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Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation (KONNECTION)

23 października 2014 zaktualizowane przez: Vertex Pharmaceuticals Incorporated

A Phase 3, Two-Part, Randomized, Double-Blind, Placebo-Controlled, Crossover Study With an Open-Label Period to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation

The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following CFTR mutations: G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D).

Przegląd badań

Status

Zakończony

Warunki

Interwencja / Leczenie

Szczegółowy opis

Ivacaftor is the first CFTR modulator to show an improvement in CFTR function and clinical benefit in subjects with CF. Results from Phase 3 studies (VX08-770-102 [Study 102] [NCT00909532] and VX08-770-103 [Study 103] [NCT00909727]) showed that ivacaftor is effective in the treatment of subjects 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 subjects 6 years of age and older who have a G551D mutation in the CFTR gene.

Typ studiów

Interwencyjne

Zapisy (Rzeczywisty)

39

Faza

  • Faza 3

Kontakty i lokalizacje

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

Lokalizacje studiów

  • Belgia
      • Leuven, Belgia
  • Francja
      • Lyon, Francja
      • Montpellier, Francja
      • Paris, Francja
  • Stany Zjednoczone
    • Florida
      • Tampa, Florida, Stany Zjednoczone
    • Georgia
      • Atlanta, Georgia, Stany Zjednoczone
    • Illinois
      • Chicago, Illinois, Stany Zjednoczone
    • Massachusetts
      • Boston, Massachusetts, Stany Zjednoczone
    • Michigan
      • Ann Arbor, Michigan, Stany Zjednoczone
    • Minnesota
      • Minneapolis, Minnesota, Stany Zjednoczone
    • Missouri
      • St. Louis, Missouri, Stany Zjednoczone
    • Texas
      • Houston, Texas, Stany Zjednoczone

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

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

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Wszystko

Opis

Inclusion Criteria:

  • Male or female with confirmed diagnosis of CF
  • At least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D
  • Percent predicted forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) predicted normal for age, sex, and height
  • 6 years of age or older
  • Minimum weight of 15 kilogram (kg) at screening
  • Females of childbearing potential must not be pregnant
  • Willing to comply with contraception requirements

Exclusion Criteria:

  • G551D-CFTR mutation on at least 1 allele
  • History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the subject
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before the first dose of study drug
  • History of solid organ or hematological transplantation
  • History of alcohol, medication or illicit drug abuse within 1 year before the first dose of study drug
  • Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days before screening
  • Use of inhaled hypertonic saline treatment
  • Use of any inhibitors or inducers of cytochrome (CYP) P450 3A
  • Evidence of cataract or lens opacity at screening

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: Zadanie krzyżowe
  • Maskowanie: Poczwórny

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: Part 1: Ivacaftor First, Then Placebo
Ivacaftor 150 milligram (mg) tablet orally twice daily for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
Lek: Ivacaftor
150 mg tablet, oral use, administered twice a day (q12h)
Inne nazwy:
  • VX-770
  • Kalydeco
Lek: Placebo
oral use, administered twice a day (q12h)
Eksperymentalny: Part 1: Placebo First, Then Ivacaftor
Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 1 followed by ivacaftor 150 mg tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
Lek: Ivacaftor
150 mg tablet, oral use, administered twice a day (q12h)
Inne nazwy:
  • VX-770
  • Kalydeco
Lek: Placebo
oral use, administered twice a day (q12h)
Eksperymentalny: Part 2: Ivacaftor
Ivacaftor 150 mg tablet orally twice daily for 16 weeks.
Lek: Ivacaftor
150 mg tablet, oral use, administered twice a day (q12h)
Inne nazwy:
  • VX-770
  • Kalydeco

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Part 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8
Ramy czasowe: Part 1: Baseline (pre-dose Day 1), Week 8
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 subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.
Part 1: Baseline (pre-dose Day 1), Week 8
Part 2: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through 24 Weeks of Treatment (Week 36 Visit)
Ramy czasowe: Baseline (pre-dose Week 12), Week 36
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 subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Absolute change in percent predicted FEV1 over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2, as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.
Baseline (pre-dose Week 12), Week 36

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Part 1: Change From Baseline in Body Mass Index (BMI) at Week 8
Ramy czasowe: Part 1: Baseline (pre-dose Day 1), Week 8
BMI was defined as weight in kilogram (kg) divided by height in meters^2 (m^2). Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.
Part 1: Baseline (pre-dose Day 1), Week 8
Part 2: Change From Baseline in Body Mass Index (BMI) at 24 Weeks of Treatment (Week 36 Visit)
Ramy czasowe: Baseline (pre-dose Week 12), Week 36
BMI was defined as weight in kg divided by height in m^2. Change in BMI over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2 as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.
Baseline (pre-dose Week 12), Week 36
Part 1: Change From Baseline in Sweat Chloride Through Week 8
Ramy czasowe: Part 1: Baseline (pre-dose Day 1), Week 8
Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.
Part 1: Baseline (pre-dose Day 1), Week 8
Part 2: Change From Baseline in Sweat Chloride Through 24 Weeks of Treatment (Week 36 Visit)
Ramy czasowe: Baseline (pre-dose Week 12), Week 36
Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Change in sweat chloride over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2 as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.
Baseline (pre-dose Week 12), Week 36
Part 1: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8
Ramy czasowe: Part 1: Baseline (pre-dose Day 1), Week 8
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects 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. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.
Part 1: Baseline (pre-dose Day 1), Week 8
Part 2: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through 24 Weeks of Treatment (Week 36 Visit)
Ramy czasowe: Baseline (pre-dose Week 12), Week 36
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects 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 in CFQ-R respiratory domain score over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2 as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.
Baseline (pre-dose Week 12), Week 36
Part 1: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Ramy czasowe: Part 1: From signing of informed consent up to Week 20
AE: any adverse change from subject's baseline (pre-treatment) condition, including any adverse experience, abnormal recording/clinical laboratory assessment which occurs during course of study, whether it is considered related to study drug or not. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event.
Part 1: From signing of informed consent up to Week 20
Part 2: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Ramy czasowe: Part 2: Week 20 up to Week 40
AE: any adverse change from subject's baseline (pre-treatment) condition, including any adverse experience, abnormal recording/clinical laboratory assessment which occurs during course of study, whether it is considered related to study drug or not. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event.
Part 2: Week 20 up to Week 40

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Vertex Pharmaceuticals Incorporated

Współpracownicy

Cystic Fibrosis Foundation

Śledczy

  • Główny śledczy: Christine De Boeck, MD, PhD, University of Leuven

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 lipca 2012

Zakończenie podstawowe (Rzeczywisty)

1 października 2013

Ukończenie studiów (Rzeczywisty)

1 października 2013

Daty rejestracji na studia

Pierwszy przesłany

5 czerwca 2012

Pierwszy przesłany, który spełnia kryteria kontroli jakości

7 czerwca 2012

Pierwszy wysłany (Oszacować)

8 czerwca 2012

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Oszacować)

29 października 2014

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

23 października 2014

Ostatnia weryfikacja

1 października 2014

Więcej informacji

Terminy związane z tym badaniem

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • VX12-770-111

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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