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- Registro degli studi clinici negli Stati Uniti
- Sperimentazione clinica NCT00953706
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation (DISCOVER)
27 agosto 2015 aggiornato da: 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.
Panoramica dello studio
Descrizione dettagliata
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.
Tipo di studio
Interventistico
Iscrizione (Effettivo)
140
Fase
- Fase 2
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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Alabama
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Birmingham, Alabama, Stati Uniti, 35294
- University of Alabama
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Alaska
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Anchorage, Alaska, Stati Uniti, 99508
- Providence Medical Center
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California
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Oakland, California, Stati Uniti, 94611
- Kaiser Permanente Medical Care Program
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Connecticut
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Hartford, Connecticut, Stati Uniti, 06106
- Connecticut Children's Medical Center
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Florida
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Miami, Florida, Stati Uniti, 33136
- University of Miami Miller School of Medicine
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Orlando, Florida, Stati Uniti, 32801
- Nemours Children's Clinic
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Idaho
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Boise, Idaho, Stati Uniti, 83712
- St. Luke's CF Clinic
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Illinois
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Chicago, Illinois, Stati Uniti, 60637
- University of Chicago
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Indiana
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Indianapolis, Indiana, Stati Uniti, 46202
- Riley Hospital For Children
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Maine
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Portland, Maine, Stati Uniti, 04102
- Maine Medical Center
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Massachusetts
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Boston, Massachusetts, Stati Uniti, 02114
- Massachusetts General Hospital
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Worcester, Massachusetts, Stati Uniti, 01655
- University of Massachussetts Medical School
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Michigan
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Grand Rapids, Michigan, Stati Uniti, 49503
- Helen DeVos Children's Hospital; Spectrum Health Hospitals
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Missouri
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Kansas City, Missouri, Stati Uniti, 64108
- The Children's Mercy Hospital
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New Hampshire
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Lebanon, New Hampshire, Stati Uniti, 03756
- Dartmouth-Hitchcock Medical Center
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New Jersey
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Long Branch, New Jersey, Stati Uniti, 07740
- Monmouth Medical Center
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Morristown, New Jersey, Stati Uniti, 07962
- Morristown Memorial Hospital
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New York
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Albany, New York, Stati Uniti, 12208
- Albany Medical College
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Buffalo, New York, Stati Uniti, 14222
- Women and Children's Hospital of Buffalo
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Hawthorne, New York, Stati Uniti, 10532
- New York Medical College
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New York, New York, Stati Uniti, 10032
- Columbia University Medical Center
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New York City, New York, Stati Uniti, 10003
- The CF Center, Beth Israel Medical Center
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Ohio
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Akron, Ohio, Stati Uniti, 44308
- Akron Children's Hospital
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Cincinnati, Ohio, Stati Uniti, 45229
- Cincinnati Children's Hospital
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Toledo, Ohio, Stati Uniti, 43606
- Toldedo Children's Hospital
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Oklahoma
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Oklahoma City, Oklahoma, Stati Uniti, 73104
- University of Oklahoma Health Sciences Center
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Pennsylvania
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Hershey, Pennsylvania, Stati Uniti, 17033
- Hershey Medical Center
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Philadelphia, Pennsylvania, Stati Uniti, 19134
- St. Christopher's Hospital for Children
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South Carolina
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Charleston, South Carolina, Stati Uniti, 29425
- Medical University of South Carolina
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Tennessee
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Memphis, Tennessee, Stati Uniti, 38103
- University of Tennessee
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Texas
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Fort Worth, Texas, Stati Uniti, 76104
- Cook Children's Medical Center
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Utah
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Salt Lake City, Utah, Stati Uniti, 84132
- Univeristy of Utah
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Vermont
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Colchester, Vermont, Stati Uniti, 05446
- Vermont Lung Center at The University of Vermont
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Virginia
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Richmond, Virginia, Stati Uniti, 23298
- Medical College of Virginia
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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:
- 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)
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: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Quadruplicare
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
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Comparatore 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).
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Tavoletta
Tablet
Altri nomi:
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Sperimentale: 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).
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Tablet
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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Part A : Absolute Change From Part A Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 16
Lasso di tempo: Part A baseline through Week 16
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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.
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Part A baseline through Week 16
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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
---|---|---|
Part A : Absolute Change From Part A Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 16
Lasso di tempo: Part A baseline through Week 16
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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.
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Part A baseline through Week 16
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Part A : Absolute Change From Part A Baseline in Sweat Chloride Concentration Through Week 16
Lasso di tempo: Part A baseline through Week 16
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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.
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Part A baseline through Week 16
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Part A : Rate of Change From Baseline in Weight Through Week 16
Lasso di tempo: Part A baseline through Week 16
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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.
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Part A baseline through Week 16
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Part B : Absolute Change From Part A and Part B Baseline in ppFEV1 Through Week 64
Lasso di tempo: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
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ppFEV1 is defined in Outcome Measure 1.
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Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
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Part B : Rate of Change From Part A Baseline in ppFEV1 Through Week 64
Lasso di tempo: Part A baseline through Week 64
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ppFEV1 is defined in Outcome Measure 1.
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Part A baseline through Week 64
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Part B : Rate of Change From Part B Baseline in ppFEV1 Through Week 64
Lasso di tempo: Part B baseline through Week 64
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ppFEV1 is defined in Outcome Measure 1.
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Part B baseline through Week 64
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Part B : Absolute Change From Part A and Part B Baseline in CFQ-R Respiratory Domain Score Through Week 64
Lasso di tempo: 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.
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Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
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Part B : Absolute Change From Part A and Part B Baseline in Sweat Chloride Concentration Through Week 64
Lasso di tempo: 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.
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Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
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Part B : Absolute Change From Part A and Part B Baseline in Weight Through Week 64
Lasso di tempo: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
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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.
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Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64
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Part B : Number of Participants With Pulmonary Exacerbations
Lasso di tempo: Part B baseline through Week 64
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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.
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Part B baseline through Week 64
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Part B : Number of Pulmonary Exacerbation Events
Lasso di tempo: Part B baseline through Week 64
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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.
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Part B baseline through Week 64
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Part B : Number of Pulmonary Exacerbation Events Per Participant Per Year
Lasso di tempo: Part B baseline through Week 64
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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.
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Part B baseline through Week 64
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Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Collaboratori
Investigatori
- Investigatore principale: Patrick A Flume, MD, Medical University of South Carolina
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.
Collegamenti utili
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
1 settembre 2009
Completamento primario (Effettivo)
1 luglio 2010
Completamento dello studio (Effettivo)
1 maggio 2013
Date di iscrizione allo studio
Primo inviato
4 agosto 2009
Primo inviato che soddisfa i criteri di controllo qualità
5 agosto 2009
Primo Inserito (Stima)
6 agosto 2009
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Stima)
11 settembre 2015
Ultimo aggiornamento inviato che soddisfa i criteri QC
27 agosto 2015
Ultimo verificato
1 agosto 2015
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
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
- VX08-770-104
- 2009-010261-23 (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