Trial Outcomes & Findings for A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation (NCT NCT02516410)

NCT ID: NCT02516410

Last Updated: 2018-06-12

Results Overview

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

168 participants

Primary outcome timeframe

Baseline, Through Week 12

Results posted on

2018-06-12

Participant Flow

The study was conducted across 38 sites in 7 countries.

A total of 168 participants were randomized and treated in the study.

Participant milestones

Participant milestones
Measure
Placebo
Placebo matched to VX-661 plus Ivacaftor (IVA, VX-770) fixed dose combination (FDC) tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
VX-661 100 milligram (mg) plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Overall Study
STARTED
85
83
Overall Study
COMPLETED
85
81
Overall Study
NOT COMPLETED
0
2

Reasons for withdrawal

Reasons for withdrawal
Measure
Placebo
Placebo matched to VX-661 plus Ivacaftor (IVA, VX-770) fixed dose combination (FDC) tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
VX-661 100 milligram (mg) plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Overall Study
Adverse Event
0
1
Overall Study
Withdrawal by Subject
0
1

Baseline Characteristics

A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=83 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Total
n=168 Participants
Total of all reporting groups
Age, Continuous
26 years
STANDARD_DEVIATION 8.8 • n=5 Participants
26.2 years
STANDARD_DEVIATION 9.6 • n=7 Participants
26.1 years
STANDARD_DEVIATION 9.2 • n=5 Participants
Sex: Female, Male
Female
42 Participants
n=5 Participants
39 Participants
n=7 Participants
81 Participants
n=5 Participants
Sex: Female, Male
Male
43 Participants
n=5 Participants
44 Participants
n=7 Participants
87 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Through Week 12

Population: Full Analysis Set (FAS) included all randomized participants who received at least 1 dose of study drug. Here 'Number of participants analysed' signifies those participants who were evaluable for this outcome measure.

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years.

Outcome measures

Outcome measures
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=82 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12
-0.1 Percentage of predicted FEV1
Standard Error 0.6
1 Percentage of predicted FEV1
Standard Error 0.6

SECONDARY outcome

Timeframe: Baseline, Through Week 12

Population: FAS included all randomized participants who received at least 1 dose of study drug.

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.

Outcome measures

Outcome measures
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=83 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12
3.8 Units on a scale
Interval 1.1 to 6.4
5.9 Units on a scale
Interval 3.2 to 8.5

SECONDARY outcome

Timeframe: Baseline through Week 12

Population: FAS included all randomized participants who received at least 1 dose of study drug.

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. The number of events were reported.

Outcome measures

Outcome measures
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=83 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Number of Pulmonary Exacerbation Events
23 Pulmonary exacerbation events
22 Pulmonary exacerbation events

SECONDARY outcome

Timeframe: Baseline through Week 12

Population: FAS included all randomized participants who received at least 1 dose of study drug.

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. Total number of days on study is equal to the Week 12 date or the last dose date (whichever occurs last) minus first dose date plus 1. The total number of years (48 weeks) on study is equal to the total number of days on study divided by 336. Pulmonary exacerbation events per year (48 weeks) are reported.

Outcome measures

Outcome measures
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=83 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Number of Pulmonary Exacerbation Events Per Year
0.98 Pulmonary exacerbation events per year
0.97 Pulmonary exacerbation events per year

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: FAS included all randomized participants who received at least 1 dose of study drug. Here 'Number of participants analysed' signifies those participants who were evaluable for this outcome measure.

BMI was defined as weight in kilogram (kg) divided by height\*height in square meter (m\^2).

Outcome measures

Outcome measures
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=82 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Absolute Change From Baseline in Body Mass Index (BMI) at Week 12
0.22 Kg/m^2
Interval 0.07 to 0.36
0.14 Kg/m^2
Interval -0.01 to 0.28

SECONDARY outcome

Timeframe: Baseline, Through Week 12

Population: FAS included all randomized participants who received at least 1 dose of study drug. Here 'Number of participants analysed' signifies those participants who were evaluable for this outcome measure.

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years.

Outcome measures

Outcome measures
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=82 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Relative Change From Baseline in Percent Predicted FEV1 Through Week 12
0.1 Percent Change
Interval -2.1 to 2.3
2.1 Percent Change
Interval -0.1 to 4.3

SECONDARY outcome

Timeframe: Baseline, Through Week 12

Population: FAS included all randomized participants who received at least 1 dose of study drug. Here 'Number of participants analysed' signifies those participants who were evaluable for this outcome measure.

Sweat samples were collected using an approved collection device.

Outcome measures

Outcome measures
Measure
Placebo
n=84 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=79 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Absolute Change From Baseline in Sweat Chloride Through Week 12
-1.2 Millimole per liter (mmol/L)
Interval -3.1 to 0.7
-4.7 Millimole per liter (mmol/L)
Interval -6.6 to -2.8

SECONDARY outcome

Timeframe: Baseline through Week 12

Population: FAS included all randomized participants who received at least 1 dose of study drug.

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. Time-to-first pulmonary exacerbation was planned to be estimated using Kaplan-Meier (KM) estimates. However, due to less than 50% of events, time-to-first event data was not estimated. Instead, number of participants with at least one pulmonary exacerbation event were collected and are reported.

Outcome measures

Outcome measures
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=83 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Number of Participants With at Least One Pulmonary Exacerbation Through Week 12
21 Participants
19 Participants

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: Analysis population included all randomized participants who received at least 1 dose of study drug and were \<20 years of age at the time of screening.

Z-score is a statistical measure to evaluate how a single data point compares to a standard. It describes whether a mean was above or below the standard and how unusual the measurement is, with range from infinity to +infinity; where 0: same mean, \>0: a greater mean, and \<0: a lesser mean than the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age Z-score (BMI z-score). BMI-for-age z-score was calculated by using centers for disease control and prevention (CDC) growth charts for the paediatric population.

Outcome measures

Outcome measures
Measure
Placebo
n=22 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=25 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Absolute Change From Baseline in BMI Z-score at Week 12 (in Participants Less Than [<] 20 Years Old at the Time of Screening)
0.07 Z-score
Interval -0.05 to 0.19
0.02 Z-score
Interval -0.09 to 0.13

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: FAS included all randomized participants who received at least 1 dose of study drug. Here 'Number of participants analysed' signifies those participants who were evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=82 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Absolute Change From Baseline in Body Weight at Week 12
0.7 Kilograms (kg)
Interval 0.3 to 1.1
0.6 Kilograms (kg)
Interval 0.2 to 1.0

SECONDARY outcome

Timeframe: Baseline up to Week 16

Population: Safety Set included all participants who received at least 1 dose of the study drug.

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. Any AE that increased in severity or newly developed at or after initial dosing of study drug to Week 16 was considered treatment-emergent.

Outcome measures

Outcome measures
Measure
Placebo
n=85 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=83 Participants
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with any AEs
68 Participants
64 Participants
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with any SAEs
14 Participants
11 Participants

SECONDARY outcome

Timeframe: Pre-morning dose on Week 2, Week 4, Week 8 and Week 12

Population: Pharmacokinetic (PK) set included all randomized participants who received any amount of study drug and had a PK assessment. Here, 'Number of participants analyzed' = those participants who were evaluable for this endpoint and 'Number Analyzed' = those participants who were evaluable at the specified time points for each arm, respectively.

This outcome was not planned to be assessed in Placebo arm.

Outcome measures

Outcome measures
Measure
Placebo
n=82 Participants
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 2: VX-661
1880 nanogram per milliliter (ng/mL)
Standard Deviation 1230
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 2: M1 VX-661
4600 nanogram per milliliter (ng/mL)
Standard Deviation 1430
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 2: IVA
831 nanogram per milliliter (ng/mL)
Standard Deviation 607
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 2: M1-IVA
1580 nanogram per milliliter (ng/mL)
Standard Deviation 879
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 4: VX-661
1780 nanogram per milliliter (ng/mL)
Standard Deviation 1010
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 4: M1 VX-661
4420 nanogram per milliliter (ng/mL)
Standard Deviation 1340
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 4: IVA
770 nanogram per milliliter (ng/mL)
Standard Deviation 596
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 4: M1-IVA
1500 nanogram per milliliter (ng/mL)
Standard Deviation 898
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 8: VX-661
1920 nanogram per milliliter (ng/mL)
Standard Deviation 2170
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 8: M1 VX-661
4330 nanogram per milliliter (ng/mL)
Standard Deviation 1980
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 8: IVA
747 nanogram per milliliter (ng/mL)
Standard Deviation 745
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 8: M1-IVA
1370 nanogram per milliliter (ng/mL)
Standard Deviation 1070
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 12: VX-661
1700 nanogram per milliliter (ng/mL)
Standard Deviation 1220
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 12: M1 VX-661
4290 nanogram per milliliter (ng/mL)
Standard Deviation 1730
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 12: IVA
776 nanogram per milliliter (ng/mL)
Standard Deviation 690
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Week 12: M1-IVA
1620 nanogram per milliliter (ng/mL)
Standard Deviation 1160

Adverse Events

Placebo

Serious events: 14 serious events
Other events: 63 other events
Deaths: 0 deaths

VX-661/IVA

Serious events: 11 serious events
Other events: 62 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Placebo
n=85 participants at risk
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=83 participants at risk
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Investigations
Pulmonary function test decreased
1.2%
1/85 • Baseline up to Week 16
0.00%
0/83 • Baseline up to Week 16
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma of colon
0.00%
0/85 • Baseline up to Week 16
1.2%
1/83 • Baseline up to Week 16
Respiratory, thoracic and mediastinal disorders
Haemoptysis
1.2%
1/85 • Baseline up to Week 16
2.4%
2/83 • Baseline up to Week 16
Respiratory, thoracic and mediastinal disorders
Productive cough
1.2%
1/85 • Baseline up to Week 16
0.00%
0/83 • Baseline up to Week 16
Gastrointestinal disorders
Distal intestinal obstruction syndrome
0.00%
0/85 • Baseline up to Week 16
3.6%
3/83 • Baseline up to Week 16
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
14.1%
12/85 • Baseline up to Week 16
7.2%
6/83 • Baseline up to Week 16
Infections and infestations
Lower respiratory tract infection bacterial
0.00%
0/85 • Baseline up to Week 16
1.2%
1/83 • Baseline up to Week 16
Infections and infestations
Influenza
1.2%
1/85 • Baseline up to Week 16
0.00%
0/83 • Baseline up to Week 16
Infections and infestations
Sinusitis
1.2%
1/85 • Baseline up to Week 16
0.00%
0/83 • Baseline up to Week 16

Other adverse events

Other adverse events
Measure
Placebo
n=85 participants at risk
Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
VX-661/IVA
n=83 participants at risk
VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Respiratory, thoracic and mediastinal disorders
Cough
31.8%
27/85 • Baseline up to Week 16
22.9%
19/83 • Baseline up to Week 16
Respiratory, thoracic and mediastinal disorders
Haemoptysis
7.1%
6/85 • Baseline up to Week 16
9.6%
8/83 • Baseline up to Week 16
Respiratory, thoracic and mediastinal disorders
Nasal congestion
9.4%
8/85 • Baseline up to Week 16
8.4%
7/83 • Baseline up to Week 16
Respiratory, thoracic and mediastinal disorders
Sputum increased
14.1%
12/85 • Baseline up to Week 16
8.4%
7/83 • Baseline up to Week 16
Respiratory, thoracic and mediastinal disorders
Dyspnoea
8.2%
7/85 • Baseline up to Week 16
6.0%
5/83 • Baseline up to Week 16
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
7.1%
6/85 • Baseline up to Week 16
6.0%
5/83 • Baseline up to Week 16
Nervous system disorders
Headache
8.2%
7/85 • Baseline up to Week 16
6.0%
5/83 • Baseline up to Week 16
General disorders
Fatigue
4.7%
4/85 • Baseline up to Week 16
12.0%
10/83 • Baseline up to Week 16
General disorders
Pyrexia
5.9%
5/85 • Baseline up to Week 16
6.0%
5/83 • Baseline up to Week 16
Gastrointestinal disorders
Nausea
7.1%
6/85 • Baseline up to Week 16
7.2%
6/83 • Baseline up to Week 16
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
17.6%
15/85 • Baseline up to Week 16
20.5%
17/83 • Baseline up to Week 16
Infections and infestations
Nasopharyngitis
5.9%
5/85 • Baseline up to Week 16
2.4%
2/83 • Baseline up to Week 16

Additional Information

Medical Monitor

Vertex Pharmaceuticals Incorporated

Phone: 617-341-6777

Results disclosure agreements

  • Principal investigator is a sponsor employee Restriction Description: PI is free to publish results of the study after (1)first multi-center publication, (2)if 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
  • Publication restrictions are in place

Restriction type: OTHER