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- Registro degli studi clinici negli Stati Uniti
- Sperimentazione clinica NCT02875366
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 marzo 2019 aggiornato da: 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.
Panoramica dello studio
Tipo di studio
Interventistico
Iscrizione (Effettivo)
70
Fase
- Fase 4
Contatti e Sedi
Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.
Luoghi di studio
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Adelaide, Australia
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Camperdown, Australia
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Clayton, Australia
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Nedlands, Australia
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New Lambton Heights, Australia
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Randwick, Australia
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South Brisbane, Australia
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Subiaco, Australia
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Westmead, Australia
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Victoria
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Melbourne, Victoria, Australia
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Parkville, Victoria, Australia
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Edinburgh, Regno Unito
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Criteri di partecipazione
I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.
Criteri di ammissibilità
Età idonea allo studio
12 anni e precedenti (Bambino, Adulto, Adulto più anziano)
Accetta volontari sani
No
Sessi ammissibili allo studio
Tutto
Descrizione
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
Piano di studio
Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Altro
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Triplicare
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
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Comparatore placebo: Placebo
Placebo matched to LUM/IVA fixed-dose combination tablet orally every 12 hours (q12h) for 24 weeks.
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Sperimentale: LUM/IVA
LUM 400 milligram (mg)/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
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Altri nomi:
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
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Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24
Lasso di tempo: Baseline, Week 24
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CPET was used to assess change in exercise tolerance, as measured by VO2max.
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Baseline, Week 24
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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
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Relative (Percent) Change From Baseline in Exercise Duration During CPET at Week 24
Lasso di tempo: Baseline, Week 24
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Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
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Baseline, Week 24
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Absolute Change From Baseline in Exercise Duration During CPET at Week 24
Lasso di tempo: Baseline, Week 24
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Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
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Baseline, Week 24
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Absolute Change From Baseline in VO2max During CPET at Week 24
Lasso di tempo: Baseline, Week 24
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CPET was used to assess change in exercise tolerance, as measured by VO2max.
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Baseline, Week 24
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Absolute Change From Baseline in Oxygen Consumption (VO2) at Anaerobic Threshold at Week 24
Lasso di tempo: Baseline, Week 24
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Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in VO2 at Anaerobic Threshold at Week 24
Lasso di tempo: Baseline, Week 24
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Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
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Baseline, Week 24
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Absolute Change From Baseline in Functional VO2 Gain at Week 24
Lasso di tempo: Baseline, Week 24
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Functional VO2 Gain at Week 24
Lasso di tempo: Baseline, Week 24
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Baseline, Week 24
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Absolute Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
Lasso di tempo: Baseline, Week 24
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
Lasso di tempo: Baseline, Week 24
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Baseline, Week 24
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Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
Lasso di tempo: Baseline, Week 24
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
Lasso di tempo: Baseline, Week 24
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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Baseline, Week 24
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Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
Lasso di tempo: Baseline, Week 24
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BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2).
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Baseline, Week 24
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Relative (Percent) Change From Baseline in BMI at Week 24
Lasso di tempo: Baseline, Week 24
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BMI was defined as weight in kg divided by height in m^2.
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Baseline, Week 24
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Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24
Lasso di tempo: Baseline, Week 24
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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.
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Baseline, Week 24
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Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Lasso di tempo: Baseline, Week 24
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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.
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Baseline, Week 24
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Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Lasso di tempo: Baseline, Week 24
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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.
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Baseline, Week 24
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Absolute Change From Baseline in Daily Physical Activity Counts as Determined by Actigraphy at Week 24
Lasso di tempo: Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activity counts.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Physical Activity as Determined by Actigraphy at Week 24
Lasso di tempo: Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activities.
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Baseline, Week 24
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Absolute Change From Baseline in Duration of Sleep Time at Week 24
Lasso di tempo: Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Duration of Sleep Time at Week 24
Lasso di tempo: Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
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Baseline, Week 24
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Absolute Change From Baseline in Time Above Sedentary Duration at Week 24
Lasso di tempo: Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Time Above Sedentary Duration at Week 24
Lasso di tempo: Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
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Baseline, Week 24
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Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Lasso di tempo: Day 1 up to Week 28
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Day 1 up to Week 28
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Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Pubblicazioni e link utili
La persona responsabile dell'inserimento delle informazioni sullo studio fornisce volontariamente queste pubblicazioni. Questi possono riguardare qualsiasi cosa relativa allo studio.
Studiare le date dei record
Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.
Studia le date principali
Inizio studio (Effettivo)
1 settembre 2016
Completamento primario (Effettivo)
1 settembre 2017
Completamento dello studio (Effettivo)
1 ottobre 2017
Date di iscrizione allo studio
Primo inviato
15 agosto 2016
Primo inviato che soddisfa i criteri di controllo qualità
22 agosto 2016
Primo Inserito (Stima)
23 agosto 2016
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
17 giugno 2019
Ultimo aggiornamento inviato che soddisfa i criteri QC
18 marzo 2019
Ultimo verificato
1 marzo 2019
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
- Malattie dell'apparato digerente
- Processi patologici
- Malattie delle vie respiratorie
- Malattie polmonari
- Infante, neonato, malattie
- Malattie genetiche, congenite
- Malattie pancreatiche
- Fibrosi
- Fibrosi cistica
- Meccanismi molecolari dell'azione farmacologica
- Modulatori di trasporto a membrana
- Agonisti del canale del cloruro
- Ivacaftor
Altri numeri di identificazione dello studio
- VX15-809-112
- 2016-000066-34 (Numero EudraCT)
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .
Prove cliniche su Fibrosi cistica
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National Institute of Allergy and Infectious Diseases...Completato