Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
27 april 2021 uppdaterad av: Vertex Pharmaceuticals Incorporated
A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233).
Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.
Studieöversikt
Status
Avslutad
Betingelser
Intervention / Behandling
Studietyp
Interventionell
Inskrivning (Faktisk)
246
Fas
- Fas 3
Kontakter och platser
Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.
Studieorter
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Herston, Australien
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New Lambton Heights, Australien
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Subiaco, Australien
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Westmead, Australien
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Victoria
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Parkville, Victoria, Australien
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Brussels, Belgien
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Leuven, Belgien
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Copenhagen, Danmark
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Bordeaux Cedex, Frankrike
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Paris, Frankrike
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Paris Cedex 15, Frankrike
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Cedex
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Bron, Cedex, Frankrike
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Alabama
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Birmingham, Alabama, Förenta staterna
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Arizona
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Tucson, Arizona, Förenta staterna
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California
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Long Beach, California, Förenta staterna
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Palo Alto, California, Förenta staterna
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Colorado
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Aurora, Colorado, Förenta staterna
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Delaware
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Wilmington, Delaware, Förenta staterna
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Florida
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Jacksonville, Florida, Förenta staterna
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Orlando, Florida, Förenta staterna
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Georgia
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Atlanta, Georgia, Förenta staterna
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Illinois
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Chicago, Illinois, Förenta staterna
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Indiana
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Indianapolis, Indiana, Förenta staterna
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Iowa
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Iowa City, Iowa, Förenta staterna
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Massachusetts
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Boston, Massachusetts, Förenta staterna
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Minnesota
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Minneapolis, Minnesota, Förenta staterna
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Missouri
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Kansas City, Missouri, Förenta staterna
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Saint Louis, Missouri, Förenta staterna
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Nebraska
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Omaha, Nebraska, Förenta staterna
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New Hampshire
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Lebanon, New Hampshire, Förenta staterna
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New York
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Buffalo, New York, Förenta staterna
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Syracuse, New York, Förenta staterna
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North Carolina
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Chapel Hill, North Carolina, Förenta staterna
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Ohio
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Cincinnati, Ohio, Förenta staterna
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Cleveland, Ohio, Förenta staterna
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Dayton, Ohio, Förenta staterna
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Oregon
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Portland, Oregon, Förenta staterna
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Pennsylvania
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Pittsburgh, Pennsylvania, Förenta staterna
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South Carolina
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Charleston, South Carolina, Förenta staterna
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Texas
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Austin, Texas, Förenta staterna
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Houston, Texas, Förenta staterna
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Utah
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Salt Lake City, Utah, Förenta staterna
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Vermont
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Colchester, Vermont, Förenta staterna
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Virginia
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Charlottesville, Virginia, Förenta staterna
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Norfolk, Virginia, Förenta staterna
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Richmond, Virginia, Förenta staterna
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Washington
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Seattle, Washington, Förenta staterna
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Wisconsin
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Madison, Wisconsin, Förenta staterna
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Milwaukee, Wisconsin, Förenta staterna
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British Columbia
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Vancouver, British Columbia, Kanada
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Ontario
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Toronto, Ontario, Kanada
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Quebec
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Montreal, Quebec, Kanada
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Belfast, Storbritannien
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Edinburgh, Storbritannien
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London, Storbritannien
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West Yorkshire
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Leeds, West Yorkshire, Storbritannien
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Stockholm, Sverige
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Berlin, Tyskland
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Giessen, Tyskland
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Hanover, Tyskland
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Koeln, Tyskland
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Deltagandekriterier
Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.
Urvalskriterier
Åldrar som är berättigade till studier
6 år och äldre (Barn, Vuxen, Äldre vuxen)
Tar emot friska volontärer
Nej
Kön som är behöriga för studier
Allt
Beskrivning
Inclusion Criteria:
Subjects entering the Treatment Cohort must meet both of the following criteria:
- Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment
- Willing to remain on a stable CF medication through the Safety Follow-up Visit.
Subjects entering the Observational Cohort must meet 1 of the following criteria:
- Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B.
- Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.
Exclusion Criteria (Treatment Cohort Only):
- History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject (e.g., cirrhosis with portal hypertension).
- Pregnant and nursing females.
- Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
- History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator
- History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator.
- Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor).
Studieplan
Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: Icke-randomiserad
- Interventionsmodell: Parallellt uppdrag
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
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Experimentell: Treatment Period 1: LUM/IVA to LUM/IVA
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Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age). LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).
Andra namn:
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andra namn:
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Experimentell: Treatment Period 1: Placebo (PBO) to LUM/IVA
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Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age). LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).
Andra namn:
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andra namn:
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Inget ingripande: Treatment Period 1: Observational Cohort
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Experimentell: Treatment Period 2: LUM/IVA
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Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age). LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).
Andra namn:
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andra namn:
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Vad mäter studien?
Primära resultatmått
Resultatmått |
Tidsram |
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Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Tidsram: Day 1 up to Week 100
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Day 1 up to Week 100
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Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
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Absolute Change in Lung Clearance Index (LCI) 2.5
Tidsram: From Parent Study Baseline at Week 96
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LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
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From Parent Study Baseline at Week 96
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Absolute Change in Sweat Chloride
Tidsram: From Parent Study Baseline at Week 96
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Sweat samples were collected using an approved collection device.
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From Parent Study Baseline at Week 96
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Absolute Change in Body Mass Index (BMI)
Tidsram: From Parent Study Baseline at Week 96
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BMI was defined as weight in kilograms divided by height in square meter (m^2).
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From Parent Study Baseline at Week 96
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Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
Tidsram: From Parent Study Baseline at Week 96
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis.
Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
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From Parent Study Baseline at Week 96
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Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)
Tidsram: Day 1 up to Week 100
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Day 1 up to Week 100
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Absolute Change in LCI 5.0
Tidsram: From Parent Study Baseline at Week 96
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LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value.
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From Parent Study Baseline at Week 96
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Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Tidsram: From Parent Study Baseline at Week 96
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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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From Parent Study Baseline at Week 96
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Relative Change in ppFEV1
Tidsram: From Parent Study Baseline at Week 96
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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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From Parent Study Baseline at Week 96
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Absolute Change in BMI-for-age Z-score
Tidsram: From Parent Study Baseline at Week 96
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BMI was defined as weight in kilograms divided by height in m^2.
z-score is a statistical measure to describe whether a mean was above or below the standard.
A z-score of 0 is equal to the mean and is considered normal.
Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
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From Parent Study Baseline at Week 96
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Absolute Change in Weight
Tidsram: From Parent Study Baseline at Week 96
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From Parent Study Baseline at Week 96
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Absolute Change in Weight-for-age Z-score
Tidsram: From Parent Study Baseline at Week 96
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z-score is a statistical measure to describe whether a mean was above or below the standard.
A z-score of 0 is equal to the mean and is considered normal.
Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
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From Parent Study Baseline at Week 96
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Absolute Change in Height
Tidsram: From Parent Study Baseline at Week 96
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From Parent Study Baseline at Week 96
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Absolute Change in Height-for-age Z-score
Tidsram: From Parent Study Baseline at Week 96
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z-score is a statistical measure to describe whether a mean was above or below the standard.
A z-score of 0 is equal to the mean and is considered normal.
Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
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From Parent Study Baseline at Week 96
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Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score
Tidsram: From Parent Study Baseline at Week 96
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The TSQM measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction.
For each dimension, responses are added and transformed in the total domain score, which ranges from 0 to 100, where higher scores indicate greater satisfaction.
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From Parent Study Baseline at Week 96
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Time-to-first Pulmonary Exacerbation
Tidsram: From Parent Study Baseline through Week 96
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Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
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From Parent Study Baseline through Week 96
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Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event
Tidsram: From Parent Study Baseline through Week 96
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Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
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From Parent Study Baseline through Week 96
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Number of Pulmonary Exacerbation Events Per Patient-year
Tidsram: From Parent Study Baseline through Week 96
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Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
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From Parent Study Baseline through Week 96
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Rate of Change in LCI 2.5
Tidsram: Day 15 after first dose of LUM/IVA through Week 96
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Rate of change analysis evaluates the change in LCI 2.5 after long term treatment with LUM/IVA.
A rate of change equal to zero would indicate that treatment effects were stable.
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Day 15 after first dose of LUM/IVA through Week 96
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Rate of Change in LCI 5.0
Tidsram: Day 15 after first dose of LUM/IVA through Week 96
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Rate of change analysis evaluates the change in LCI 5.0 after long term treatment with LUM/IVA.
A rate of change equal to zero would indicate that treatment effects were stable.
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Day 15 after first dose of LUM/IVA through Week 96
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Rate of Change in ppFEV1
Tidsram: Day 15 after first dose of LUM/IVA through Week 96
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Rate of change analysis evaluates the change in ppFEV1 after long term treatment with LUM/IVA.
A rate of change equal to zero would indicate that treatment effects were stable.
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Day 15 after first dose of LUM/IVA through Week 96
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Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Tidsram: Day 1 up to Week 168
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Day 1 up to Week 168
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Samarbetspartners och utredare
Det är här du hittar personer och organisationer som är involverade i denna studie.
Publikationer och användbara länkar
Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.
Studieavstämningsdatum
Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.
Studera stora datum
Studiestart (Faktisk)
1 augusti 2015
Primärt slutförande (Faktisk)
1 augusti 2018
Avslutad studie (Faktisk)
1 april 2020
Studieregistreringsdatum
Först inskickad
1 september 2015
Först inskickad som uppfyllde QC-kriterierna
4 september 2015
Första postat (Uppskatta)
9 september 2015
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
24 maj 2021
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
27 april 2021
Senast verifierad
1 februari 2021
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- VX15-809-110
Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .