zakończenie podstawowe (Rzeczywisty)

1 lipca 2010

Ukończenie studiów (Rzeczywisty)

1 maja 2013

Daty rejestracji na studia

Pierwszy przesłany

4 sierpnia 2009

Pierwszy przesłany, który spełnia kryteria kontroli jakości

5 sierpnia 2009

Pierwszy wysłany (Oszacować)

6 sierpnia 2009

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Oszacować)

11 września 2015

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

27 sierpnia 2015

Ostatnia weryfikacja

1 sierpnia 2015

Więcej informacji

Terminy związane z tym badaniem

Słowa kluczowe

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • VX08-770-104
  • 2009-010261-23 (Numer EudraCT)

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

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Badania kliniczne na Placebo

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