NCT05787574 · Memorial Sloan Kettering Cancer Center
A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition
What this study 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.
View original scientific description
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.
Interventions
DRUG
Emapalumab
Emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1.
DRUG
Fludarabine and Dexamethasone
Fludarabine and dexamethasone for 5 days in a row on Days -22 through -18.
PROCEDURE
Allogeneic hematopoietic stem cell transplant (allo-HCT)
Participants in both groups will receive their standard-of-care stem cell transplant on Day 0.
Primary outcome measures
Engraftment
Time frame: Day 100 post-alloHCT
Engraftment is defined as the first of three days of absolute neutrophil count \>500,000/µL and the first of seven days of platelets \>20,000/µL in the absence of transfusional support.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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 inclus
Where
- San Francisco, California
- Atlanta, Georgia
- Basking Ridge, New Jersey
- Middletown, New Jersey
- Montvale, New Jersey
- Commack, New York
- Harrison, New York
- New York, New York
- Rockville Centre, New York
- Philadelphia, Pennsylvania
- Houston, Texas
- Milwaukee, Wisconsin
Collaborators
Sobi, Inc.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 2, 2026 · Source of record for eligibility and locations