Effectiveness of Etanercept for Idiopathic Pneumonia Syndrome Following Stem Cell Transplantation (BMT CTN 0403)
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ClinicalTrials.gov Identifier: NCT00421174 |
Recruitment Status
:
Completed
First Posted
: January 11, 2007
Results First Posted
: May 16, 2016
Last Update Posted
: September 25, 2017
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Tracking Information | |||||||||||||||
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First Submitted Date ICMJE | January 9, 2007 | ||||||||||||||
First Posted Date ICMJE | January 11, 2007 | ||||||||||||||
Results First Submitted Date | January 28, 2016 | ||||||||||||||
Results First Posted Date | May 16, 2016 | ||||||||||||||
Last Update Posted Date | September 25, 2017 | ||||||||||||||
Study Start Date ICMJE | August 2007 | ||||||||||||||
Actual Primary Completion Date | June 2010 (Final data collection date for primary outcome measure) | ||||||||||||||
Current Primary Outcome Measures ICMJE |
Response Rate [ Time Frame: Day 28 ] 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.
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Original Primary Outcome Measures ICMJE |
Day 28 response rate (response will be defined as (a) survival to Day 28 of study, plus (b) discontinuation of all supplemental oxygen support for more than 72 consecutive hours by Day 28) | ||||||||||||||
Change History | Complete list of historical versions of study NCT00421174 on ClinicalTrials.gov Archive Site | ||||||||||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Outcome Measures ICMJE | Not Provided | ||||||||||||||
Original Other Outcome Measures ICMJE | Not Provided | ||||||||||||||
Descriptive Information | |||||||||||||||
Brief Title ICMJE | Effectiveness of Etanercept for Idiopathic Pneumonia Syndrome Following Stem Cell Transplantation (BMT CTN 0403) | ||||||||||||||
Official Title ICMJE | A Randomized Double-Blind, Placebo-Controlled Trial of Soluble Tumor Necrosis Factor Receptor: Enbrel (Etanercept) for the Treatment of Acute Non-Infectious Pulmonary Dysfunction (Idiopathic Pneumonia Syndrome) Following Allogeneic Cell Transplantation (BMT CTN #0403) | ||||||||||||||
Brief Summary | The study is designed as a Phase III, multi-center randomized, double-blind, placebo-controlled trial investigating the use of etanercept for the treatment of acute, non-infectious pulmonary dysfunction (IPS) occurring after allogeneic hematopoietic cell transplantation (HCT). | ||||||||||||||
Detailed Description | BACKGROUND: Over the last two decades, allogeneic hematopoietic cell transplantation (HCT) has emerged as an important treatment for a number of malignant and non-malignant disorders. Unfortunately, several complications, including graft-versus-host disease (GVHD) and pulmonary dysfunction, limit the utility of this aggressive form of therapy. Infectious and non-infectious lung complications occur in 25% to 55% of HCT recipients and account for up to 50% of transplant-related mortality. In about half of affected patients, no infectious organisms are identified in the lungs. Two major types of non-infectious pulmonary injury are recognized: acute idiopathic pneumonia syndrome (IPS) and sub-acute lung injury (obstructive airway disease or bronchiolitis obliterans [BrOb] and restrictive lung disease). The current study will examine the use of etanercept in patients with IPS. DESIGN NARRATIVE: Eligible patients will be randomized to receive one of two arms of therapy: (A) etanercept plus corticosteroids, or (B) placebo plus corticosteroids. Patients will receive a total of eight doses of etanercept (or placebo) over a 4-week period. The initial dose of etanercept (or placebo) will be administered intravenously on Day 0, with subsequent doses administered subcutaneously (SQ). Dosing will be administered twice weekly over 4 consecutive weeks. The placebo will be the inert diluent used for the etanercept formulation. Additionally, patients in both arms will receive corticosteroids (2 mg/kg/day) Day 0 through Day 7, with subsequent taper as clinically indicated. Chest radiographs shall be obtained weekly through Day 28. Plasma cytokine profiles will be obtained on Days 0, 7, and 28. For patients < 30 days post-transplant: If the patient's clinical condition is such that a broncho-alveolar lavage (BAL) is deemed "not possible to be performed" by the treating physician (or pulmonologist), then the "on study" BAL may be waived. In such circumstances, the patient may register and be randomized to study therapy without the BAL being undertaken. For patients not on mechanical ventilation: If a BAL is not done, appropriate virology studies on a nasal swab (or nasal washing) are required as a minimum procedure to study entry. For patients on mechanical ventilation: Microbiologic studies of a deep endotracheal aspirate are allowed in lieu of a formal bronchoscopy procedure. However, no protocol-specified biologic studies (see Section 4.4) will be done on these specimens. For patients 31-180 days post-transplant: An "on study" bronchoscopy is required in all cases. If, at any point following initiation of study drug therapy, previously obtained BAL fluid cultures or other BAL fluid analysis become positive for an infectious pathogen, study drug therapy shall be discontinued at that point, and not re-instituted. The patient will discontinue study drug therapy, but will still be followed for outcome. The primary study endpoint is response at Day 28. Patients who discontinue study drug therapy for any reason will still be followed for primary and secondary study endpoints. |
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Study Type ICMJE | Interventional | ||||||||||||||
Study Phase | Phase 3 | ||||||||||||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double (Participant, Investigator) Primary Purpose: Treatment |
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Condition ICMJE |
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Intervention ICMJE |
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Study Arms |
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Publications * | Yanik GA, Horowitz MM, Weisdorf DJ, Logan BR, Ho VT, Soiffer RJ, Carter SL, Wu J, Wingard JR, Difronzo NL, Ferrara JL, Giralt S, Madtes DK, Drexler R, White ES, Cooke KR. Randomized, double-blind, placebo-controlled trial of soluble tumor necrosis factor receptor: enbrel (etanercept) for the treatment of idiopathic pneumonia syndrome after allogeneic stem cell transplantation: blood and marrow transplant clinical trials network protocol. Biol Blood Marrow Transplant. 2014 Jun;20(6):858-64. doi: 10.1016/j.bbmt.2014.02.026. Epub 2014 Mar 7. | ||||||||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||||||||
Recruitment Status ICMJE | Completed | ||||||||||||||
Actual Enrollment ICMJE |
37 | ||||||||||||||
Original Enrollment ICMJE |
120 | ||||||||||||||
Actual Study Completion Date | July 2013 | ||||||||||||||
Actual Primary Completion Date | June 2010 (Final data collection date for primary outcome measure) | ||||||||||||||
Eligibility Criteria ICMJE | Inclusion Criteria: Patients fulfilling the following criteria will be eligible for registration in this study:
Patients fulfilling the following criteria will be eligible for random assignment in this study:
Exclusion Criteria:
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Sex/Gender |
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Ages | 18 Years and older (Adult, Senior) | ||||||||||||||
Accepts Healthy Volunteers | No | ||||||||||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||||||||||
Listed Location Countries ICMJE | United States | ||||||||||||||
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Administrative Information | |||||||||||||||
NCT Number ICMJE | NCT00421174 | ||||||||||||||
Other Study ID Numbers ICMJE | 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 ) |
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Has Data Monitoring Committee | Yes | ||||||||||||||
U.S. FDA-regulated Product | Not Provided | ||||||||||||||
IPD Sharing Statement |
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Responsible Party | National Heart, Lung, and Blood Institute (NHLBI) | ||||||||||||||
Study Sponsor ICMJE | National Heart, Lung, and Blood Institute (NHLBI) | ||||||||||||||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | National Heart, Lung, and Blood Institute (NHLBI) | ||||||||||||||
Verification Date | August 2017 | ||||||||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |