NCT03459040|CompletedPhase 2ResultsDMCFDA Drug
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Sponsor study identifier
Secondary identifiers
NIH
interventional
30
1 country
5
Study Start
August 17, 2018
Actual (reported)Primary Completion
August 21, 2020
Actual (reported)Study Completion
August 21, 2020
Actual (reported)Bone marrow transplant (BMT) patients can develop graft-versus-host disease (GVHD), a serious and potentially fatal complication. The researchers have developed a blood test to identify patients most at risk for developing severe GVHD. Patients who consent to this study will have their blood tested up to two times after BMT to determine if they are at high risk for severe GVHD. The tests will be performed one week and two weeks after BMT. Patients who are high risk will be treated with a drug called alpha-1-antitrypsin (AAT) to see if it prevents the development of severe GVHD. Patients will receive 16 doses of AAT through a catheter placed into a blood vessel over eight weeks. AAT will be given either in the hospital or the outpatient clinic two times per week. Patients will be followed for the development of severe GVHD for up to four months from the BMT and will continue to be followed at routine clinic visits for up to one year after BMT.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
August 21, 2020
Actual (reported)Click on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
March 2, 2018
Actual (reported)March 8, 2018
Actual (reported)August 17, 2018
Actual (reported)August 21, 2020
Actual (reported)August 21, 2020
Actual (reported)May 24, 2021
Actual (reported)Number of High Risk Patients Who Develop Steroid Refractory GVHD
Number of High Risk patients who develop steroid refractory GVHD by day 100 post Hematopoietic cell transplant (HCT) . Steroid refractory GVHD defined as patients who did not achieve Complete Response (CR) or Partial Response (PR) by day 28 of systemic steroid treatment OR if additional immunosuppression beyond steroids was given for treatment of GVHD prior to 28 days of steroid treatment. * CR: All evaluable organs (skin, liver, GI tract) stage 0. For a response to be scored as CR on day 28, the patient must be in CR on that day and have had no intervening additional GVHD therapy. * PR: An improvement in one or more organ involved with GVHD symptoms without worsening in others. For a response to be scored as PR on day 28, the patient must be in PR on that day and have had no intervening additional GVHD therapy.
Day 100 post HCT.
Number of Participants Alive at 6 Months and 1 Year
6 months and 1 year
Number of Participants With Non-relapse Mortality (NRM)
6 months and 1 year
Number of Participants With Relapse
1 year
Number of Participants With Clinically Relevant GVHD States Grade II-IV GVHD
100 days
Number of Participants Achieving Overall Response
Day 28
16 doses of AAT through a catheter placed into a blood vessel over eight weeks.
AAT will be given either in the hospital or the outpatient clinic two times per week.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
City of Hope
Duarte, California, 91010, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Ohio State University
Columbus, Ohio, 43210, United States
Vanderbilt University
Nashville, Tennessee, 37232, United States
Gergoudis SC, DeFilipp Z, Ozbek U, Sandhu KS, Etra AM, Choe HK, Kitko CL, Ayuk F, Aziz M, Baez J, Ben-David K, Bunworasate U, Gandhi I, Hexner EO, Hogan WJ, Holler E, Kasikis S, Kowalyk SM, Lin JY, Merli P, Morales G, Nakamura R, Reshef R, Rosler W, Srinagesh H, Young R, Chen YB, Ferrara JLM, Levine JE. Biomarker-guided preemption of steroid-refractory graft-versus-host disease with alpha-1-antitrypsin. Blood Adv. 2020 Dec 22;4(24):6098-6105. doi: 10.1182/bloodadvances.2020003336.
John Levine, MD
Icahn School of Medicine at Mount Sinai
30 participants (actual enrollment reported by the registry).
First Submitted
March 2, 2018
First Posted
March 8, 2018
Study Start
August 17, 2018
Actual (reported)A Phase I/II GVHD Prevention Trial Combining Pacritinib With Sirolimus-Based Immune Suppression
Contact the study team to confirm eligibility.
Primary Completion
August 21, 2020
Actual (reported)Study Completion
August 21, 2020
Actual (reported)Last Updated
July 12, 2021
Results First Posted
May 24, 2021
Record last verified: 2021-06
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Source: ClinicalTrials.gov.
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