A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition
Eligible age
All ages
Accepts
All genders
Locations
7 states
Healthy volunteers
No
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About this study
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"
Sponsor: Joseph Oved
You may qualify if…
- ✓ 1. 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)12 - Patients with autoinflammatory disorders evidenced by cytokine or inflammation assays with at least 1.5x ULN of measured cytokines (defined in section 4) and/or an elevated ferritin or ESR \> 2 ULN
- ✓ For inclusion on the emapalumab group, the lesion must be related 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 IFNy pathway defect
- ✓ 2. Able to tolerate cytoreduction (based on adequate organ function as described below)
- ✓ 3. Patients of any age can enroll so long as they meet all other eligibility criteria
- ✓ 4. Adequate organ function is required, defined as follows:
- ✓ Hepatic: Serum bilirubin ≤ 2 mg/dL, unless benign congenital hyperbilirubinemia. Patients with hyperbilirubinemia related to paroxysmal nocturnal hemoglobinuria or other hemolytic disorders related to their PIRD diagnosis are eligible.
You may not qualify if…
- ✕ 1. Uncontrolled infection at the time of enrollment.
- ✕ 2. Patients who have undergone previous allo-HCT.
- ✕ 3. Patient seropositivity for HIV I/II and/or HTLV I/II.
- ✕ 4. Females who are pregnant or breastfeeding.
- ✕ 5. Patients unwilling to use contraception during the study period.
- ✕ 6. Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.
Where it's recruiting
San Francisco
Atlanta
Basking Ridge · Middletown · Montvale
Commack · Harrison · New York · Rockville Centre
Philadelphia
Houston
Milwaukee
Source: ClinicalTrials.gov · NCT05787574 · last updated 2026-06-02