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The objective of this study was to evaluate the safety and efficacy of VX-661in combination with ivacaftor in participants with cystic fibrosis (CF) who are homozygous for F508del cystic fibrosis transmembrane conductance regulator (CFTR) mutation
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h | Experimental | Participants received VX-661 50 milligram (mg) tablet plus Ivacaftor (IVA) 150 mg tablet every 12 hours (q12h) for 12 weeks. |
|
| PC Phase: VX 661 placebo q12h + IVA placebo q12h | Placebo Comparator | Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks. |
|
| PC Phase: VX-661 100 mg qd + IVA 150 mg q12h | Experimental | Participants received two VX-661 50 mg tablets once daily (qd) plus IVA 150 mg tablet q12h for 12 weeks. |
|
| PC Phase: VX -661 placebo qd + IVA placebo q12h | Placebo Comparator | Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. |
|
| OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h | Experimental | Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-661 | Drug | Tablet, oral use |
|
| Measure | Description | Time Frame |
|---|---|---|
| PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of PC Phase. | Baseline (PC Phase) up to 112 days |
| OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs | AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of the OLE Phase. | Baseline (OLE Phase) up to 364 days |
| Measure | Description | Time Frame |
|---|---|---|
| PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase. | Baseline (PC Phase), Through Week 12 |
| OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | United States | ||||
The study consisted of 2 phases: a randomized, double-blind, placebo-controlled (PC) phase in which participants received either VX-661 in combination with Ivacaftor (IVA), or matched placebo and an open-label extension (OLE) phase in which participants received VX-661 in combination with IVA.
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| ID | Title | Description |
|---|---|---|
| FG000 | PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h | Participants received VX-661 50 milligram (mg) tablet plus IVA 150 mg tablet every 12 hours (q12h) for 12 weeks. |
| FG001 | PC Phase: VX-661 Placebo q12h + IVA Placebo q12h |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| PC Phase (12 Weeks) |
|
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|
| Ivacaftor | Drug | Film coated tablet, oral use |
|
| Placebo matched to VX-661 | Drug | Tablet, oral use |
|
| Placebo matched to Ivacaftor | Drug | Film coated tablet, oral use |
|
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase. |
| Baseline (OLE Phase), Through Week 40 |
| PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase. | Baseline (PC Phase), Through Week 12 |
| OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase. | Baseline (OLE Phase), Through Week 40 |
| PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12 | Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of PC Phase. | Baseline (PC Phase), Through Week 12 |
| OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40 | Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of the OLE Phase. | Baseline (OLE Phase), Through Week 40 |
| PC Phase: Absolute Change From Baseline in Body Weight at Week 12 | Baseline was defined as Day 1 of PC Phase. | Baseline (PC Phase), Week 12 |
| OLE Phase: Absolute Change From Baseline in Body Weight at Week 40 | Baseline was defined as Day 1 of the OLE Phase. | Baseline (OLE Phase), Week 40 |
| PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12 | BMI was calculated using following formula: BMI = Weight in kg/height in square meter (m^2). Baseline was defined as Day 1 of PC Phase. | Baseline (PC Phase), Week 12 |
| OLE Phase: Absolute Change From Baseline BMI at Week 40 | BMI was calculated using following formula: BMI = Weight in kg/height in m^2. Baseline was defined as Day 1 of the OLE Phase. | Baseline (OLE Phase), Week 40 |
| PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12 | 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 (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Baseline was defined as Day 1 of PC Phase. | Baseline (PC Phase), Through Week 12 |
| OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40 | 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 (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Baseline was defined as Day 1 of the OLE Phase. | Baseline (OLE Phase), Through Week 40 |
| PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA | Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85 |
| PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661 | Pharmacokinetic (PK) sampling was performed up to 12 hours post-dose on Day 85. For Arm VX-661 50 mg q12h + IVA 150 mg q12h (VX-661 q12h regimen), area under the concentration versus time curve from time 0 to 12 hours (AUC0-12h) was multiplied by 2 to obtain AUC0-24h. | Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85 |
| PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA | Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85 |
| PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA | Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85 |
| Palo Alto |
| California |
| United States |
| Stanford | California | United States |
| Altamonte Springs | Florida | United States |
| Miami | Florida | United States |
| Orlando | Florida | United States |
| Tampa | Florida | United States |
| Boise | Idaho | United States |
| Chicago | Illinois | United States |
| Boston | Massachusetts | United States |
| New Brunswick | New Jersey | United States |
| New York | New York | United States |
| Durham | North Carolina | United States |
| Cincinnati | Ohio | United States |
| Oklahoma City | Oklahoma | United States |
| Pittsburgh | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Memphis | Tennessee | United States |
| Houston | Texas | United States |
| Burlington | Vermont | United States |
| Colchester | Vermont | United States |
| Seattle | Washington | United States |
| Milwaukee | Wisconsin | United States |
Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.
| FG002 | PC Phase: VX-661 100 mg qd + IVA 150 mg q12h | Participants received two VX-661 50 mg tablets once daily (qd) plus IVA 150 mg tablet q12h for 12 weeks. |
| FG003 | PC Phase: VX -661 Placebo qd + IVA Placebo q12h | Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. |
| FG004 | OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h | Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase. |
| Treated |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| OLE Phase (48 Weeks) |
|
|
Full Analysis Set (FAS) was defined as all randomized participants who received at least 1 dose of study drug in PC Phase.
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| ID | Title | Description |
|---|---|---|
| BG000 | PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h | Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks. |
| BG001 | PC Phase: VX-661 Placebo q12h + IVA Placebo q12h | Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks. |
| BG002 | PC Phase: VX-661 100 mg qd + IVA 150 mg q12h | Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks. |
| BG003 | PC Phase: VX -661 Placebo qd + IVA Placebo q12h | Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of PC Phase. | Safety Set was defined as all participants who received at least 1 dose of study drug in PC Phase. | Posted | Count of Participants | Participants | Baseline (PC Phase) up to 112 days |
|
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| |||||||||||||||||||||||||||||||||||
| Primary | OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs | AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of the OLE Phase. | Safety Set was defined as all participants who received at least 1 dose of study drug in OLE Phase. | Posted | Count of Participants | Participants | Baseline (OLE Phase) up to 364 days |
|
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| Secondary | PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase. | FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase. | Posted | Least Squares Mean | 95% Confidence Interval | Percent predicted of FEV1 | Baseline (PC Phase), Through Week 12 |
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| Secondary | OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase. | Analysis population was defined as all participants who received at least 1 dose of study drug in OLE phase. | Posted | Least Squares Mean | 95% Confidence Interval | Percent predicted of FEV1 | Baseline (OLE Phase), Through Week 40 |
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| Secondary | PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase. | FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase. | Posted | Least Squares Mean | 95% Confidence Interval | Percent change | Baseline (PC Phase), Through Week 12 |
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| Secondary | OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase. | Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase. | Posted | Least Squares Mean | 95% Confidence Interval | Percent change | Baseline (OLE Phase), Through Week 40 |
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| Secondary | PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12 | Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of PC Phase. | FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase. Here, "Number of participants analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Millimole per liter (mmol/L) | Baseline (PC Phase), Through Week 12 |
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| Secondary | OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40 | Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of the OLE Phase. | Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase. Here, "Number of participants analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | mmol/L | Baseline (OLE Phase), Through Week 40 |
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| Secondary | PC Phase: Absolute Change From Baseline in Body Weight at Week 12 | Baseline was defined as Day 1 of PC Phase. | FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase. | Posted | Least Squares Mean | 95% Confidence Interval | kilogram (kg) | Baseline (PC Phase), Week 12 |
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| Secondary | OLE Phase: Absolute Change From Baseline in Body Weight at Week 40 | Baseline was defined as Day 1 of the OLE Phase. | Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase. | Posted | Least Squares Mean | 95% Confidence Interval | kg | Baseline (OLE Phase), Week 40 |
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| Secondary | PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12 | BMI was calculated using following formula: BMI = Weight in kg/height in square meter (m^2). Baseline was defined as Day 1 of PC Phase. | FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase. | Posted | Least Squares Mean | 95% Confidence Interval | Kilogram per square meter (kg/m^2) | Baseline (PC Phase), Week 12 |
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| Secondary | OLE Phase: Absolute Change From Baseline BMI at Week 40 | BMI was calculated using following formula: BMI = Weight in kg/height in m^2. Baseline was defined as Day 1 of the OLE Phase. | Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase. | Posted | Least Squares Mean | 95% Confidence Interval | kg/m^2 | Baseline (OLE Phase), Week 40 |
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| Secondary | PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12 | 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 (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Baseline was defined as Day 1 of PC Phase. | FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Baseline (PC Phase), Through Week 12 |
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| Secondary | OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40 | 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 (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Baseline was defined as Day 1 of the OLE Phase. | Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Baseline (OLE Phase), Through Week 40 |
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| Secondary | PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA | Analysis population included all participants who received a dose of VX-661 and IVA, whether the participant completed dosing or not and if the dataset(s) supported the noncompartmental analyses. | Posted | Mean | Standard Deviation | Nanogram per milliliter (ng/mL) | Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85 |
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| Secondary | PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661 | Pharmacokinetic (PK) sampling was performed up to 12 hours post-dose on Day 85. For Arm VX-661 50 mg q12h + IVA 150 mg q12h (VX-661 q12h regimen), area under the concentration versus time curve from time 0 to 12 hours (AUC0-12h) was multiplied by 2 to obtain AUC0-24h. | Analysis population included all participants who received a dose of VX-661 and IVA, whether the participant completed dosing or not and if the dataset(s) supported the noncompartmental analyses. Here, "Number of participants analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Hour*nanogram per milliliter (hr*ng/mL) | Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85 |
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| Secondary | PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA | Analysis population included all participants who received a dose of VX-661 and IVA, whether the participant completed dosing or not and if the dataset(s) supported the noncompartmental analyses. Here, "Number of participants analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | hr*ng/mL | Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85 |
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| Secondary | PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA | Analysis population included all participants who received a dose of VX-661 and IVA, whether the participant completed dosing or not and if the dataset(s) supported the noncompartmental analyses. | Posted | Median | Full Range | hour | Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85 |
|
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Baseline (Day 1 of PC Phase) up to 112 days (Placebo control phase); Baseline (Day 1 of OLE Phase) up to 364 days (OLE phase)
Safety Set was defined as all participants who received at least 1 dose of study drug in PC Phase and OLE Phase.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h | Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks. | 0 | 6 | 1 | 6 | 6 | 6 |
| EG001 | PC Phase: VX 661 Placebo q12h + IVA Placebo q12h | Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks. | 0 | 5 | 2 | 5 | 5 | 5 |
| EG002 | PC Phase: VX-661 100 mg qd + IVA 150 mg q12h | Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks. | 0 | 15 | 4 | 15 | 15 | 15 |
| EG003 | PC Phase: VX -661 Placebo qd + IVA Placebo q12h | Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. | 0 | 13 | 5 | 13 | 13 | 13 |
| EG004 | OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h | Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase. | 0 | 27 | 6 | 27 | 25 | 27 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Chronic sinusitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Cystic fibrosis related diabetes | Congenital, familial and genetic disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Sputum discoloured | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Nasal oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Painful respiration | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pleurisy | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Heart rate increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood bilirubin unconjugated increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood triglycerides increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Breath sounds abnormal | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Urine output decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Vitamin D decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Prothrombin time prolonged | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Vitamin A decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Vitamin K decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| White blood cells urine positive | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Bacterial vaginosis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Acute sinusitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Chronic sinusitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Vulvovaginal mycotic infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Peptic ulcer | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hyposmia | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Sinus headache | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Vaccination site discomfort | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Vitamin E deficiency | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Menorrhagia | Reproductive system and breast disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Testicular pain | Reproductive system and breast disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Amenorrhoea | Reproductive system and breast disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Breast tenderness | Reproductive system and breast disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Lipoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 19.0 | Systematic Assessment |
| |
| Disorientation | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
PI is free to publish results of the study after (1) the first multi-center publication, (2) if the sponsor elects not to publish the results, or (3) 18 months after close of the study, whichever occurs first. Proposed publications are to be submitted to the sponsor for review and comment for a period of at least 45 days (which may be extended under certain circumstances related to protection of intellectual property); the sponsor cannot require changes to the proposed publications.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000625213 | tezacaftor |
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Other |
|
| Male |
|
| Participants with SAEs |
|
| Counts |
|---|
| Participants |
|
|
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. |
|
|
|
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
|
|
|
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. |
|
|
|
|
|
|
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
|
|
|
| OG003 | PC Phase: VX -661 Placebo qd + IVA Placebo q12h | Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. |
|
|
|
|
| Participants |
|
|
|
|