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Safety, Pharmacokinetic (PK) and Pharmacodynamic (PD) Study of Repeat Doses of Inhaled Nemiralisib in Patients With APDS/PASLI

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: NCT02593539
Recruitment Status : Completed
First Posted : November 1, 2015
Results First Posted : June 18, 2021
Last Update Posted : June 18, 2021
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Activated PI3K-delta Syndrome
Intervention Drug: Nemiralisib
Enrollment 5
Recruitment Details This is an open-label study conducted to investigate safety, pharmacokinetics and pharmacodynamics of repeat doses of inhaled nemiralisib (NEMI) in participants with activated phosphoinositide 3-kinase (PI3K) delta syndrome /p110 delta-activating mutation causing senescent T Cells, lymphadenopathy and immunodeficiency (APDS/PASLI)
Pre-assignment Details Participants received either 1000 mcg NEMI DISKUS or 700 mcg NEMI ELLIPTA or 500 mcg NEMI ELLIPTA. All NEMI DISKUS and NEMI ELLIPTA dose levels were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices. The study had protocol amendments to reflect changes in dose and device administration.
Arm/Group Title All NEMI
Hide Arm/Group Description Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Period Title: Overall Study
Started 5
Completed 4
Not Completed 1
Reason Not Completed
Adverse Event             1
Arm/Group Title All NEMI
Hide Arm/Group Description Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Baseline Participants 5
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 5 participants
36.6  (12.36)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Female
3
  60.0%
Male
2
  40.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
White-White/Caucasian/European Heritage
5
 100.0%
1.Primary Outcome
Title Number of Participants With Any Serious Adverse Events (SAEs) and Any Non-serious Adverse Events (Non-SAEs)
Hide Description An adverse event is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is defined as any untoward medical occurrence that; results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, is a congenital anomaly/birth defect, other situations as judged by physician. Number of participants with any SAE and non-SAEs are presented.
Time Frame Upto 7.5 months
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population consisted of all participants who received at least one dose of the study treatment.
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Measure Type: Count of Participants
Unit of Measure: Participants
Any non-SAE
5
 100.0%
Any SAE
0
   0.0%
2.Primary Outcome
Title Change From Baseline in Diastolic Blood Pressure (DBP) and Systolic Blood Pressure (SBP)
Hide Description SBP and DBP were measured in participants in a semi-supine position after 5 minutes rest. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Not applicable (NA) indicates that standard deviation could not be calculated as a single participant was analyzed.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Millimeters of Mercury (mmHg)
DBP, Day 14, n= 5 Number Analyzed 5 participants
1.0  (6.44)
DBP, Day 28, n= 4 Number Analyzed 4 participants
4.0  (6.98)
DBP, Day 56, n= 4 Number Analyzed 4 participants
8.5  (5.80)
DBP, Day 83, n= 4 Number Analyzed 4 participants
2.0  (4.40)
DBP, Day 84 n= 1 Number Analyzed 1 participants
17.0 [1]   (NA)
SBP, Day 14, n= 5 Number Analyzed 5 participants
-1.0  (3.67)
SBP, Day 28, n= 4 Number Analyzed 4 participants
5.3  (8.54)
SBP, Day 56, n= 4 Number Analyzed 4 participants
10.0  (9.31)
SBP, Day 83, n= 4 Number Analyzed 4 participants
1.5  (3.51)
SBP, Day 84, n= 1 Number Analyzed 1 participants
16.0 [1]   (NA)
[1]
NA indicates that standard deviation could not be calculated as a single participant was analyzed.
3.Primary Outcome
Title Change From Baseline in Pulse Rate
Hide Description Pulse rate was measured in participants in a semi-supine position after 5 minutes rest. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. NA indicates that standard deviation could not be calculated as a single participant was analyzed.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Beats per minute
Day 14, n= 5 Number Analyzed 5 participants
3.2  (7.73)
Day 28, n= 4 Number Analyzed 4 participants
2.0  (10.86)
Day 56, n= 4 Number Analyzed 4 participants
6.8  (9.43)
Day 83, n= 4 Number Analyzed 4 participants
4.8  (7.76)
Day 84 n= 1 Number Analyzed 1 participants
7.0 [1]   (NA)
[1]
NA indicates that standard deviation could not be calculated as a single participant was analyzed.
4.Primary Outcome
Title Change From Baseline in Respiratory Rate
Hide Description Respiratory rate was measured in participants in a semi-supine position after 5 minutes rest. . Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. NA indicates that standard deviation could not be calculated as a single participant was analyzed.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Breaths per minute
Day 14, n= 5 Number Analyzed 5 participants
0.2  (2.28)
Day 28, n= 4 Number Analyzed 4 participants
0.8  (4.57)
Day 56, n= 4 Number Analyzed 4 participants
1.5  (5.26)
Day 83, n= 4 Number Analyzed 4 participants
1.0  (2.45)
Day 84 n= 1 Number Analyzed 1 participants
2.0 [1]   (NA)
[1]
NA indicates that standard deviation could not be calculated as a single participant was analyzed.
5.Primary Outcome
Title Change From Baseline in Body Temperature
Hide Description Temperature was measured in participants in a semi-supine position after 5 minutes rest. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. NA indicates that standard deviation could not be calculated as a single participant was analyzed.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Degrees celsius
Day 14, n= 5 Number Analyzed 5 participants
0.10  (0.158)
Day 28, n= 4 Number Analyzed 4 participants
0.17  (0.350)
Day 56, n= 4 Number Analyzed 4 participants
0.07  (0.171)
Day 83, n= 4 Number Analyzed 4 participants
0.17  (0.479)
Day 84 n= 1 Number Analyzed 1 participants
0.50 [1]   (NA)
[1]
NA indicates that standard deviation could not be calculated as a single participant was analyzed.
6.Primary Outcome
Title Change From Baseline in Electrocardiogram (ECG) Mean Heart Rate
Hide Description Single 12-lead ECGs were recorded at indicated timepoints using an ECG machine that automatically calculated the heart rate. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Beats per minute
Day 14, n= 5 Number Analyzed 5 participants
-1.0  (3.94)
Day 28, n= 4 Number Analyzed 4 participants
-0.8  (2.99)
Day 56, n= 4 Number Analyzed 4 participants
5.3  (7.54)
Day 83, n= 4 Number Analyzed 4 participants
1.0  (2.31)
7.Primary Outcome
Title Change From Baseline in PR Interval, QRS Duration, Uncorrected QT Interval, QT Corrected Interval-Fredericia Interval (QTcF) and QTc Corrected by Bazett's Formula (QTcB)
Hide Description Twelve lead ECGs were recorded at indicated timepoints. At each time point, ECG machine automatically measured QRS duration, uncorrected QT interval, QTcF interval and QTcB. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Milliseconds
PR, Day 14, n= 5 Number Analyzed 5 participants
-2.2  (9.23)
PR, Day 28, n= 4 Number Analyzed 4 participants
-4.8  (4.27)
PR, Day 56, n= 4 Number Analyzed 4 participants
-3.3  (11.18)
PR, Day 83, n= 4 Number Analyzed 4 participants
-6.0  (11.80)
QRS Duration, Day 14, n= 5 Number Analyzed 5 participants
-5.6  (7.33)
QRS Duration, Day 28, n= 4 Number Analyzed 4 participants
-4.5  (11.03)
QRS Duration, Day 56, n= 4 Number Analyzed 4 participants
-6.5  (8.81)
QRS Duration, Day 83, n= 4 Number Analyzed 4 participants
-6.0  (7.79)
QT Interval, Day 14, n= 5 Number Analyzed 5 participants
-7.2  (10.66)
QT Interval, Day 28, n= 4 Number Analyzed 4 participants
-2.8  (28.89)
QT Interval, Day 56, n= 4 Number Analyzed 4 participants
-22.3  (16.01)
QT Interval, Day 83, n= 4 Number Analyzed 4 participants
-16.3  (9.74)
QTcB, Day 14, n= 5 Number Analyzed 5 participants
-9.4  (4.93)
QTcB, Day 28, n= 4 Number Analyzed 4 participants
-4.5  (22.55)
QTcB, Day 56, n= 4 Number Analyzed 4 participants
-8.3  (24.55)
QTcB, Day 83, n= 4 Number Analyzed 4 participants
-12.5  (8.19)
QTcF, Day 14, n= 5 Number Analyzed 5 participants
-8.2  (4.32)
QTcF, Day 28, n= 4 Number Analyzed 4 participants
-3.5  (24.09)
QTcF, Day 56, n= 4 Number Analyzed 4 participants
-13.0  (19.92)
QTcF, Day 83, n= 4 Number Analyzed 4 participants
-14.0  (7.79)
8.Primary Outcome
Title Change From Baseline in Clinical Chemistry Parameters: Alanine Aminotransferase (ALT), Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST)
Hide Description Blood samples were collected for the analysis of clinical parameters including ALT, ALP and AST. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day -1) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: International units per liter (IU/L)
ALT, Day 14, n= 5 Number Analyzed 5 participants
1.0  (2.12)
ALT, Day 28, n= 4 Number Analyzed 4 participants
0.8  (1.71)
ALT, Day 56, n= 4 Number Analyzed 4 participants
0.0  (1.63)
ALT, Day 83, n= 4 Number Analyzed 4 participants
1.3  (7.50)
AST, Day 14, n= 5 Number Analyzed 5 participants
-0.8  (1.48)
AST, Day 28, n= 4 Number Analyzed 4 participants
0.8  (2.22)
AST, Day 56, n= 4 Number Analyzed 4 participants
-0.8  (4.79)
AST, Day 83, n= 4 Number Analyzed 4 participants
-1.0  (7.70)
ALP, Day 14, n= 5 Number Analyzed 5 participants
2.6  (5.77)
ALP, Day 28, n= 4 Number Analyzed 4 participants
3.8  (5.68)
ALP, Day 56, n= 4 Number Analyzed 4 participants
7.5  (5.00)
ALP, Day 83, n= 4 Number Analyzed 4 participants
6.0  (2.94)
9.Primary Outcome
Title Change From Baseline in Clinical Chemistry Parameters : Albumin and Total Protein
Hide Description Blood samples were collected for the analysis of clinical chemistry parameter-albumin and total protein. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day -1) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Grams per liter
Albumin, Day 14, n= 5 Number Analyzed 5 participants
2.6  (2.07)
Albumin, Day 28, n= 4 Number Analyzed 4 participants
1.3  (3.20)
Albumin, Day 56, n= 4 Number Analyzed 4 participants
2.8  (2.36)
Albumin, Day 83, n= 4 Number Analyzed 4 participants
2.0  (3.16)
Total Protein, Day 14, n= 5 Number Analyzed 5 participants
4.0  (1.58)
Total Protein, Day 28, n= 4 Number Analyzed 4 participants
1.3  (3.59)
Total Protein, Day 56, n= 4 Number Analyzed 4 participants
4.0  (4.76)
Total Protein, Day 83, n= 4 Number Analyzed 4 participants
4.3  (2.50)
10.Primary Outcome
Title Change From Baseline Values in Clinical Chemistry Parameters: Sodium, Potassium, Calcium, Glucose and Urea
Hide Description Blood samples were collected for the analysis of clinical parameters including sodium, potassium, calcium, glucose and urea. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day -1) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Millimoles per liter
Sodium, Day 14, n= 5 Number Analyzed 5 participants
-0.2  (2.68)
Sodium, Day 28, n= 4 Number Analyzed 4 participants
0.5  (0.58)
Sodium, Day 56, n= 4 Number Analyzed 4 participants
-0.8  (2.06)
Sodium, Day 83, n= 4 Number Analyzed 4 participants
0.0  (1.83)
Potassium, Day 14, n= 5 Number Analyzed 5 participants
0.24  (0.397)
Potassium, Day 28, n= 4 Number Analyzed 4 participants
0.30  (0.346)
Potassium, Day 56, n= 4 Number Analyzed 4 participants
0.35  (0.370)
Potassium, Day 83, n= 4 Number Analyzed 4 participants
0.38  (0.275)
Calcium, Day 14, n= 5 Number Analyzed 5 participants
0.040  (0.0990)
Calcium, Day 28, n= 4 Number Analyzed 4 participants
0.063  (0.0704)
Calcium, Day 56, n= 4 Number Analyzed 4 participants
0.133  (0.1343)
Calcium, Day 83, n= 4 Number Analyzed 4 participants
0.082  (0.1325)
Glucose, Day 14, n= 5 Number Analyzed 5 participants
-0.64  (1.557)
Glucose, Day 28, n= 4 Number Analyzed 4 participants
-0.60  (1.774)
Glucose, Day 56, n= 4 Number Analyzed 4 participants
-0.48  (1.578)
Glucose, Day 83, n= 4 Number Analyzed 4 participants
-0.68  (1.333)
Urea, Day 14, n= 5 Number Analyzed 5 participants
4.732  (5.8283)
Urea, Day 28, n= 4 Number Analyzed 4 participants
8.330  (2.6837)
Urea, Day 56, n= 4 Number Analyzed 4 participants
2.610  (5.1954)
Urea, Day 83, n= 4 Number Analyzed 4 participants
2.185  (4.1025)
11.Primary Outcome
Title Change From Baseline Values in Clinical Chemistry Parameters: Direct Bilirubin, Total Bilirubin and Creatinine
Hide Description Blood samples were collected for the analysis of clinical chemistry parameters: direct bilirubin, total bilirubin and creatinine. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day -1) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Micromoles per liter
Direct bilirubin, Day 14, n= 5 Number Analyzed 5 participants
-1.0  (0.71)
Direct bilirubin, Day 28, n= 4 Number Analyzed 4 participants
-1.8  (0.96)
Direct bilirubin, Day 56, n= 4 Number Analyzed 4 participants
-0.5  (1.00)
Direct bilirubin, Day 83, n= 4 Number Analyzed 4 participants
-0.5  (1.00)
Total bilirubin, Day 14, n= 5 Number Analyzed 5 participants
-1.8  (1.64)
Total bilirubin, Day 28, n= 4 Number Analyzed 4 participants
-3.0  (2.45)
Total bilirubin, Day 56, n= 4 Number Analyzed 4 participants
-0.8  (1.89)
Total bilirubin, Day 83, n= 4 Number Analyzed 4 participants
-1.5  (2.52)
Creatinine , Day 14, n= 5 Number Analyzed 5 participants
-2.0  (12.43)
Creatinine , Day 28, n= 4 Number Analyzed 4 participants
-4.3  (6.08)
Creatinine , Day 56, n= 4 Number Analyzed 4 participants
5.0  (14.07)
Creatinine , Day 83, n= 4 Number Analyzed 4 participants
-2.5  (14.93)
12.Primary Outcome
Title Change From Baseline Values in Clinical Chemistry Parameter: C-Reactive Protein
Hide Description Blood samples were collected for the analysis of clinical chemistry parameter:C-Reactive Protein. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Milligrams per liter
Day 14, n= 5 Number Analyzed 5 participants
0.56  (1.592)
Day 28, n= 4 Number Analyzed 4 participants
0.75  (1.085)
Day 56, n= 4 Number Analyzed 4 participants
2.75  (6.222)
Day 83, n= 4 Number Analyzed 4 participants
3.90  (7.141)
13.Primary Outcome
Title Change From Baseline for Hematology Parameters: Basophil, Eosinophils, White Blood Cells (WBC), Lymphocytes, Neutrophils, Monocytes and Platelets
Hide Description Blood samples were collected for the analysis of hematology parameters including basophils, eosinophils, WBC, lymphocytes, neutrophils, monocytes and platelets at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day -1) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: 10^9 cells per liters
Basophils, Day 14, n= 5 Number Analyzed 5 participants
-0.002  (0.0045)
Basophils, Day 28, n= 4 Number Analyzed 4 participants
0.008  (0.0096)
Basophils, Day 56, n= 4 Number Analyzed 4 participants
0.008  (0.0236)
Basophils, Day 83, n= 4 Number Analyzed 4 participants
0.013  (0.0222)
Eosinophils, Day 14, n= 5 Number Analyzed 5 participants
0.108  (0.1542)
Eosinophils, Day 28, n= 4 Number Analyzed 4 participants
0.088  (0.0650)
Eosinophils, Day 56, n= 4 Number Analyzed 4 participants
0.128  (0.2241)
Eosinophils, Day 83, n= 4 Number Analyzed 4 participants
0.075  (0.0810)
Lymphocytes, Day 14, n= 5 Number Analyzed 5 participants
0.122  (0.2411)
Lymphocytes, Day 28, n= 4 Number Analyzed 4 participants
-0.133  (0.0918)
Lymphocytes, Day 56, n= 4 Number Analyzed 4 participants
-0.105  (0.1115)
Lymphocytes, Day 83, n= 4 Number Analyzed 4 participants
0.037  (0.3140)
Monocytes, Day 14, n= 5 Number Analyzed 5 participants
0.096  (0.2893)
Monocytes, Day 28, n= 4 Number Analyzed 4 participants
0.030  (0.0572)
Monocytes, Day 56, n= 4 Number Analyzed 4 participants
0.115  (0.0614)
Monocytes, Day 83, n= 4 Number Analyzed 4 participants
0.065  (0.0592)
Neutrophils , Day 14, n= 5 Number Analyzed 5 participants
0.334  (1.2388)
Neutrophils , Day 28, n= 4 Number Analyzed 4 participants
0.575  (0.6125)
Neutrophils , Day 56, n= 4 Number Analyzed 4 participants
0.553  (0.7647)
Neutrophils , Day 83, n= 4 Number Analyzed 4 participants
0.653  (0.7857)
Platelet, Day 14, n= 5 Number Analyzed 5 participants
49.4  (52.53)
Platelet, Day 28, n= 4 Number Analyzed 4 participants
9.0  (25.86)
Platelet, Day 56, n= 4 Number Analyzed 4 participants
17.0  (31.79)
Platelet, Day 83, n= 4 Number Analyzed 4 participants
61.8  (43.41)
WBC, Day 14, n= 5 Number Analyzed 5 participants
0.656  (1.0304)
WBC, Day 28, n= 4 Number Analyzed 4 participants
0.568  (0.6004)
WBC, Day 56, n= 4 Number Analyzed 4 participants
0.698  (1.0605)
WBC, Day 83, n= 4 Number Analyzed 4 participants
0.845  (0.6608)
14.Primary Outcome
Title Change From Baseline for Hematology Parameter: Hemoglobin
Hide Description Blood samples were collected for the analysis of hematology parameter: hemoglobin at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Grams per liter
Day 14, n= 5 Number Analyzed 5 participants
10.0  (5.24)
Day 28, n= 4 Number Analyzed 4 participants
8.3  (3.77)
Day 56, n= 4 Number Analyzed 4 participants
13.5  (8.50)
Day 83, n= 4 Number Analyzed 4 participants
13.3  (5.74)
15.Primary Outcome
Title Change From Baseline for Hematology Parameter: Hematocrit
Hide Description Blood samples were collected for the analysis of hematology parameter: hematocrit at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Percentage of red blood cells in blood
Day 14, n= 5 Number Analyzed 5 participants
0.0320  (0.01488)
Day 28, n= 4 Number Analyzed 4 participants
0.0333  (0.01204)
Day 56, n= 4 Number Analyzed 4 participants
0.0518  (0.02337)
Day 83, n= 4 Number Analyzed 4 participants
0.0465  (0.01396)
16.Primary Outcome
Title Change From Baseline in Hematology Parameter: Mean Corpuscular Volume (MCV)
Hide Description Blood samples were collected for the analysis of hematology parameter: MCV at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Femtoliters
Day 14, n= 5 Number Analyzed 5 participants
1.00  (2.187)
Day 28, n= 4 Number Analyzed 4 participants
2.33  (2.287)
Day 56, n= 4 Number Analyzed 4 participants
2.02  (2.331)
Day 83, n= 4 Number Analyzed 4 participants
1.03  (3.407)
17.Primary Outcome
Title Change From Baseline in Hematology Parameter: Mean Corpuscular Hemoglobin (MCH)
Hide Description Blood samples were collected for the analysis of hematology parameters including MCH at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Picograms
Day 14, n= 5 Number Analyzed 5 participants
0.10  (0.485)
Day 28, n= 4 Number Analyzed 4 participants
-0.15  (0.676)
Day 56, n= 4 Number Analyzed 4 participants
-0.43  (0.763)
Day 83, n= 4 Number Analyzed 4 participants
-0.47  (0.525)
18.Primary Outcome
Title Change From Baseline for Hematology Parameter: Red Blood Cell Count
Hide Description Blood samples were collected for the analysis of hematology parameter: Blood Cell Count at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.
Time Frame Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: 10^12 cells per liter
Day 14, n= 5 Number Analyzed 5 participants
0.304  (0.1417)
Day 28, n= 4 Number Analyzed 4 participants
0.280  (0.0673)
Day 56, n= 4 Number Analyzed 4 participants
0.488  (0.1640)
Day 83, n= 4 Number Analyzed 4 participants
0.485  (0.1034)
19.Primary Outcome
Title Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1)
Hide Description FEV1 is used to assess pulmonary function using a spirometer at indicated timepoints. Baseline value is defined as the maximum measurement of the planned pre-dose measurements on Day 1, predose. Change from Baseline is defined as post-dose visit value minus Baseline value. Spirometry assessments were performed in accordance with American Thoracic Society (ATS)/European Respiratory Society (ERS) guidelines
Time Frame Baseline (Day 1: pre-dose) and at Day 1: 1 Hour post-dose; Day 2: 1 Hour post-dose; Day 14: Pre-dose; Day 14: 1 Hour post-dose and Day 83: Pre-dose
Hide Outcome Measure Data
Hide Analysis Population Description
All Subjects Population.
Arm/Group Title All NEMI
Hide Arm/Group Description:
Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Liters
Day 1, 1 Hour post-dose Number Analyzed 4 participants
0.226  (0.2166)
Day 2, 1 Hour post-dose Number Analyzed 4 participants
0.290  (0.2884)
Day 14, Pre-dose Number Analyzed 4 participants
-0.165  (0.5024)
Day 14, 1 Hour post-dose Number Analyzed 5 participants
0.016  (0.4812)
Day 83, Pre-dose Number Analyzed 5 participants
-0.002  (0.4584)
20.Secondary Outcome
Title Plasma Concentration Following Administration of NEMI
Hide Description Blood samples for pharmacokinetic analysis was collected at the indicated time points following administration of NEMI. NA indicates that standard deviation could not be calculated as a single participant was analyzed
Time Frame Day 1: Pre-dose, 5 minutes, 3 hours and 24 hours post-dose; Days 14 and 83: pre-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic (PK) Population consisted of all participants in the 'All Subjects' population who had at least 1 non-missing PK assessment. Only those participants with data available at specified time point were analyzed (represented by n=X in category titles).
Arm/Group Title NEMI 1000 mcg Via DISKUS NEMI 700 mcg Via ELLIPTA NEMI 500 mcg Via ELLIPTA
Hide Arm/Group Description:
Participants were administered NEMI 1000 mcg using DISKUS DPI once daily in the morning.
Participants were administered NEMI 700 mcg using ELLIPTA DPI once daily in the morning.
Participants were administered NEMI 500 mcg using ELLIPTA DPI once daily in the morning.
Overall Number of Participants Analyzed 1 1 3
Mean (Standard Deviation)
Unit of Measure: Picograms per milliliter
Day 1: Pre-dose; n=1,1,3 Number Analyzed 1 participants 1 participants 3 participants
0.00 [1]   (NA) 0.00 [1]   (NA) 0.00  (0.00)
Day 1: 5 minutes post-dose; n=1,1,3 Number Analyzed 1 participants 1 participants 3 participants
467.00 [1]   (NA) 1524.70 [1]   (NA) 658.27  (394.468)
Day 1: 3 hours post-dose; n=1,1,3 Number Analyzed 1 participants 1 participants 3 participants
895.10 [1]   (NA) 568.60 [1]   (NA) 397.07  (172.226)
Day 1: 24 hours post-dose; n=1,1,3 Number Analyzed 1 participants 1 participants 3 participants
444.40 [1]   (NA) 304.90 [1]   (NA) 198.17  (82.442)
Day 14: Pre-dose; n=1,1,3 Number Analyzed 1 participants 1 participants 3 participants
1396.70 [1]   (NA) 1242.60 [1]   (NA) 750.83  (526.337)
Day 83: Pre-dose; n=0,1,3 Number Analyzed 0 participants 1 participants 3 participants
1419.60 [1]   (NA) 1525.83  (1181.340)
[1]
NA indicates that standard deviation could not be calculated as a single participant was analyzed
Time Frame Non-serious and serious adverse events were collected up to 7.5 months
Adverse Event Reporting Description Non-serious and serious adverse events were collected in the All Subjects population. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
 
Arm/Group Title All NEMI
Hide Arm/Group Description Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
All-Cause Mortality
All NEMI
Affected / at Risk (%)
Total   0/5 (0.00%)    
Hide Serious Adverse Events
All NEMI
Affected / at Risk (%) # Events
Total   0/5 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
All NEMI
Affected / at Risk (%) # Events
Total   5/5 (100.00%)    
Gastrointestinal disorders   
Vomiting  1  2/5 (40.00%)  4
Abdominal discomfort  1  1/5 (20.00%)  3
Abdominal pain  1  1/5 (20.00%)  1
Abdominal tenderness  1  1/5 (20.00%)  1
Constipation  1  1/5 (20.00%)  1
Diarrhoea  1  1/5 (20.00%)  1
Mouth ulceration  1  1/5 (20.00%)  1
General disorders   
Chest discomfort  1  1/5 (20.00%)  1
Fatigue  1  1/5 (20.00%)  1
Feeling hot  1  1/5 (20.00%)  1
Feeling of body temperature change  1  1/5 (20.00%)  1
Pain  1  1/5 (20.00%)  1
Swelling face  1  1/5 (20.00%)  1
Immune system disorders   
Seasonal allergy  1  2/5 (40.00%)  2
Infections and infestations   
Nasopharyngitis  1  3/5 (60.00%)  7
Oral candidiasis  1  1/5 (20.00%)  2
Sinusitis  1  1/5 (20.00%)  1
Fungal skin infection  1  1/5 (20.00%)  1
Skin bacterial infection  1  1/5 (20.00%)  1
Injury, poisoning and procedural complications   
Contusion  1  1/5 (20.00%)  1
Skin abrasion  1  1/5 (20.00%)  1
Metabolism and nutrition disorders   
Iron deficiency  1  1/5 (20.00%)  1
Vitamin B complex deficiency  1  1/5 (20.00%)  1
Vitamin D deficiency  1  1/5 (20.00%)  1
Musculoskeletal and connective tissue disorders   
Back pain  1  1/5 (20.00%)  1
Myalgia  1  1/5 (20.00%)  1
Nervous system disorders   
Headache  1  3/5 (60.00%)  22
Migraine  1  1/5 (20.00%)  1
Product Issues   
Product taste abnormal  1  1/5 (20.00%)  1
Respiratory, thoracic and mediastinal disorders   
Bronchospasm  1  1/5 (20.00%)  1
Cough  1  3/5 (60.00%)  4
Epistaxis  1  1/5 (20.00%)  1
Nasal congestion  1  1/5 (20.00%)  1
Oropharyngeal pain  1  1/5 (20.00%)  1
Pleuritic pain  1  1/5 (20.00%)  1
Rhinorrhoea  1  1/5 (20.00%)  2
Wheezing  1  1/5 (20.00%)  2
Skin and subcutaneous tissue disorders   
Rash  1  1/5 (20.00%)  2
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.
GSK agreements may vary with individual investigators, but will not prohibit any investigator from publishing. GSK supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: GSK Reponse Center
Organization: GlaxoSmithKline
Phone: 866-435-7343
EMail: GSKClinicalSupportHD@gsk.com
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Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT02593539    
Other Study ID Numbers: 204745
2015-004876-31 ( EudraCT Number )
First Submitted: October 29, 2015
First Posted: November 1, 2015
Results First Submitted: May 26, 2021
Results First Posted: June 18, 2021
Last Update Posted: June 18, 2021