Back to resultsStudy of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
Primary Purpose
Cystic Fibrosis
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Lumacaftor
Ivacaftor
Lumacaftor Placebo
Ivacaftor Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis
Eligibility Criteria
Inclusion Criteria:
- Male or female participants with confirmed diagnosis of CF
- Must have the F508del-CFTR mutation on at least 1 allele.
- FEV1 greater than equal (>=) 40% of predicted normal for age, gender, and height (Knudson standards)(Cohort 1, 2, and 3); FEV1 40-90% of predicted normal for age, gender, and height (Hankinson standards (Cohort 4)
- Participant of child-bearing potential and who are sexually active must meet the contraception requirements
Exclusion Criteria:
- History of any illness or condition 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 participant (e.g., cirrhosis with portal hypertension).
- An acute illness including acute upper or lower respiratory infection, pulmonary exacerbation or changes in therapy (including antibiotics) for pulmonary disease within 14 days (Cohort 1, 2, and 3) or 28 days (Cohort 4) before receiving the first dose of study drug.
- History of solid organ or hematological transplantation.
- History of alcohol abuse or drug addiction in the past year, including cannabis, cocaine, and opiates.
- Ongoing participation in another therapeutic clinical study, or prior participation in an investigational drug study without appropriate washout
- Pregnant or nursing females; females of childbearing potential who are unwilling or unable to use an acceptable method of non-hormonal contraception
- Participants enrolled in Cohort 1 or Cohort 2 will not be eligible for Cohort 3
- Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit
- Heterozygous participants who participated in Cohort 2 and meet the eligibility criteria for Cohort 4 may participate in Cohort 4
- Evidence of lens opacity or cataract as determined by the ophthalmologic examination (Cohort 4 only)
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Arm 7
Arm 8
Arm 9
Arm 10
Arm Type
Placebo Comparator
Experimental
Experimental
Placebo Comparator
Experimental
Experimental
Experimental
Experimental
Placebo Comparator
Experimental
Arm Label
Cohort 1: Placebo
Cohort 1: LUM 200 mg qd/LUM 200 mg qd+IVA 150 mg q12h
Cohort 1: LUM 200 mg qd/LUM 200 mg qd+IVA 250 mg q12h
Cohort 2 and 3: Placebo (HO and HE)
Cohort 2: LUM 200 mg qd/LUM 200 mg qd+IVA 250 mg q12h (HO)
Cohort 2: LUM 400 mg qd/LUM 400 mg qd+IVA 250 mg q12h (HO)
Cohort 2: LUM 600 mg qd/LUM 600 mg qd+IVA 250 mg q12h (HO&HE)
Cohort 3: LUM 400 mg q12h/LUM 400 mg q12h+IVA 250 mg q12h (HO)
Cohort 4: Placebo
Cohort 4: LUM 400 mg q12h+IVA 250 mg q12h
Arm Description
Participants homozygous (HO) for the F508del-CF transmembrane conductance regulator gene (CFTR) mutation received lumacaftor matched placebo once daily (qd) (Day 1 through Day 14), followed by lumacaftor matched placebo qd in combination with ivacaftor matched placebo every 12 hours (q12h) (Day 15 through Day 21).
Participants homozygous for the F508del-CFTR mutation received 200 milligram (mg) of lumacaftor (LUM) qd (Day 1 through Day 14), followed by 200 mg of lumacaftor qd in combination with 150 mg of ivacaftor (IVA) q12h (Day 15 through Day 21).
Participants homozygous for the F508del-CFTR mutation received 200 mg of lumacaftor alone qd (Day 1 through Day 14), followed by 200 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 15 through Day 21).
Participants homozygous or heterozygous for the F508del-CFTR mutation received lumacaftor matched placebo qd (Day 1 through Day 28), followed by lumacaftor matched placebo in combination with ivacaftor matched placebo q12h (Day 29 through Day 56).
Participants homozygous for the F508del-CFTR mutation received 200 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 200 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Participants homozygous for the F508del-CFTR mutation received 400 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 400 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Participants homozygous or heterozygous for the F508del-CFTR mutation received 600 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 600 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Participants homozygous for the F508del-CFTR mutation received 400 mg of lumacaftor alone q12h (Day 1 through Day 28), followed by 400 mg of lumacaftor q12h in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Participants heterozygous for the F508del-CFTR mutation received lumacaftor matched placebo in combination with ivacaftor matched placebo q12h (Day 1 through Day 56).
Participants heterozygous for the F508del-CFTR mutation received 400 mg of lumacaftor q12h in combination with 250 mg of ivacaftor q12h (Day 1 through Day 56).
Outcomes
Primary Outcome Measures
Cohort 1: Safety and Tolerability Based on Adverse Events (AEs)
AE: any untoward medical occurrence in a participant during study; irrespective of relationship with treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent. AE includes serious AEs (SAEs) as well as Non-SAEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. AE that started at/after initial dosing of study drug, or increased in severity after initial dosing of study drug is considered treatment-emergent. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 14) and combination therapy period (Period 2: Day 15 to Day 21).
Cohort 2 and 3: Safety and Tolerability Based on Adverse Events (AEs)
Detailed description is provided in Outcome Measure 1. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 28) and combination therapy period (Period 2: Day 29 to Day 56).
Cohort 4: Safety and Tolerability Assessed by Number of Participants With AEs and SAEs
AEs and SAEs are defined in Outcome Measure 1.
Cohort 1: Absolute Change From Day 14 in Sweat Chloride at Day 21
Cohort 2 And 3: Absolute Change From Day 28 in Sweat Chloride at Day 56
Cohort 4: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 56
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 Hankinson method.
Secondary Outcome Measures
Cohort 1: Absolute Change From Baseline in Sweat Chloride at Day 14
Cohort 2 And 3: Absolute Change From Baseline in Sweat Chloride at Day 14
Cohort 4: Absolute Change From Baseline in Sweat Chloride at Day 56
Cohort 1: Absolute Change From Day 14 in FEV1 at Day 21
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Cohort 1: Absolute Change From Day 14 in ppFEV1 at Day 21
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Cohort 2 and 3: Absolute Change From Day 28 in ppFEV1 at Day 56
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Cohort 2 and 3: Relative Change From Day 28 in ppFEV1 at Day 56
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Cohort 2 and 3: Absolute Change From Baseline in ppFEV1 at Day 28 and 56
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Cohort 2 and 3: Relative Change From Baseline in FEV1 at Day 28 and 56
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Cohort 4: Relative Change From Baseline in Percent Predicted FEV1 at Day 56
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Cohort 2 and 3: Absolute Change From Day 28 in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Day 56
The CFQ-R is a validated participant-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.
Cohort 4: Absolute Change From Baseline in CFQ-R Respiratory Domain Score at Day 56
CFQ-R respiratory domain is defined in Outcome Measure 17.
Cohort 4: Absolute Change From Baseline in Body Mass Index (BMI) at Day 56
BMI was defined as weight in kilogram (kg) divided by height*height in square meter (m^2).
Cohort 4: Absolute Change From Baseline in Weight at Day 56
Full Information
NCT ID
NCT01225211
First Posted
October 15, 2010
Last Updated
September 2, 2015
Sponsor
Vertex Pharmaceuticals Incorporated
1. Study Identification
Unique Protocol Identification Number
NCT01225211
Brief Title
Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
Official Title
A Phase 2, Multicenter, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Study Type
Interventional
2. Study Status
Record Verification Date
September 2015
Overall Recruitment Status
Completed
Study Start Date
October 2010 (undefined)
Primary Completion Date
April 2014 (Actual)
Study Completion Date
April 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the F508del-CFTR mutation.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Factorial Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
312 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1: Placebo
Arm Type
Placebo Comparator
Arm Description
Participants homozygous (HO) for the F508del-CF transmembrane conductance regulator gene (CFTR) mutation received lumacaftor matched placebo once daily (qd) (Day 1 through Day 14), followed by lumacaftor matched placebo qd in combination with ivacaftor matched placebo every 12 hours (q12h) (Day 15 through Day 21).
Arm Title
Cohort 1: LUM 200 mg qd/LUM 200 mg qd+IVA 150 mg q12h
Arm Type
Experimental
Arm Description
Participants homozygous for the F508del-CFTR mutation received 200 milligram (mg) of lumacaftor (LUM) qd (Day 1 through Day 14), followed by 200 mg of lumacaftor qd in combination with 150 mg of ivacaftor (IVA) q12h (Day 15 through Day 21).
Arm Title
Cohort 1: LUM 200 mg qd/LUM 200 mg qd+IVA 250 mg q12h
Arm Type
Experimental
Arm Description
Participants homozygous for the F508del-CFTR mutation received 200 mg of lumacaftor alone qd (Day 1 through Day 14), followed by 200 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 15 through Day 21).
Arm Title
Cohort 2 and 3: Placebo (HO and HE)
Arm Type
Placebo Comparator
Arm Description
Participants homozygous or heterozygous for the F508del-CFTR mutation received lumacaftor matched placebo qd (Day 1 through Day 28), followed by lumacaftor matched placebo in combination with ivacaftor matched placebo q12h (Day 29 through Day 56).
Arm Title
Cohort 2: LUM 200 mg qd/LUM 200 mg qd+IVA 250 mg q12h (HO)
Arm Type
Experimental
Arm Description
Participants homozygous for the F508del-CFTR mutation received 200 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 200 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Arm Title
Cohort 2: LUM 400 mg qd/LUM 400 mg qd+IVA 250 mg q12h (HO)
Arm Type
Experimental
Arm Description
Participants homozygous for the F508del-CFTR mutation received 400 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 400 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Arm Title
Cohort 2: LUM 600 mg qd/LUM 600 mg qd+IVA 250 mg q12h (HO&HE)
Arm Type
Experimental
Arm Description
Participants homozygous or heterozygous for the F508del-CFTR mutation received 600 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 600 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Arm Title
Cohort 3: LUM 400 mg q12h/LUM 400 mg q12h+IVA 250 mg q12h (HO)
Arm Type
Experimental
Arm Description
Participants homozygous for the F508del-CFTR mutation received 400 mg of lumacaftor alone q12h (Day 1 through Day 28), followed by 400 mg of lumacaftor q12h in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Arm Title
Cohort 4: Placebo
Arm Type
Placebo Comparator
Arm Description
Participants heterozygous for the F508del-CFTR mutation received lumacaftor matched placebo in combination with ivacaftor matched placebo q12h (Day 1 through Day 56).
Arm Title
Cohort 4: LUM 400 mg q12h+IVA 250 mg q12h
Arm Type
Experimental
Arm Description
Participants heterozygous for the F508del-CFTR mutation received 400 mg of lumacaftor q12h in combination with 250 mg of ivacaftor q12h (Day 1 through Day 56).
Intervention Type
Drug
Intervention Name(s)
Lumacaftor
Other Intervention Name(s)
VX-809, LUM
Intervention Description
Tablet
Intervention Type
Drug
Intervention Name(s)
Ivacaftor
Other Intervention Name(s)
VX-770, IVA
Intervention Description
Tablet.
Intervention Type
Drug
Intervention Name(s)
Lumacaftor Placebo
Intervention Description
Matching placebo tablet.
Intervention Type
Drug
Intervention Name(s)
Ivacaftor Placebo
Intervention Description
Matching placebo tablet.
Primary Outcome Measure Information:
Title
Cohort 1: Safety and Tolerability Based on Adverse Events (AEs)
Description
AE: any untoward medical occurrence in a participant during study; irrespective of relationship with treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent. AE includes serious AEs (SAEs) as well as Non-SAEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. AE that started at/after initial dosing of study drug, or increased in severity after initial dosing of study drug is considered treatment-emergent. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 14) and combination therapy period (Period 2: Day 15 to Day 21).
Time Frame
Cohort 1: Day 1 up to 28 days after last dose (Last dose = Day 21)
Title
Cohort 2 and 3: Safety and Tolerability Based on Adverse Events (AEs)
Description
Detailed description is provided in Outcome Measure 1. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 28) and combination therapy period (Period 2: Day 29 to Day 56).
Time Frame
Cohort 2 and 3: Day 1 up to 28 days after last dose (Last dose = Day 56)
Title
Cohort 4: Safety and Tolerability Assessed by Number of Participants With AEs and SAEs
Description
AEs and SAEs are defined in Outcome Measure 1.
Time Frame
Cohort 4: Day 1 up to 28 days after last dose (Last dose = Day 56)
Title
Cohort 1: Absolute Change From Day 14 in Sweat Chloride at Day 21
Time Frame
Cohort 1: Day 14, Day 21
Title
Cohort 2 And 3: Absolute Change From Day 28 in Sweat Chloride at Day 56
Time Frame
Cohort 2 and 3: Day 28, Day 56
Title
Cohort 4: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 56
Description
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 Hankinson method.
Time Frame
Cohort 4: Baseline, Day 56
Secondary Outcome Measure Information:
Title
Cohort 1: Absolute Change From Baseline in Sweat Chloride at Day 14
Time Frame
Cohort 1: Baseline, Day 14
Title
Cohort 2 And 3: Absolute Change From Baseline in Sweat Chloride at Day 14
Time Frame
Cohort 2: Baseline, Day 14
Title
Cohort 4: Absolute Change From Baseline in Sweat Chloride at Day 56
Time Frame
Cohort 4: Baseline, Day 56
Title
Cohort 1: Absolute Change From Day 14 in FEV1 at Day 21
Description
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time Frame
Cohort 1: Day 14, Day 21
Title
Cohort 1: Absolute Change From Day 14 in ppFEV1 at Day 21
Description
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Time Frame
Cohort 1: Day 14, Day 21
Title
Cohort 2 and 3: Absolute Change From Day 28 in ppFEV1 at Day 56
Description
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Time Frame
Cohort 2 and 3: Day 28, Day 56
Title
Cohort 2 and 3: Relative Change From Day 28 in ppFEV1 at Day 56
Description
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Time Frame
Cohort 2 and 3: Day 28, Day 56
Title
Cohort 2 and 3: Absolute Change From Baseline in ppFEV1 at Day 28 and 56
Description
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Time Frame
Cohort 2 and 3: Baseline, Day 28 and 56
Title
Cohort 2 and 3: Relative Change From Baseline in FEV1 at Day 28 and 56
Description
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Time Frame
Cohort 2 and 3: Baseline, Day 28 and 56
Title
Cohort 4: Relative Change From Baseline in Percent Predicted FEV1 at Day 56
Description
FEV1 and ppFEV1 are defined in Outcome Measure 6.
Time Frame
Cohort 4: Baseline, Day 56
Title
Cohort 2 and 3: Absolute Change From Day 28 in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Day 56
Description
The CFQ-R is a validated participant-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.
Time Frame
Cohort 2 and 3: Day 28, Day 56
Title
Cohort 4: Absolute Change From Baseline in CFQ-R Respiratory Domain Score at Day 56
Description
CFQ-R respiratory domain is defined in Outcome Measure 17.
Time Frame
Cohort 4: Baseline, Day 56
Title
Cohort 4: Absolute Change From Baseline in Body Mass Index (BMI) at Day 56
Description
BMI was defined as weight in kilogram (kg) divided by height*height in square meter (m^2).
Time Frame
Cohort 4: Baseline, Day 56
Title
Cohort 4: Absolute Change From Baseline in Weight at Day 56
Time Frame
Cohort 4: Baseline, Day 56
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female participants with confirmed diagnosis of CF
Must have the F508del-CFTR mutation on at least 1 allele.
FEV1 greater than equal (>=) 40% of predicted normal for age, gender, and height (Knudson standards)(Cohort 1, 2, and 3); FEV1 40-90% of predicted normal for age, gender, and height (Hankinson standards (Cohort 4)
Participant of child-bearing potential and who are sexually active must meet the contraception requirements
Exclusion Criteria:
History of any illness or condition 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 participant (e.g., cirrhosis with portal hypertension).
An acute illness including acute upper or lower respiratory infection, pulmonary exacerbation or changes in therapy (including antibiotics) for pulmonary disease within 14 days (Cohort 1, 2, and 3) or 28 days (Cohort 4) before receiving the first dose of study drug.
History of solid organ or hematological transplantation.
History of alcohol abuse or drug addiction in the past year, including cannabis, cocaine, and opiates.
Ongoing participation in another therapeutic clinical study, or prior participation in an investigational drug study without appropriate washout
Pregnant or nursing females; females of childbearing potential who are unwilling or unable to use an acceptable method of non-hormonal contraception
Participants enrolled in Cohort 1 or Cohort 2 will not be eligible for Cohort 3
Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit
Heterozygous participants who participated in Cohort 2 and meet the eligibility criteria for Cohort 4 may participate in Cohort 4
Evidence of lens opacity or cataract as determined by the ophthalmologic examination (Cohort 4 only)
Facility Information:
City
Birmingham
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Alabama
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United States
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Anchorage
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Alaska
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La Jolla
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Long Beach
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Palo Alto
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Sacramento
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Denver
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Colorado
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New Haven
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Miami
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Orlando
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Tampa
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Atlanta
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Boise
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Chicago
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Iowa City
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Lexington
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New Orleans
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Baltimore
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Boston
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St. Louis
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Omaha
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Lebanon
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Buffalo
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New Hyde Park
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New York
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New York
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Rochester
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New York
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Syracuse
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New York
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Chapel Hill
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North Carolina
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Akron
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Ohio
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Cleveland
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Columbus
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Oklahoma City
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Oklahoma
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Philadelphia
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Pittsburgh
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Pittsburg
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Charleston
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South Carolina
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Sioux Falls
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South Dakota
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Adelaide
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Australia
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Brisbane
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Australia
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Chermside
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Australia
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Nedlands
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Australia
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Parkville Victoria
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Australia
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Westmead
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Australia
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Leuven
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Belgium
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Pierre Benite Cedex
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Rhone
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France
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Paris
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France
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Jena
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Thueringen
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Germany
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Berlin
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Germany
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Bochum
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Germany
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Essen
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Germany
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Koeln
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Germany
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Auckland
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New Zealand
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Christchurch
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New Zealand
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London
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United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
33331662
Citation
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Results Reference
derived
PubMed Identifier
27898234
Citation
Rowe SM, McColley SA, Rietschel E, Li X, Bell SC, Konstan MW, Marigowda G, Waltz D, Boyle MP; VX09-809-102 Study Group. Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR. Ann Am Thorac Soc. 2017 Feb;14(2):213-219. doi: 10.1513/AnnalsATS.201609-689OC.
Results Reference
derived
PubMed Identifier
24973281
Citation
Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D; VX09-809-102 study group. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24.
Results Reference
derived
Learn more about this trial
Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
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