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About
The researchers are doing this study to find out whether emapalumab or a combination of fludarabine and dexamethasone are effective in preparing people with a primary immune regulatory disorder (PIRD) and/or an autoinflammatory condition to receive a stem cell transplant. The researchers will look at how well the study treatments reduce inflammation and aid in the engraftment process (the process of donated stem cells traveling to the bone marrow, where they begin to make new immune cells.
"Funding Source - FDA OOPD"
Enrollment
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Inclusion criteria
Patients receiving first allo-HCT for the following immunologic conditions:
Primary Immune Regulatory Disorder with or without a genetic lesion as defined by the Primary Immune Deficiency Treatment Consortium (PIDTC)
Patients with autoinflammatory disorders evidenced by cytokine or inflammation assays with at least 1.5x ULN of measured cytokines and/or an elevated ferritin or ESR > 2 ULN
For inclusion on the emapalumab group, the lesion must be isolated to the IFNγ pathway (or mediators thereof) with an elevated CXCL9 >1.5 ULN OR sIL2R >1.5 ULN (or already controlled on immune modulation, provided that CXCL9 or sIL2R levels were elevated prior to initiation of immune modulation). Inclusion on the Fludarabine/dexamethasone group requires inflammation (as defined above) other than an isolated IFNγ pathway
Able to tolerate cytoreduction (based on adequate organ function as described below)
Patients of any age can enroll so long as they meet other inclusion criteria:
Adequate organ function is required, defined as follows:
Adequate performance status:
Each patient must be willing to participate as a research subject and must sign an informed consent form or legal guardian with assent as appropriate.
Exclusion criteria
Donor Inclusion Criteria:
Related Donors:
Unrelated Donors:
o 8/8 or 7/8 matched at A, B, C, and DRB1 loci, as tested by DNA analysis.
Able to provide informed consent for the donation process per institutional standards.
Meet standard criteria for donor collection (e.g. National Marrow Donor Program Guidelines or collecting center guidelines as approved by treating physician).
Primary purpose
Allocation
Interventional model
Masking
39 participants in 2 patient groups
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Central trial contact
Joseph Oved, MD; Roni Tamari, MD
Data sourced from clinicaltrials.gov
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