Trial Outcomes & Findings for Effectiveness of Etanercept for Idiopathic Pneumonia Syndrome Following Stem Cell Transplantation (BMT CTN 0403) (NCT NCT00421174)
NCT ID: NCT00421174
Last Updated: 2021-11-01
Results Overview
Response will be defined as survival to Day 28 of study, plus discontinuation of all supplemental oxygen support for more than 72 consecutive hours by Day 28.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
37 participants
Primary outcome timeframe
Day 28
Results posted on
2021-11-01
Participant Flow
Patients were enrolled from September 2007 to August 2011 from 12 different sites.
Participant milestones
| Measure |
Etanercept
Etanercept plus corticosteroids
|
Placebo
Placebo plus Corticosteroids
|
|---|---|---|
|
Overall Study
STARTED
|
18
|
19
|
|
Overall Study
COMPLETED
|
16
|
18
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
Etanercept
Etanercept plus corticosteroids
|
Placebo
Placebo plus Corticosteroids
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
|
Overall Study
Ineligible due to infection
|
2
|
0
|
Baseline Characteristics
Effectiveness of Etanercept for Idiopathic Pneumonia Syndrome Following Stem Cell Transplantation (BMT CTN 0403)
Baseline characteristics by cohort
| Measure |
Etanercept
n=16 Participants
Etanercept plus corticosteroids
|
Placebo
n=18 Participants
Placebo plus Corticosteroids
|
Total
n=34 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
47.7 years
n=5 Participants
|
46.4 years
n=7 Participants
|
46.6 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Primary Disease
Acute Myelogenous Leukemia (AML)
|
1 participants
n=5 Participants
|
9 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
Primary Disease
Myelodysplastic Syndrome (MDS)
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Primary Disease
Acute Lymphoblastic Leukemia (ALL)
|
3 participants
n=5 Participants
|
4 participants
n=7 Participants
|
7 participants
n=5 Participants
|
|
Primary Disease
Lymphoma
|
3 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Primary Disease
Other
|
7 participants
n=5 Participants
|
3 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
Karnofsky Performance
70 - 90
|
3 participants
n=5 Participants
|
1 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Karnofsky Performance
50 - 60
|
5 participants
n=5 Participants
|
5 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
Karnofsky Performance
< 50
|
5 participants
n=5 Participants
|
10 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Karnofsky Performance
Unknown
|
3 participants
n=5 Participants
|
2 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Conditioning Regimen
Myeloablative
|
9 participants
n=5 Participants
|
11 participants
n=7 Participants
|
20 participants
n=5 Participants
|
|
Conditioning Regimen
Non-myeloablative
|
7 participants
n=5 Participants
|
7 participants
n=7 Participants
|
14 participants
n=5 Participants
|
|
Recipient Cytomegalovirus Status
Positive
|
8 participants
n=5 Participants
|
10 participants
n=7 Participants
|
18 participants
n=5 Participants
|
|
Recipient Cytomegalovirus Status
Negative
|
8 participants
n=5 Participants
|
7 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Recipient Cytomegalovirus Status
Unknown
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Total Bilirubin
|
1.2 mg/dl
n=5 Participants
|
0.9 mg/dl
n=7 Participants
|
1.0 mg/dl
n=5 Participants
|
|
Creatinine
|
1.3 mg/dl
n=5 Participants
|
1.4 mg/dl
n=7 Participants
|
1.3 mg/dl
n=5 Participants
|
|
Oxygen Support
Nasal cannula
|
10 participants
n=5 Participants
|
6 participants
n=7 Participants
|
16 participants
n=5 Participants
|
|
Oxygen Support
Face mask
|
3 participants
n=5 Participants
|
5 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Oxygen Support
Mechanical ventilation
|
2 participants
n=5 Participants
|
6 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Oxygen Support
Unknown
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 28Response will be defined as survival to Day 28 of study, plus discontinuation of all supplemental oxygen support for more than 72 consecutive hours by Day 28.
Outcome measures
| Measure |
Etanercept
n=16 Participants
Etanercept plus corticosteroids
|
Placebo
n=18 Participants
Placebo plus Corticosteroids
|
|---|---|---|
|
Response Rate
|
62.5 percentage of participants
Interval 35.4 to 84.8
|
66.7 percentage of participants
Interval 41.0 to 86.7
|
SECONDARY outcome
Timeframe: Day 56Response will be defined as the ability to survive to Day 56 of study, plus the ability to completely discontinue all supplemental oxygen support for \> 72 consecutive hours during this time period.
Outcome measures
| Measure |
Etanercept
n=16 Participants
Etanercept plus corticosteroids
|
Placebo
n=18 Participants
Placebo plus Corticosteroids
|
|---|---|---|
|
Response to Therapy
|
56.3 percentage of participants
Interval 29.9 to 80.3
|
50.0 percentage of participants
Interval 26.0 to 74.0
|
SECONDARY outcome
Timeframe: Day 56The "time required to discontinue supplemental oxygen" will be measured in the number of days from study entry.
Outcome measures
| Measure |
Etanercept
n=16 Participants
Etanercept plus corticosteroids
|
Placebo
n=18 Participants
Placebo plus Corticosteroids
|
|---|---|---|
|
Discontinuation of Supplemental Oxygen
|
9 days
Interval 1.0 to 36.0
|
7 days
Interval 2.0 to 30.0
|
SECONDARY outcome
Timeframe: Day 14 and 28Patients were treated with systemic corticosteroids with methylprednisolone at 2 mg/kg/day on day 0, with taper allowed after day 7.
Outcome measures
| Measure |
Etanercept
n=16 Participants
Etanercept plus corticosteroids
|
Placebo
n=18 Participants
Placebo plus Corticosteroids
|
|---|---|---|
|
Corticosteroid Dose
Day 14
|
0.94 mg/kg/day
Interval 0.56 to 1.92
|
1.00 mg/kg/day
Interval 0.25 to 2.21
|
|
Corticosteroid Dose
Day 28
|
0.57 mg/kg/day
Interval 0.0 to 2.0
|
0.49 mg/kg/day
Interval 0.03 to 1.04
|
SECONDARY outcome
Timeframe: Year 1Percentage of patients that survived after one year
Outcome measures
| Measure |
Etanercept
n=16 Participants
Etanercept plus corticosteroids
|
Placebo
n=18 Participants
Placebo plus Corticosteroids
|
|---|---|---|
|
Overall Survival
|
18.8 percentage of participants
Interval 6.5 to 46.3
|
16.7 percentage of participants
Interval 4.1 to 36.5
|
SECONDARY outcome
Timeframe: Day 56Outcome measures
| Measure |
Etanercept
n=20 Total number of infections
Etanercept plus corticosteroids
|
Placebo
n=33 Total number of infections
Placebo plus Corticosteroids
|
|---|---|---|
|
Incidence of Infection
Bacterial infection
|
9 Infections
|
21 Infections
|
|
Incidence of Infection
Viral infection
|
7 Infections
|
10 Infections
|
|
Incidence of Infection
Fungal infection
|
4 Infections
|
2 Infections
|
SECONDARY outcome
Timeframe: Day 56Outcome measures
| Measure |
Etanercept
n=14 Total number of toxicities
Etanercept plus corticosteroids
|
Placebo
n=25 Total number of toxicities
Placebo plus Corticosteroids
|
|---|---|---|
|
Incidence of Toxicity
Renal
|
4 Toxcities
|
3 Toxcities
|
|
Incidence of Toxicity
Hepatic
|
7 Toxcities
|
14 Toxcities
|
|
Incidence of Toxicity
Cardiac
|
1 Toxcities
|
2 Toxcities
|
|
Incidence of Toxicity
Central nervous system
|
2 Toxcities
|
6 Toxcities
|
SECONDARY outcome
Timeframe: Year 1Outcome measures
| Measure |
Etanercept
n=16 Participants
Etanercept plus corticosteroids
|
Placebo
n=18 Participants
Placebo plus Corticosteroids
|
|---|---|---|
|
Incidence of Graft-vs-Host-Disease (GVHD)
Acute GVHD
|
31.2 percentage of participants
Interval 11.0 to 58.7
|
44.4 percentage of participants
Interval 21.5 to 69.2
|
|
Incidence of Graft-vs-Host-Disease (GVHD)
GVHD
|
25.0 percentage of participants
Interval 7.3 to 52.4
|
27.8 percentage of participants
Interval 9.7 to 53.5
|
|
Incidence of Graft-vs-Host-Disease (GVHD)
Chronic GVHD
|
12.5 percentage of participants
Interval 1.6 to 38.4
|
0 percentage of participants
Interval 0.0 to 18.5
|
SECONDARY outcome
Timeframe: Year 1Percentage of patients who experience relapse. Deaths without relapse are considered as a competing risk.
Outcome measures
| Measure |
Etanercept
n=16 Participants
Etanercept plus corticosteroids
|
Placebo
n=18 Participants
Placebo plus Corticosteroids
|
|---|---|---|
|
Incidence of Relapse
|
3 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Year 2Outcome measures
| Measure |
Etanercept
n=16 Participants
Etanercept plus corticosteroids
|
Placebo
n=18 Participants
Placebo plus Corticosteroids
|
|---|---|---|
|
Overall Mortality
|
13 participants
|
15 participants
|
SECONDARY outcome
Timeframe: Day 28Population: No data collected
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 28Population: No data collected
Outcome measures
Outcome data not reported
Adverse Events
Etanercept
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Placebo
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Etanercept
n=16 participants at risk
Etanercept plus corticosteroids
|
Placebo
n=18 participants at risk
Placebo plus Corticosteroids
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary alveolar haemorrhage
|
0.00%
0/16 • 1-year post transplant
Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected adverse events are reported here.
|
5.6%
1/18 • 1-year post transplant
Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected adverse events are reported here.
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place