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A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation

18 maart 2019 bijgewerkt door: Vertex Pharmaceuticals Incorporated

A Phase 4, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Study of the Effect of Lumacaftor/Ivacaftor Combination Therapy on Exercise Tolerance in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

This is a Phase 4, randomized, double-blind, placebo-controlled, parallel-group study in subjects aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation. This study is designed to evaluate the effect of LUM/IVA on exercise tolerance in subjects with CF, homozygous for the F508del-CFTR mutation.

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

70

Fase

  • Fase 4

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

  • Australië
      • Adelaide, Australië
      • Camperdown, Australië
      • Clayton, Australië
      • Nedlands, Australië
      • New Lambton Heights, Australië
      • Randwick, Australië
      • South Brisbane, Australië
      • Subiaco, Australië
      • Westmead, Australië
    • Victoria
      • Melbourne, Victoria, Australië
      • Parkville, Victoria, Australië
  • Verenigd Koninkrijk
      • Edinburgh, Verenigd Koninkrijk

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

12 jaar en ouder (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Inclusion Criteria:

  • Homozygous for the F508del-CFTR mutation
  • Confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis
  • Stable CF disease as judged by the investigator
  • Forced expiratory volume in 1 second (FEV1) at least 40% and not greater than 90% of predicted

Exclusion Criteria:

  • History of any comorbidity that might confound the results of the study, interfere with the use of cardiopulmonary exercise tests (CPETs) as an assessment, or pose an additional risk in administering study drug to the subject
  • Any previous exposure to LUM or IVA
  • History of cardiac arrhythmia, ischemic heart disease, congestive heart failure, or other clinically significant cardiac condition, or medical condition requiring chronic use of a beta blocker, non-dihydropyridine calcium channel blocker, or other cardiac medication known to affect exercise tolerance
  • History of solid organ or hematological transplantation
  • For subjects under 18 years of age at Screening, except those who have had bilateral lens removal, selected findings on a screening ophthalmologic examination will be exclusionary
  • Using or expected to require any concomitant medication that is prohibited in this study
  • History of alcohol or drug abuse, as deemed by the investigator, in the past year, including but not limited to cannabis, cocaine, and opiates
  • Participation in an investigational drug study within 30 days before the Screening Visit
  • Pregnant or nursing females; males with a female partner who is pregnant or nursing
  • Colonization with organisms associated with a more rapid decline in pulmonary status

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Ander
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Verdrievoudigen

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Placebo-vergelijker: Placebo
Placebo matched to LUM/IVA fixed-dose combination tablet orally every 12 hours (q12h) for 24 weeks.
Geneesmiddel: Placebo
Experimenteel: LUM/IVA
LUM 400 milligram (mg)/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
Geneesmiddel: LUM/IVA
Andere namen:
  • Lumacaftor/Ivacaftor

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24
Tijdsspanne: Baseline, Week 24
CPET was used to assess change in exercise tolerance, as measured by VO2max.
Baseline, Week 24

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Relative (Percent) Change From Baseline in Exercise Duration During CPET at Week 24
Tijdsspanne: Baseline, Week 24
Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
Baseline, Week 24
Absolute Change From Baseline in Exercise Duration During CPET at Week 24
Tijdsspanne: Baseline, Week 24
Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
Baseline, Week 24
Absolute Change From Baseline in VO2max During CPET at Week 24
Tijdsspanne: Baseline, Week 24
CPET was used to assess change in exercise tolerance, as measured by VO2max.
Baseline, Week 24
Absolute Change From Baseline in Oxygen Consumption (VO2) at Anaerobic Threshold at Week 24
Tijdsspanne: Baseline, Week 24
Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
Baseline, Week 24
Relative (Percent) Change From Baseline in VO2 at Anaerobic Threshold at Week 24
Tijdsspanne: Baseline, Week 24
Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
Baseline, Week 24
Absolute Change From Baseline in Functional VO2 Gain at Week 24
Tijdsspanne: Baseline, Week 24
Baseline, Week 24
Relative (Percent) Change From Baseline in Functional VO2 Gain at Week 24
Tijdsspanne: Baseline, Week 24
Baseline, Week 24
Absolute Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
Tijdsspanne: Baseline, Week 24
Baseline, Week 24
Relative (Percent) Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
Tijdsspanne: Baseline, Week 24
Baseline, Week 24
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
Tijdsspanne: Baseline, Week 24
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline, Week 24
Relative (Percent) Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
Tijdsspanne: Baseline, Week 24
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline, Week 24
Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
Tijdsspanne: Baseline, Week 24
BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2).
Baseline, Week 24
Relative (Percent) Change From Baseline in BMI at Week 24
Tijdsspanne: Baseline, Week 24
BMI was defined as weight in kg divided by height in m^2.
Baseline, Week 24
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24
Tijdsspanne: Baseline, Week 24
The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life.
Baseline, Week 24
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Tijdsspanne: Baseline, Week 24
The PHQ-8 is an eight item self-reported measure of depression. Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day). Total score is the sum of individual eight items and ranges from 0 to 24, with higher scores indicating more severe depression symptoms. Total score of 0 to 5 indicates none to minimal depression, 6 to 10 indicates mild depression, 11 to 15 indicates moderate depression, 16 to 20 indicates moderately severe depression and 21 to 24 indicates severe depression.
Baseline, Week 24
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Tijdsspanne: Baseline, Week 24
The GAD-7 is a seven item, self-reported measurement of GAD severity. Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day). Total score is the sum of individual seven items and ranges from 0 to 21, with higher scores indicating more severe anxiety symptoms. Total score of 0 to 5 indicates none to minimal anxiety, 6 to 10 indicates mild anxiety, 11 to 15 indicates moderate anxiety, 16 to 21 indicates severe anxiety.
Baseline, Week 24
Absolute Change From Baseline in Daily Physical Activity Counts as Determined by Actigraphy at Week 24
Tijdsspanne: Baseline, Week 24
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activity counts.
Baseline, Week 24
Relative (Percent) Change From Baseline in Physical Activity as Determined by Actigraphy at Week 24
Tijdsspanne: Baseline, Week 24
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activities.
Baseline, Week 24
Absolute Change From Baseline in Duration of Sleep Time at Week 24
Tijdsspanne: Baseline, Week 24
Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
Baseline, Week 24
Relative (Percent) Change From Baseline in Duration of Sleep Time at Week 24
Tijdsspanne: Baseline, Week 24
Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
Baseline, Week 24
Absolute Change From Baseline in Time Above Sedentary Duration at Week 24
Tijdsspanne: Baseline, Week 24
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
Baseline, Week 24
Relative (Percent) Change From Baseline in Time Above Sedentary Duration at Week 24
Tijdsspanne: Baseline, Week 24
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
Baseline, Week 24
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Tijdsspanne: Day 1 up to Week 28
Day 1 up to Week 28

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Vertex Pharmaceuticals Incorporated

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

1 september 2016

Primaire voltooiing (Werkelijk)

1 september 2017

Studie voltooiing (Werkelijk)

1 oktober 2017

Studieregistratiedata

Eerst ingediend

15 augustus 2016

Eerst ingediend dat voldeed aan de QC-criteria

22 augustus 2016

Eerst geplaatst (Schatting)

23 augustus 2016

Updates van studierecords

Laatste update geplaatst (Werkelijk)

17 juni 2019

Laatste update ingediend die voldeed aan QC-criteria

18 maart 2019

Laatst geverifieerd

1 maart 2019

Meer informatie

Termen gerelateerd aan deze studie

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • VX15-809-112
  • 2016-000066-34 (EudraCT-nummer)

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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