Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Effectiveness of Etanercept for Idiopathic Pneumonia Syndrome Following Stem Cell Transplantation (BMT CTN 0403)

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: NCT00421174
Recruitment Status : Completed
First Posted : January 11, 2007
Results First Posted : May 16, 2016
Last Update Posted : September 25, 2017
Sponsor:
Collaborators:
Blood and Marrow Transplant Clinical Trials Network
National Cancer Institute (NCI)
National Marrow Donor Program
Information provided by (Responsible Party):
National Heart, Lung, and Blood Institute (NHLBI)

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Conditions Pneumonia
Idiopathic Pneumonia Syndrome
Interventions Drug: Etanercept
Drug: Placebo plus corticosteroid
Enrollment 37
Recruitment Details Patients were enrolled from September 2007 to August 2011 from 12 different sites.
Pre-assignment Details  
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description Etanercept plus corticosteroids Placebo plus Corticosteroids
Period Title: Overall Study
Started 18 19
Completed 16 18
Not Completed 2 1
Reason Not Completed
Withdrawal by Subject             0             1
Ineligible due to infection             2             0
Arm/Group Title Etanercept Placebo Total
Hide Arm/Group Description Etanercept plus corticosteroids Placebo plus Corticosteroids Total of all reporting groups
Overall Number of Baseline Participants 16 18 34
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 16 participants 18 participants 34 participants
47.7
(22.9 to 70.1)
46.4
(21.8 to 68.8)
46.6
(21.8 to 70.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 18 participants 34 participants
Female
8
  50.0%
10
  55.6%
18
  52.9%
Male
8
  50.0%
8
  44.4%
16
  47.1%
Primary Disease  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 18 participants 34 participants
Acute Myelogenous Leukemia (AML) 1 9 10
Myelodysplastic Syndrome (MDS) 2 2 4
Acute Lymphoblastic Leukemia (ALL) 3 4 7
Lymphoma 3 0 3
Other 7 3 10
Karnofsky Performance   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 18 participants 34 participants
70 - 90 3 1 4
50 - 60 5 5 10
< 50 5 10 15
Unknown 3 2 5
[1]
Measure Description: Assesses patient self-perceived global quality of life and functioning (excellent = 100, good = 70-90, fair = 50-60, poor < 50), where 100 equals perfect quality of life.
Conditioning Regimen  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 18 participants 34 participants
Myeloablative 9 11 20
Non-myeloablative 7 7 14
Recipient Cytomegalovirus Status  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 18 participants 34 participants
Positive 8 10 18
Negative 8 7 15
Unknown 0 1 1
Total Bilirubin   [1] 
Median (Full Range)
Unit of measure:  Mg/dl
Number Analyzed 16 participants 18 participants 34 participants
1.2
(0.2 to 18.9)
0.9
(0.2 to 10.7)
1.0
(0.2 to 18.9)
[1]
Measure Description: Serum total bilirubin (mg/dl) at study entry.
Creatinine   [1] 
Median (Full Range)
Unit of measure:  Mg/dl
Number Analyzed 16 participants 18 participants 34 participants
1.3
(0.6 to 2.5)
1.4
(0.4 to 2.9)
1.3
(0.4 to 2.9)
[1]
Measure Description: Serum creatinine (mg/dl) at study entry.
Oxygen Support   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 18 participants 34 participants
Nasal cannula 10 6 16
Face mask 3 5 8
Mechanical ventilation 2 6 8
Unknown 1 1 2
[1]
Measure Description: Method of supplemental oxygen support at study entry.
1.Primary Outcome
Title Response Rate
Hide Description 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.
Time Frame Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
62.5
(35.4 to 84.8)
66.7
(41.0 to 86.7)
2.Secondary Outcome
Title Response to Therapy
Hide Description Response 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.
Time Frame Day 56
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
56.3
(29.9 to 80.3)
50.0
(26.0 to 74.0)
3.Secondary Outcome
Title Discontinuation of Supplemental Oxygen
Hide Description The “time required to discontinue supplemental oxygen” will be measured in the number of days from study entry.
Time Frame Day 56
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Median (Full Range)
Unit of Measure: days
9
(1 to 36)
7
(2 to 30)
4.Secondary Outcome
Title Corticosteroid Dose
Hide Description Patients were treated with systemic corticosteroids with methylprednisolone at 2 mg/kg/day on day 0, with taper allowed after day 7.
Time Frame Day 14 and 28
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Median (Full Range)
Unit of Measure: mg/kg/day
Day 14
0.94
(0.56 to 1.92)
1.00
(0.25 to 2.21)
Day 28
0.57
(0.0 to 2.0)
0.49
(0.03 to 1.04)
5.Secondary Outcome
Title Overall Survival
Hide Description Percentage of patients that survived after one year
Time Frame Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
18.8
(6.5 to 46.3)
16.7
(4.1 to 36.5)
6.Secondary Outcome
Title Incidence of Infection
Hide Description [Not Specified]
Time Frame Day 56
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Overall Number of Units Analyzed
Type of Units Analyzed: Total number of infections
20 33
Measure Type: Number
Unit of Measure: Infections
Bacterial infection 9 21
Viral infection 7 10
Fungal infection 4 2
7.Secondary Outcome
Title Incidence of Toxicity
Hide Description [Not Specified]
Time Frame Day 56
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Overall Number of Units Analyzed
Type of Units Analyzed: Total number of toxicities
14 25
Measure Type: Number
Unit of Measure: Toxcities
Renal 4 3
Hepatic 7 14
Cardiac 1 2
Central nervous system 2 6
8.Secondary Outcome
Title Incidence of Graft-vs-Host-Disease (GVHD)
Hide Description [Not Specified]
Time Frame Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
Acute GVHD
31.2
(11.0 to 58.7)
44.4
(21.5 to 69.2)
GVHD
25.0
(7.3 to 52.4)
27.8
(9.7 to 53.5)
Chronic GVHD
12.5
(1.6 to 38.4)
0
(0 to 18.5)
9.Secondary Outcome
Title Incidence of Relapse
Hide Description Percentage of patients who experience relapse. Deaths without relapse are considered as a competing risk.
Time Frame Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Measure Type: Count of Participants
Unit of Measure: Participants
3
  18.8%
2
  11.1%
10.Secondary Outcome
Title Overall Mortality
Hide Description [Not Specified]
Time Frame Year 2
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 16 18
Measure Type: Number
Unit of Measure: participants
13 15
11.Secondary Outcome
Title Dermatologic Reaction
Hide Description [Not Specified]
Time Frame Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
No data collected
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
12.Secondary Outcome
Title Pro-inflammatory Markers of Pulmonary Disease, in Both BAL Fluid and Plasma
Hide Description [Not Specified]
Time Frame Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
No data collected
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description:
Etanercept plus corticosteroids
Placebo plus Corticosteroids
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 1-year post transplant
Adverse Event Reporting Description 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.
 
Arm/Group Title Etanercept Placebo
Hide Arm/Group Description Etanercept plus corticosteroids Placebo plus Corticosteroids
All-Cause Mortality
Etanercept Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Etanercept Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/16 (0.00%)   1/18 (5.56%) 
Respiratory, thoracic and mediastinal disorders     
Pulmonary alveolar haemorrhage * 1  0/16 (0.00%)  1/18 (5.56%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Etanercept Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/16 (0.00%)   0/18 (0.00%) 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Adam Mendizabal, PhD
Organization: The EMMES Corporation
Phone: (301) 251-1161 ext 221
EMail: amendizabal@emmes.com
Layout table for additonal information
Responsible Party: National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT00421174     History of Changes
Other Study ID Numbers: BMTCTN0403
BMT CTN 0403 ( Other Identifier: Blood and Marrow Transplant Clinicial Trials Network )
U01HL069294-05 ( U.S. NIH Grant/Contract )
465 ( Other Identifier: BMT CTN )
First Submitted: January 9, 2007
First Posted: January 11, 2007
Results First Submitted: January 28, 2016
Results First Posted: May 16, 2016
Last Update Posted: September 25, 2017