An observational study in GVHD, Bone Marrow Transplant Failure and Alpha 1-Antitrypsin, sponsored by Rambam Health Care Campus. Completed at 4 sites in Israel. Open to participants aged 18 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2023-04-04.
Sponsored by Rambam Health Care Campus · Observational
Create a personalized time and context curve of patient circulating α1-antitrypsin (AAT) levels and functions before hematopoietic stem cell transplantation and throughout progression into GVHD.
PRIMARY ENDPOINT 1. Serum AAT levels and activity, before myeloablative preconditioning, as well as on days (-3),0,7,14,28 from HSCT and every 21 days thereafter.
SECONDARY ENDPOINTS 1. Correlation between AAT patterns and:
This study will focus on each individual patient from the early point of myeloablative conditioning through HSCT and GVHD. The rationale for this individualized approach is to account for the anticipated variability in patient age and primary disease, background pathology and individual therapeutic course, considering the enormous heterogeneity of this condition. To compensate for this limitation, we intend to create an individualized algorithm, based on a novel dynamic biomarker, i.e., AAT functionality, individualized per patient and placed on a timeline, with the aim of minimizing future occurrences of GVHD, and by using readily available laboratory measurements. The study is designed around patient sample collection, there is no change in standard of care, therefore there is no intervention as well.
Three types of sample tubes will be collected per indicated time point:
After donor and recipient informed consent forms were signed, a single blood sample should be obtained from the donor on the day of transplant extraction. Time points for recipient's samples include the day of myeloablation and again immediately prior to HSCT (set as day 0 and possibly -3 in MUDs). Serum and lysed blood samples will be collected on days 7, 14, 28, and every 21 days thereafter, through the development and progression of GVHD (where relevant). Blood samples for FACS will be collected by days 0 and 28. Sample collection is planned to end within 1 year from HSCT, or two months after appearance of symptoms of GVHD, or two months after a change is introduced in the immunosuppressive therapy.
94 studies on the registry are indexed under Alpha 1-Antitrypsin Deficiency; 5 are open to participants now.
This study's enrollment of 27 is below the median of 109 across 27 observational studies indexed under Alpha 1-Antitrypsin Deficiency.
Browse Alpha 1-Antitrypsin Deficiency studies →Rambam Health Care Campus is the lead sponsor of 456 studies on the registry; 33 are open to participants now.
Counted across the registry records on this site, refreshed daily.
In every medical center in which a bone marrow transplantation is performed, allogeneic transplantation recipients will be recruited for the trial.
Exclusion Criteria:
Approximately 40 patients in total.No intervention planned.
Approximately 10 patients in total.No intervention planned.
Approximately 40 patients in total.No intervention planned.
Approximately 50 patients in total. No intervention planned.
Number of patients progressing into acute or chronic GVHD
Evaluation of patient circulating alpha-1-antitrypsin levels and enzymatic functions as a bio-marker for GVHD progression and grading
Time frame: Up to 1 year
Correlation between AAT pattern and survivability, response to immunosuppressive treatment. liver functions, immunocyte activities.
Correlation between AAT patterns and: * Circulating immune cell activation profiles on day of ablation, 28 days from HSCT and once GVHD is diagnosed. * Patient survival * Liver function tests * GVHD grade: skin manifestations, weight, GI and liver histopathology * Graft-versus-leukemia effect
Time frame: Up to 1 year
Plan to share: Yes — The clinical individual participant data will be further analyzed by other researchers after the study is completed.That is in order to combine this clinical data with other experiments' results. no
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This study is completed, as verified in Apr 2023. You cannot join it, but the record below documents what was studied.
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Alpha 1-Antitrypsin Deficiency→
Rambam Health Care Campus