Trial Outcomes & Findings for Single Dose of pGM169/GL67A in CF Patients (NCT NCT00789867)
NCT ID: NCT00789867
Last Updated: 2020-01-18
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
35 participants
Primary outcome timeframe
6-8h
Results posted on
2020-01-18
Participant Flow
Participant milestones
| Measure |
20 ml pGM169/GL67A
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
|---|---|---|---|
|
Overall Study
STARTED
|
17
|
10
|
8
|
|
Overall Study
COMPLETED
|
17
|
10
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Single Dose of pGM169/GL67A in CF Patients
Baseline characteristics by cohort
| Measure |
20ml pGM169/GL67A
n=17 Participants
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
n=10 Participants
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
n=8 Participants
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
Total
n=35 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
2 Participants
n=483 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
16 Participants
n=93 Participants
|
9 Participants
n=4 Participants
|
8 Participants
n=27 Participants
|
33 Participants
n=483 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Age, Continuous
|
26.7 years
n=93 Participants
|
33.2 years
n=4 Participants
|
32.6 years
n=27 Participants
|
27.4 years
n=483 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
12 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
5 Participants
n=27 Participants
|
23 Participants
n=483 Participants
|
|
Region of Enrollment
United Kingdom
|
17 participants
n=93 Participants
|
10 participants
n=4 Participants
|
8 participants
n=27 Participants
|
35 participants
n=483 Participants
|
PRIMARY outcome
Timeframe: 6-8hPopulation: A lower number of participants due to missing data
Outcome measures
| Measure |
20ml pGM169/GL67A
n=15 Participants
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
|---|---|---|---|
|
Body Maximum Temperature
|
38.6 celsius
Standard Deviation 0.5
|
38.0 celsius
Standard Deviation 0.7
|
37.4 celsius
Standard Deviation 0.3
|
PRIMARY outcome
Timeframe: 8hPopulation: A lower number of participants due to missing data
Blood leukocytes measure
Outcome measures
| Measure |
20ml pGM169/GL67A
n=15 Participants
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
|---|---|---|---|
|
Blood Leukocytes
|
15.8 x1000000000 cells/L
Standard Deviation 3.2
|
14.1 x1000000000 cells/L
Standard Deviation 4.5
|
12.8 x1000000000 cells/L
Standard Deviation 3.8
|
PRIMARY outcome
Timeframe: 8hPopulation: A lower number of participants due to missing data
Blood neutrophils measures
Outcome measures
| Measure |
20ml pGM169/GL67A
n=15 Participants
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
|---|---|---|---|
|
Blood Neutrophils
|
13.9 1000000000/L
Standard Deviation 3.4
|
11.3 1000000000/L
Standard Deviation 4.1
|
9.8 1000000000/L
Standard Deviation 3.5
|
PRIMARY outcome
Timeframe: 8hPopulation: A lower number of participants due to missing data
FEV1 relative % drop measure
Outcome measures
| Measure |
20ml pGM169/GL67A
n=15 Participants
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
|---|---|---|---|
|
FEV1 Relative % Drop
|
24.6 % of drop
Standard Deviation 9.3
|
17.5 % of drop
Standard Deviation 7.8
|
16.8 % of drop
Standard Deviation 4.0
|
PRIMARY outcome
Timeframe: 6hPopulation: A lower number of participants due to missing data
FVC relative % drop measure
Outcome measures
| Measure |
20ml pGM169/GL67A
n=15 Participants
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
|---|---|---|---|
|
FVC Relative % Drop
|
20.7 % drop
Standard Deviation 2.9
|
13.7 % drop
Standard Deviation 2.2
|
14.7 % drop
Standard Deviation 2.2
|
PRIMARY outcome
Timeframe: 8hPopulation: A lower number of participants due to missing data
Lung clearance index measure is a measure of abnormal ventilation distribution derived from the multiple breath inert gas washout technique.
Outcome measures
| Measure |
20ml pGM169/GL67A
n=15 Participants
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
n=6 Participants
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
|---|---|---|---|
|
Lung Clearance Index - LCI
|
0.75 index
Standard Deviation 0.3
|
0.32 index
Standard Deviation 0.1
|
0.32 index
Standard Deviation .01
|
Adverse Events
20ml pGM169/GL67A
Serious events: 1 serious events
Other events: 14 other events
Deaths: 0 deaths
10ml pGM169/GL67A
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
5ml pGM169/GL67A
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
20ml pGM169/GL67A
n=17 participants at risk
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
n=10 participants at risk
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
n=8 participants at risk
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
|---|---|---|---|
|
Endocrine disorders
Acute pancreatitis
|
0.00%
0/17 • 28 days
|
10.0%
1/10 • Number of events 1 • 28 days
|
0.00%
0/8 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Injury in luvula sustained from pressure of tube during bronchoscopy 03/04/2009
|
5.9%
1/17 • Number of events 1 • 28 days
|
0.00%
0/10 • 28 days
|
0.00%
0/8 • 28 days
|
Other adverse events
| Measure |
20ml pGM169/GL67A
n=17 participants at risk
Received a nebulized dose 20ml via an breath-actuated nebulizer
|
10ml pGM169/GL67A
n=10 participants at risk
Received a nebulized dose 10ml via an breath-actuated nebulizer
|
5ml pGM169/GL67A
n=8 participants at risk
Received a nebulized dose 5ml via an breath-actuated nebulizer
|
|---|---|---|---|
|
Immune system disorders
Mild self limiting influenza like systemic response
|
82.4%
14/17 • Number of events 14 • 28 days
|
70.0%
7/10 • Number of events 7 • 28 days
|
12.5%
1/8 • Number of events 1 • 28 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place