Actively Recruiting
A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition
Led by Memorial Sloan Kettering Cancer Center · Updated on 2025-10-29
39
Participants Needed
12
Research Sites
206 weeks
Total Duration
On this page
Sponsors
M
Memorial Sloan Kettering Cancer Center
Lead Sponsor
S
Sobi, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is 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"
CONDITIONS
Official Title
A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Receiving first allogeneic stem cell transplant for Primary Immune Regulatory Disorder or autoinflammatory disorders
- Primary Immune Regulatory Disorder defined by the Primary Immune Deficiency Treatment Consortium
- Autoinflammatory disorders with elevated cytokines or inflammation markers (1.5x upper limit of normal or higher)
- For emapalumab group: isolated IFN-gamma pathway lesion with elevated CXCL9 or sIL2R levels
- For fludarabine/dexamethasone group: inflammation other than isolated IFN-gamma pathway
- Able to tolerate cytoreduction with adequate organ function including:
- Liver: Bilirubin ≤ 2 mg/dL (with exceptions), AST, ALT, and alkaline phosphatase < 2.5x upper limit unless disease-related
- Kidney: Creatinine < 1.5 times normal for age or creatinine clearance > 50 mL/min/1.73m2 or GFR > 30% predicted
- Heart: Left ventricular ejection fraction ≥ 50%
- Lungs: Pulmonary function tests ≥ 50% predicted or oxygen saturation ≥ 92% on room air
- Adequate performance status (ECOG ≤ 1 or Karnofsky/Lansky ≥ 70%)
- Willingness to participate and provide informed consent or assent as appropriate
You will not qualify if you...
- Uncontrolled infection at enrollment
- Previous allogeneic stem cell transplant
- Positive for HIV I/II or HTLV I/II
- Pregnant or breastfeeding females
- Unwillingness to use contraception during the study
- Inability to provide informed consent or comply with study procedures
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 12 locations
1
University of California, San Francisco (Data collection only)
San Francisco, California, United States, 94143
Actively Recruiting
2
Children's Healthcare of Atlanta (Data Collection Only)
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities)
Basking Ridge, New Jersey, United States, 07920
Actively Recruiting
4
Memorial Sloan Kettering Monmouth (Limited Protocol Activities)
Middletown, New Jersey, United States, 07748
Actively Recruiting
5
Memorial Sloan Kettering Bergen (Limited Protocol Activities)
Montvale, New Jersey, United States, 07645
Actively Recruiting
6
Memorial Sloan Kettering Suffolk - Commack (Limited Protocol Activities)
Commack, New York, United States, 11725
Actively Recruiting
7
Memorial Sloan Kettering Westchester (Limited Protocol Activities)
Harrison, New York, United States, 10604
Actively Recruiting
8
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
9
Memorial Sloan Kettering Nassau (Limited Protocol Activities)
Rockville Centre, New York, United States, 11553
Actively Recruiting
10
Children's Hospital of Philadelphia (Data Collection Only)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
11
Texas Children's Hospital (Data Collection)
Houston, Texas, United States, 77030
Actively Recruiting
12
Children's Hospital of Wisconsin (Data Collection Only)
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
Research Team
A
Andromachi Scaradavou, MD
CONTACT
R
Roni Tamari, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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