Trial Outcomes & Findings for A Randomized Trial of GM-CSF in Patients With ALI/ARDS (NCT NCT00201409)

NCT ID: NCT00201409

Last Updated: 2015-12-29

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

132 participants

Primary outcome timeframe

Measured at Day 28

Results posted on

2015-12-29

Participant Flow

Participant milestones

Participant milestones
Measure
GM-CSF Group
Participants will be randomized to receive recombinant human GM-CSF (250 mcg/M2).
Placebo Group
Participants will be randomized to receive placebo.
Overall Study
STARTED
65
67
Overall Study
COMPLETED
64
66
Overall Study
NOT COMPLETED
1
1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Randomized Trial of GM-CSF in Patients With ALI/ARDS

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
GM-CSF Group
n=65 Participants
Participants will be randomized to receive recombinant human GM-CSF (250 mcg/M2).
Placebo Group
n=67 Participants
Participants will be randomized to receive placebo.
Total
n=132 Participants
Total of all reporting groups
Sex: Female, Male
Female
30 Participants
n=5 Participants
28 Participants
n=7 Participants
58 Participants
n=5 Participants
Sex: Female, Male
Male
35 Participants
n=5 Participants
39 Participants
n=7 Participants
74 Participants
n=5 Participants
Age, Continuous
48.5 years
STANDARD_DEVIATION 15.6 • n=5 Participants
48.3 years
STANDARD_DEVIATION 13.8 • n=7 Participants
48.4 years
STANDARD_DEVIATION 14.6 • n=5 Participants

PRIMARY outcome

Timeframe: Measured at Day 28

Outcome measures

Outcome measures
Measure
GM-CSF Group
n=64 Participants
Participants will be randomized to receive recombinant human GM-CSF (250 mcg/M2).
Placebo Group
n=66 Participants
Participants will be randomized to receive placebo.
Ventilator-free Days During Days 1-28
10.8 Days
Standard Deviation 10.5
10.7 Days
Standard Deviation 10.3

SECONDARY outcome

Timeframe: Day 1, Day 15

The oxygenation index is a calculation used in intensive care medicine to measure the fraction of inspired oxygen (FiO2) and its usage within the body. It is calculated as the fraction of inspired oxygen times Mean airway pressure)/Partial pressure of oxygen in arterial blood Day 15 minus first day drug or placebo administered (Day 1).

Outcome measures

Outcome measures
Measure
GM-CSF Group
n=64 Participants
Participants will be randomized to receive recombinant human GM-CSF (250 mcg/M2).
Placebo Group
n=66 Participants
Participants will be randomized to receive placebo.
Oxygenation Index Change at Day 15 From Day 1
-3.5 oxygenation index
Standard Deviation 10.4
-4.6 oxygenation index
Standard Deviation 4.5

SECONDARY outcome

Timeframe: Measured at Day 28

Non-respiratory

Outcome measures

Outcome measures
Measure
GM-CSF Group
n=64 Participants
Participants will be randomized to receive recombinant human GM-CSF (250 mcg/M2).
Placebo Group
n=66 Participants
Participants will be randomized to receive placebo.
Days Without Organ Failure
15.7 days
Standard Deviation 11.9
12.8 days
Standard Deviation 11.3

Adverse Events

GM-CSF Group

Serious events: 31 serious events
Other events: 39 other events
Deaths: 0 deaths

Placebo Group

Serious events: 34 serious events
Other events: 39 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
GM-CSF Group
n=64 participants at risk
Participants will be randomized to receive recombinant human GM-CSF (250 mcg/M2).
Placebo Group
n=66 participants at risk
Participants will be randomized to receive placebo.
Immune system disorders
Sepsis or Multi-organ failure
17.2%
11/64 • Number of events 11
19.7%
13/66 • Number of events 13
Respiratory, thoracic and mediastinal disorders
Pulmonary SAE
17.2%
11/64 • Number of events 11
24.2%
16/66 • Number of events 16
Infections and infestations
Infection SAE
10.9%
7/64 • Number of events 7
13.6%
9/66 • Number of events 9
General disorders
Miscellaneous
3.1%
2/64 • Number of events 2
0.00%
0/66

Other adverse events

Other adverse events
Measure
GM-CSF Group
n=64 participants at risk
Participants will be randomized to receive recombinant human GM-CSF (250 mcg/M2).
Placebo Group
n=66 participants at risk
Participants will be randomized to receive placebo.
Cardiac disorders
Cardiovascular
39.1%
25/64 • Number of events 25
37.9%
25/66 • Number of events 25
Immune system disorders
Fever
23.4%
15/64 • Number of events 15
16.7%
11/66 • Number of events 11
Gastrointestinal disorders
GI
4.7%
3/64 • Number of events 3
7.6%
5/66 • Number of events 5
Blood and lymphatic system disorders
Hematologic
40.6%
26/64 • Number of events 26
25.8%
17/66 • Number of events 17
Infections and infestations
Infection
20.3%
13/64 • Number of events 13
10.6%
7/66 • Number of events 7
Nervous system disorders
Neurologic
6.2%
4/64 • Number of events 4
1.5%
1/66 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Pulmonary
18.8%
12/64 • Number of events 12
27.3%
18/66 • Number of events 18
Hepatobiliary disorders
Renal/Hepatic
7.8%
5/64 • Number of events 5
12.1%
8/66 • Number of events 8
Metabolism and nutrition disorders
Metabolic
10.9%
7/64 • Number of events 7
10.6%
7/66 • Number of events 7
General disorders
Miscellaneous
10.9%
7/64 • Number of events 7
6.1%
4/66 • Number of events 4

Additional Information

Dr. Robert Hyzy

University of Michigan

Phone: 7349365201

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place