Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02797132
Recruitment Status : Completed
First Posted : June 13, 2016
Results First Posted : October 30, 2018
Last Update Posted : October 30, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Intervention Drug: LUM/IVA
Enrollment 62
Recruitment Details The study was conducted in 2 parts, Part A and Part B. In Parts A and B, participants received doses of lumacaftor/ivacaftor (LUM/IVA) based on weight. Participants from Part A may have also participated in Part B.
Pre-assignment Details  
Arm/Group Title Lumacaftor/Ivacaftor (LUM/IVA)
Hide Arm/Group Description

Part A (<14 kg): Participants weighing less than (<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A.

Part A (>=14 kg): Participants weighing greater than or equal to (>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A.

Part B (<14 kg): Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.

Part B (>=14 kg): Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.

Period Title: Part A (15 Days)
Started 12
Part A (<14 kg Weight) 4
Part A (>=14 kg Weight) 8
Completed 11
Not Completed 1
Reason Not Completed
Adverse Event             1
Period Title: Part B (24 Weeks)
Started 60 [1]
Part B (<14 kg Weight) 19
Part B (>=14 kg Weight) 41
Completed 57
Not Completed 3
Reason Not Completed
Adverse Event             3
[1]
Participants from Part A may have participated in Part B.
Arm/Group Title Lumacaftor/Ivacaftor (LUM/IVA)
Hide Arm/Group Description

Part A (<14 kg): Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A.

Part A (>=14 kg): Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A.

Part B (<14 kg): Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.

Part B (>=14 kg): Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.

Overall Number of Baseline Participants 62
Hide Baseline Analysis Population Description
The Safety Set included all participants who received at least 1 dose of LUM/IVA in the respective study part.
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Months
Part A (<14 kg Weight) Number Analyzed 4 participants
27.0  (6.00)
Part A (>=14 kg Weight) Number Analyzed 8 participants
48.0  (11.11)
Part B (<14 kg Weight) Number Analyzed 19 participants
31.6  (5.05)
Part B (>=14 kg Weight) Number Analyzed 41 participants
49.9  (10.63)
[1]
Measure Analysis Population Description: Data were planned to be reported separately for Part A and Part B of the study. Here “number analyzed” signifies participants who were evaluable for specified part of the study.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Part A (<14 kg Weight) Number Analyzed 4 participants
Female
2
  50.0%
Male
2
  50.0%
Part A (>=14 kg Weight) Number Analyzed 8 participants
Female
2
  25.0%
Male
6
  75.0%
Part B (<14 kg Weight) Number Analyzed 19 participants
Female
9
  47.4%
Male
10
  52.6%
Part B (>=14 kg Weight) Number Analyzed 41 participants
Female
20
  48.8%
Male
21
  51.2%
[1]
Measure Analysis Population Description: Data were planned to be reported separately for Part A and Part B of the study. Here “number analyzed” signifies participants who were evaluable for specified part of the study.
Ethnicity (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Part A (<14 kg Weight) Number Analyzed 4 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
4
 100.0%
Unknown or Not Reported
0
   0.0%
Part A (>=14 kg Weight) Number Analyzed 8 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
8
 100.0%
Unknown or Not Reported
0
   0.0%
Part B (<14 kg Weight) Number Analyzed 19 participants
Hispanic or Latino
1
   5.3%
Not Hispanic or Latino
18
  94.7%
Unknown or Not Reported
0
   0.0%
Part B (>=14 kg Weight) Number Analyzed 41 participants
Hispanic or Latino
2
   4.9%
Not Hispanic or Latino
39
  95.1%
Unknown or Not Reported
0
   0.0%
[1]
Measure Analysis Population Description: Data were planned to be reported separately for Part A and Part B of the study. Here “number analyzed” signifies participants who were evaluable for specified part of the study.
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Part A (<14 kg Weight) Number Analyzed 4 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
4
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Part A (>=14 kg Weight) Number Analyzed 8 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
8
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Part B (<14 kg Weight) Number Analyzed 19 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
18
  94.7%
More than one race
0
   0.0%
Unknown or Not Reported
1
   5.3%
Part B (>=14 kg Weight) Number Analyzed 41 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
41
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
[1]
Measure Analysis Population Description: Data were planned to be reported separately for Part A and Part B of the study. Here “number analyzed” signifies participants who were evaluable for specified part of the study.
1.Primary Outcome
Title Part A: Pre-dose Concentration (Ctrough) of LUM and IVA
Hide Description [Not Specified]
Time Frame Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic (PK) set for Part A included all participants who received at least 1 dose of LUM/IVA in Part A.
Arm/Group Title Part A (<14 kg Weight): LUM 100 mg/IVA 125 mg Part A (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A.
Overall Number of Participants Analyzed 4 7
Mean (Standard Deviation)
Unit of Measure: nanogram per milliliter (ng/mL)
LUM 8710  (3590) 12300  (5960)
IVA 94.0  (67.0) 216  (185)
2.Primary Outcome
Title Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description [Not Specified]
Time Frame Day 1 up to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all participants who received at least 1 dose of LUM/IVA in Part B.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 41
Measure Type: Count of Participants
Unit of Measure: Participants
AEs
19
 100.0%
40
  97.6%
SAEs
2
  10.5%
2
   4.9%
3.Secondary Outcome
Title Part A: Pre-dose Concentration (Ctrough) of LUM and IVA Metabolites
Hide Description [Not Specified]
Time Frame Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
The PK set for Part A included all participants who received at least 1 dose of LUM/IVA in Part A.
Arm/Group Title Part A (<14 kg Weight): LUM 100 mg/IVA 125 mg Part A (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A.
Overall Number of Participants Analyzed 4 7
Mean (Standard Deviation)
Unit of Measure: ng/mL
LUM Metabolite (M28 LUM) 1310  (590) 1370  (286)
IVA Metabolite (M1 IVA) 475  (384) 1240  (1180)
IVA Metabolite (M6 IVA) 1510  (1130) 3270  (2100)
4.Secondary Outcome
Title Part A: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description [Not Specified]
Time Frame Day 1 up to Day 25
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all participants who received at least 1 dose of LUM/IVA in Part A.
Arm/Group Title Part A (<14 kg Weight): LUM 100 mg/IVA 125 mg Part A (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A.
Overall Number of Participants Analyzed 4 8
Measure Type: Count of Participants
Unit of Measure: Participants
AEs
4
 100.0%
6
  75.0%
SAEs
0
   0.0%
0
   0.0%
5.Secondary Outcome
Title Part B: Absolute Change From Baseline in Sweat Chloride at Week 24
Hide Description Sweat samples were collected using an approved collection device.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set (FAS) included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 18 31
Mean (Standard Deviation)
Unit of Measure: millimole per liter (mmol/L)
-33.5  (14.5) -30.7  (14.0)
6.Secondary Outcome
Title Part B: Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
Hide Description BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2).
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 38
Mean (Standard Deviation)
Unit of Measure: Kilogram per meter square (kg/m^2)
0.22  (0.78) 0.29  (0.75)
7.Secondary Outcome
Title Part B: Absolute Change From Baseline in Body Mass Index (BMI) For-Age Z-Score at Week 24
Hide Description BMI was defined as weight in kg divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score (BMI z-score).
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 38
Mean (Standard Deviation)
Unit of Measure: Z-score
0.36  (0.67) 0.26  (0.53)
8.Secondary Outcome
Title Part B: Absolute Change From Baseline in Weight at Week 24
Hide Description [Not Specified]
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 38
Mean (Standard Deviation)
Unit of Measure: Kilogram (kg)
1.4  (0.7) 1.5  (1.0)
9.Secondary Outcome
Title Part B: Absolute Change From Baseline in Weight-for-age Z-Score at Week 24
Hide Description z-score is a statistical measure to describe whether a mean was above or below the standard. Weight, adjusted for age and sex, was analyzed as weight-for-age z-score (Weight z-score).
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled subjects in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 38
Mean (Standard Deviation)
Unit of Measure: Z-score
0.44  (0.52) 0.17  (0.36)
10.Secondary Outcome
Title Part B: Absolute Change From Baseline in Stature (Height) at Week 24
Hide Description [Not Specified]
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 38
Mean (Standard Deviation)
Unit of Measure: Centimeter (cm)
4.1  (1.4) 3.4  (1.0)
11.Secondary Outcome
Title Part B: Absolute Change From Baseline in Stature-for-Age Z-Score
Hide Description z-score is a statistical measure to describe whether a mean was above or below the standard. Stature (height), adjusted for age and sex, was analyzed as Stature-for-age z-score (Stature z-score).
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 38
Mean (Standard Deviation)
Unit of Measure: Z-score
0.19  (0.32) 0.04  (0.19)
12.Secondary Outcome
Title Part B: Number of Pulmonary Exacerbations
Hide Description Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol defined criteria.
Time Frame Through Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 41
Measure Type: Number
Unit of Measure: pulmonary exacerbations
5 20
13.Secondary Outcome
Title Part B: Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24
Hide Description Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol defined criteria. Time to event data was not collected and instead, number of participants with first event were collected and are reported. 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.
Time Frame Through Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 41
Measure Type: Count of Participants
Unit of Measure: Participants
3
  15.8%
15
  36.6%
14.Secondary Outcome
Title Part B: Number of Cystic Fibrosis (CF)-Related Hospitalizations
Hide Description [Not Specified]
Time Frame Through Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 41
Measure Type: Number
Unit of Measure: hospitalizations
1 3
15.Secondary Outcome
Title Part B: Absolute Change From Baseline in Fecal Elastase-1 (FE-1) Levels at Week 24
Hide Description [Not Specified]
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled subjects in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 12 23
Mean (Standard Deviation)
Unit of Measure: microgram per gram
38.4  (76.1) 60.0  (94.0)
16.Secondary Outcome
Title Part B: Absolute Change From Baseline in Serum Levels of Immunoreactive Trypsinogen (IRT) Through Week 24
Hide Description [Not Specified]
Time Frame Baseline, Through Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing less than 14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 17 38
Mean (Standard Deviation)
Unit of Measure: ng/mL
-262.1  (343.1) -71.1  (120.5)
17.Secondary Outcome
Title Part B: Number of Participants With Microbiology Culture Status (Positive or Negative) at Week 24
Hide Description Following microbial tests were performed: Burkholderia, Methicillin Resistant Staphylococcus Aureus (MRSA), Methicillin Susceptible Staphylococcus Aureus (MSSA), Pseudomonas Aeruginosa Mucoid (P. Aeruginosa Mucoid), P. Aeruginosa Non-Mucoid, P. Aeruginosa Small Colony Variant and Stenotrophomonas Maltophilia.
Time Frame Baseline and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS analysis set was used. Here "Number Analyzed" signifies those participants who were evaluated for this outcome at the specified time point..
Arm/Group Title Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing less than <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 41
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline: Burkholderia Number Analyzed 18 participants 38 participants
Negative
18
 100.0%
37
  97.4%
Positive
0
   0.0%
1
   2.6%
Week 24: Burkholderia Number Analyzed 19 participants 36 participants
Negative
19
 100.0%
36
 100.0%
Positive
0
   0.0%
0
   0.0%
Baseline: MRSA Number Analyzed 18 participants 38 participants
Negative
16
  88.9%
33
  86.8%
Positive
2
  11.1%
5
  13.2%
Week 24:MRSA Number Analyzed 19 participants 36 participants
Negative
17
  89.5%
35
  97.2%
Positive
2
  10.5%
1
   2.8%
Baseline: MSSA Number Analyzed 18 participants 38 participants
Negative
12
  66.7%
26
  68.4%
Positive
6
  33.3%
12
  31.6%
Week 24: MSSA Number Analyzed 19 participants 36 participants
Negative
13
  68.4%
26
  72.2%
Positive
6
  31.6%
10
  27.8%
Baseline: P. Aeruginosa Mucoid Number Analyzed 18 participants 38 participants
Negative
18
 100.0%
37
  97.4%
Positive
0
   0.0%
1
   2.6%
Week 24: P. Aeruginosa Mucoid Number Analyzed 19 participants 36 participants
Negative
19
 100.0%
35
  97.2%
Positive
0
   0.0%
1
   2.8%
Baseline: P. Aeruginosa Non-Mucoid Number Analyzed 18 participants 38 participants
Negative
17
  94.4%
35
  92.1%
Positive
1
   5.6%
3
   7.9%
Week 24: P. Aeruginosa Non-Mucoid Number Analyzed 19 participants 36 participants
Negative
16
  84.2%
34
  94.4%
Positive
3
  15.8%
2
   5.6%
Baseline: P. Aeruginosa Small Colony Variant Number Analyzed 18 participants 38 participants
Negative
18
 100.0%
38
 100.0%
Positive
0
   0.0%
0
   0.0%
Week 24: P. Aeruginosa Small Colony Variant Number Analyzed 19 participants 36 participants
Negative
19
 100.0%
36
 100.0%
Positive
0
   0.0%
0
   0.0%
Baseline: Stenotrophomonas Maltophilia Number Analyzed 18 participants 38 participants
Negative
17
  94.4%
37
  97.4%
Positive
1
   5.6%
1
   2.6%
Week 24: Stenotrophomonas Maltophilia Number Analyzed 19 participants 36 participants
Negative
18
  94.7%
36
 100.0%
Positive
1
   5.3%
0
   0.0%
18.Secondary Outcome
Title Part B: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
Hide Description FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population: participants >=3 years of age with data available for Baseline and Week 24. Only participants from "Part B (>=14 Kg Weight)" arm met the eligibility criteria for the specified time point and were included in the analysis.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 0 12
Mean (Standard Deviation)
Unit of Measure: percentage of predicted FEV1
0.5  (11.6)
19.Secondary Outcome
Title Part B: Absolute Change in Sweat Chloride From Week 24 at Week 26
Hide Description Sweat samples were collected using an approved collection device.
Time Frame Week 24, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled subjects in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 17 31
Mean (Standard Deviation)
Unit of Measure: mmol/L
34.4  (14.9) 32.2  (13.8)
20.Secondary Outcome
Title Part B: Acceptability/Palatability of LUM/IVA Granules Measured Using Hedonic Scale
Hide Description The acceptability and palatability of LUM/IVA granules was assessed by a visual analog scale that incorporates a 5 point facial hedonic scale (Liked it Very Much, Liked it a Little, Not sure, Disliked it a Little, Disliked it Very Much). The assessment was conducted in 2 steps: assessment of approved food/liquid (Evaluation 1), and assessment of approved food/liquid with LUM/IVA granules (Evaluation 2).
Time Frame Day 1
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS included all enrolled subjects in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 18 38
Measure Type: Count of Participants
Unit of Measure: Participants
Evaluation 1 Liked it Very Much
9
  50.0%
30
  78.9%
Liked it a Little
4
  22.2%
6
  15.8%
Not sure
3
  16.7%
1
   2.6%
Disliked it a Little
0
   0.0%
0
   0.0%
Disliked it Very Much
2
  11.1%
1
   2.6%
Evaluation 2 Liked it Very Much
4
  22.2%
6
  15.8%
Liked it a Little
2
  11.1%
5
  13.2%
Not sure
4
  22.2%
6
  15.8%
Disliked it a Little
3
  16.7%
6
  15.8%
Disliked it Very Much
5
  27.8%
15
  39.5%
21.Secondary Outcome
Title Part B: Absolute Change From Baseline in Lung Clearance Index (LCI) 2.5 at Week 24
Hide Description Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The LCI Substudy Set included all participants who had signed informed consent (and assent, if applicable) to the optional LCI Substudy in Part B and enrolled and dosed in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 3 14
Mean (Standard Deviation)
Unit of Measure: ratio
0.27  (0.48) -0.76  (1.19)
22.Secondary Outcome
Title Part B: Absolute Change From Baseline in Lung Clearance Index (LCI) 5.0 at Week 24
Hide Description LCI is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The LCI Substudy Set included all subjects who had signed informed consent (and assent, if applicable) to the optional LCI Substudy in Part B and enrolled and dosed in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 3 14
Mean (Standard Deviation)
Unit of Measure: ratio
0.24  (0.40) -0.12  (0.69)
23.Secondary Outcome
Title Part B: Pre-dose Concentration (Ctrough) of LUM and IVA and Its Metabolites
Hide Description [Not Specified]
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The PK set for Part B included all participants who received at least 1 dose of LUM/IVA in Part B. Here "Number Analyzed" signifies those participants who were evaluable for the specified category.
Arm/Group Title Part B (<14 kg Weight): LUM 100 mg/IVA 125 mg Part B (>=14 kg Weight): LUM 150 mg/IVA 188 mg
Hide Arm/Group Description:
Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.
Overall Number of Participants Analyzed 19 41
Mean (Standard Deviation)
Unit of Measure: ng/mL
LUM Number Analyzed 18 participants 36 participants
9060  (4060) 9390  (4830)
LUM Metabolite (M28 LUM) Number Analyzed 19 participants 36 participants
1420  (846) 1510  (752)
IVA Number Analyzed 18 participants 35 participants
108  (109) 110  (108)
IVA Metabolite (M1 IVA) Number Analyzed 18 participants 36 participants
555  (560) 521  (478)
IVA Metabolite (M6 IVA) Number Analyzed 18 participants 36 participants
2050  (1940) 1900  (1430)
Time Frame Part A: Day 1 up to Day 25; Part B: Day 1 up to Week 26
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Part A: LUM 100 mg/IVA 125 mg Part A: LUM 150 mg/IVA 188 mg Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg
Hide Arm/Group Description Participants weighing <14 kg at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A. Participants weighing >= 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A. Participants weighing less than <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B. Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hurs for 24 weeks in Part B.
All-Cause Mortality
Part A: LUM 100 mg/IVA 125 mg Part A: LUM 150 mg/IVA 188 mg Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/8 (0.00%)   0/19 (0.00%)   0/41 (0.00%) 
Hide Serious Adverse Events
Part A: LUM 100 mg/IVA 125 mg Part A: LUM 150 mg/IVA 188 mg Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/8 (0.00%)   2/19 (10.53%)   2/41 (4.88%) 
Gastrointestinal disorders         
Constipation  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Infections and infestations         
Infective pulmonary exacerbation of cystic fibrosis  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  2/41 (4.88%) 
Gastroenteritis viral  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
1
Term from vocabulary, MedDRA 20.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Part A: LUM 100 mg/IVA 125 mg Part A: LUM 150 mg/IVA 188 mg Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   4/4 (100.00%)   6/8 (75.00%)   19/19 (100.00%)   40/41 (97.56%) 
Blood and lymphatic system disorders         
Anaemia  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Lymphocytosis  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Cardiac disorders         
Tachycardia  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Ventricular extrasystoles  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Endocrine disorders         
Hypothyroidism  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  1/41 (2.44%) 
Eye disorders         
Eye irritation  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  1/41 (2.44%) 
Lacrimation increased  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Gastrointestinal disorders         
Faeces discoloured  1  1/4 (25.00%)  0/8 (0.00%)  0/19 (0.00%)  0/41 (0.00%) 
Faeces soft  1  2/4 (50.00%)  0/8 (0.00%)  0/19 (0.00%)  0/41 (0.00%) 
Flatulence  1  1/4 (25.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Vomiting  1  2/4 (50.00%)  0/8 (0.00%)  6/19 (31.58%)  11/41 (26.83%) 
Constipation  1  0/4 (0.00%)  0/8 (0.00%)  3/19 (15.79%)  4/41 (9.76%) 
Diarrhoea  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  4/41 (9.76%) 
Abdominal pain  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  3/41 (7.32%) 
Nausea  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  2/41 (4.88%) 
Abdominal pain upper  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Haematochezia  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  1/41 (2.44%) 
Oral discomfort  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Oral pain  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Post-tussive vomiting  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Steatorrhoea  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  1/41 (2.44%) 
Dyschezia  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Frequent bowel movements  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Gastrooesophageal reflux disease  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
General disorders         
Pyrexia  1  0/4 (0.00%)  0/8 (0.00%)  7/19 (36.84%)  10/41 (24.39%) 
Fatigue  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  2/41 (4.88%) 
Chills  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  1/41 (2.44%) 
Discomfort  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Pain  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Asthenia  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Hepatobiliary disorders         
Hepatomegaly  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Immune system disorders         
Seasonal allergy  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Hypersensitivity  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Infections and infestations         
Infective pulmonary exacerbation of cystic fibrosis  1  0/4 (0.00%)  1/8 (12.50%)  0/19 (0.00%)  2/41 (4.88%) 
Lice infestation  1  0/4 (0.00%)  1/8 (12.50%)  0/19 (0.00%)  0/41 (0.00%) 
Lower respiratory tract infection viral  1  0/4 (0.00%)  1/8 (12.50%)  0/19 (0.00%)  0/41 (0.00%) 
Upper respiratory tract infection  1  0/4 (0.00%)  0/8 (0.00%)  3/19 (15.79%)  7/41 (17.07%) 
Ear infection  1  0/4 (0.00%)  0/8 (0.00%)  3/19 (15.79%)  4/41 (9.76%) 
Otitis media  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  4/41 (9.76%) 
Sinusitis  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  3/41 (7.32%) 
Conjunctivitis  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  2/41 (4.88%) 
Rhinitis  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  2/41 (4.88%) 
Viral upper respiratory tract infection  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  2/41 (4.88%) 
Impetigo  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Cellulitis  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Labyrinthitis  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Pharyngitis streptococcal  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  1/41 (2.44%) 
Viral rash  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Vulvovaginal mycotic infection  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Candida nappy rash  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Gastroenteritis rotavirus  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Hand-foot-and-mouth disease  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  0/41 (0.00%) 
Influenza  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Oral candidiasis  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Injury, poisoning and procedural complications         
Joint dislocation  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Radial head dislocation  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Investigations         
Respiratory rate increased  1  0/4 (0.00%)  1/8 (12.50%)  0/19 (0.00%)  0/41 (0.00%) 
Alanine aminotransferase increased  1  0/4 (0.00%)  0/8 (0.00%)  3/19 (15.79%)  5/41 (12.20%) 
Aspartate aminotransferase increased  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  4/41 (9.76%) 
Forced expiratory volume decreased  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  2/41 (4.88%) 
Pseudomonas test positive  1  0/4 (0.00%)  0/8 (0.00%)  3/19 (15.79%)  2/41 (4.88%) 
Activated partial thromboplastin time prolonged  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Burkholderia test positive  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Gamma-glutamyltransferase increased  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  1/41 (2.44%) 
International normalised ratio increased  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Prothrombin time prolonged  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Pulmonary function test decreased  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Staphylococcus test positive  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Streptococcus test positive  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Bacterial test positive  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Blood creatine phosphokinase increased  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  0/41 (0.00%) 
Blood iron decreased  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Blood creatinine increased  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Enterovirus test positive  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Respirovirus test positive  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Vitamin D decreased  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Metabolism and nutrition disorders         
Decreased appetite  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  2/41 (4.88%) 
Dehydration  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  1/41 (2.44%) 
Musculoskeletal and connective tissue disorders         
Arthralgia  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Musculoskeletal pain  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Nervous system disorders         
Headache  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  4/41 (9.76%) 
Cognitive disorder  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Lethargy  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Psychiatric disorders         
Enuresis  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Sleep terror  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  0/41 (0.00%) 
Tearfulness  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  0/41 (0.00%) 
Renal and urinary disorders         
Urinary incontinence  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Respiratory, thoracic and mediastinal disorders         
Cough  1  2/4 (50.00%)  3/8 (37.50%)  12/19 (63.16%)  26/41 (63.41%) 
Rhinorrhoea  1  0/4 (0.00%)  2/8 (25.00%)  8/19 (42.11%)  7/41 (17.07%) 
Nasal congestion  1  1/4 (25.00%)  0/8 (0.00%)  4/19 (21.05%)  6/41 (14.63%) 
Oropharyngeal pain  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  3/41 (7.32%) 
Dyspnoea  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  2/41 (4.88%) 
Nasal discharge discolouration  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  2/41 (4.88%) 
Productive cough  1  0/4 (0.00%)  0/8 (0.00%)  2/19 (10.53%)  2/41 (4.88%) 
Wheezing  1  0/4 (0.00%)  0/8 (0.00%)  1/19 (5.26%)  2/41 (4.88%) 
Epistaxis  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Respiration abnormal  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Sinus congestion  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Sputum discoloured  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Tonsillar inflammation  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Upper respiratory tract congestion  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Skin and subcutaneous tissue disorders         
Eczema  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Rash  1  0/4 (0.00%)  0/8 (0.00%)  3/19 (15.79%)  1/41 (2.44%) 
Urticaria  1  0/4 (0.00%)  0/8 (0.00%)  0/19 (0.00%)  1/41 (2.44%) 
Hyperhidrosis  1  1/4 (25.00%)  0/8 (0.00%)  0/19 (0.00%)  0/41 (0.00%) 
1
Term from vocabulary, MedDRA 20.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Monitor
Organization: Vertex Pharmaceuticals Incorporated
Phone: 617-341-6777
EMail: medicalinfo@vrtx.com
Layout table for additonal information
Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02797132    
Other Study ID Numbers: VX15-809-115
2016-001004-33 ( EudraCT Number )
First Submitted: May 25, 2016
First Posted: June 13, 2016
Results First Submitted: September 30, 2018
Results First Posted: October 30, 2018
Last Update Posted: October 30, 2018