Actively Recruiting
Phase 2 Study of Pre-Transplant Inflammation Treatment with Emapalumab or Fludarabine and Dexamethasone for Primary Immune Regulatory Disorders and Autoinflammatory Conditions
Led by Joseph Oved · Updated on 2026-06-02
39
Participants Needed
13
Research Sites
N/A
Total Duration
On this page
Sponsors
J
Joseph Oved
Lead Sponsor
S
Sobi, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating whether emapalumab or a combination of fludarabine and dexamethasone can effectively prepare people with primary immune regulatory disorders (PIRD) and/or autoinflammatory conditions for stem cell transplants. The study aims to see if these treatments reduce inflammation and help donor stem cells successfully engraft, enabling the immune system to produce fully functioning cells. This is a phase 2 study supported by the FDA Office of Orphan Products Development. Participants are assigned to one of two groups based on their inflammation type. Group A participants with a high CXCL9 cytokine level receive emapalumab on days -22, -15, -8, and -1 before the transplant. Group B participants with generalized inflammation receive fludarabine and dexamethasone for five consecutive days from days -22 to -18. All participants undergo a standard stem cell transplant on day 0 and may receive an additional emapalumab dose within 30 days post-transplant if inflammation markers rise. During the study, participants remain hospitalized according to usual care and have follow-up visits on days 0, 7, 14, 21, 30, 45, 60, 70, 100, 180, 270, and 365, then quarterly for up to three years. Researchers monitor engraftment success, survival rates, graft-versus-host disease incidence, immune recovery, quality of life, and other health outcomes. Data collection continues long-term to assess transplant effects and safety.
CONDITIONS
Brief 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...
- Patients receiving their first allogeneic hematopoietic stem cell transplant for primary immune regulatory disorder or autoinflammatory conditions
- For emapalumab group, lesion related to IFNγ pathway with elevated CXCL9 or sIL2R levels
- For fludarabine/dexamethasone group, inflammation other than IFNγ pathway defect
- Able to tolerate cytoreduction with adequate organ function including liver, kidney, heart, and lung
- Patients of any age can enroll
- Adequate performance status: ECOG ≤1 or Karnofsky ≥70% for age ≥16; Lansky ≥70% for age <16
- Willing to participate and able to provide informed consent or assent as appropriate
- Donors must meet HLA matching and standard collection criteria and provide informed consent
You will not qualify if you...
- Uncontrolled infection at enrollment
- Previous allogeneic stem cell transplant
- HIV I/II or HTLV I/II positive
- Female participants who are pregnant or breastfeeding
- Unwillingness to use contraception during the study
- Inability to give informed consent or comply with treatment and research procedures
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 22 days before transplant
Participants receive inflammation treatment with either emapalumab or fludarabine and dexamethasone to prepare the body for a stem cell transplant.
4 infusions for emapalumab group on Days -22, -15, -8, and -1; or 5 days of fludarabine and dexamethasone on Days -22 through -18
Duration - Day 0
Participants receive their standard-of-care allogeneic hematopoietic stem cell transplant.
1 inpatient procedure
Duration - Up to 3 years post-transplant
Participants are monitored with follow-up examinations and data collection to assess engraftment, survival, and health status up to 3 years after transplant.
Visits on Days 0, 7, 14, 21, 30, 45, 60, 70, 100, 180, 270, 365, then quarterly until 3 years post-transplant
Trial Site Locations
Total: 13 locations
1
University of California, San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
2
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
Memorial Sloan Kettering Basking Ridge
Basking Ridge, New Jersey, United States, 07920
Active, Not Recruiting
4
Memorial Sloan Kettering Monmouth
Middletown, New Jersey, United States, 07748
Active, Not Recruiting
5
Memorial Sloan Kettering Bergen
Montvale, New Jersey, United States, 07645
Active, Not Recruiting
6
Memorial Sloan Kettering Suffolk - Commack
Commack, New York, United States, 11725
Active, Not Recruiting
7
Memorial Sloan Kettering Westchester
Harrison, New York, United States, 10604
Active, Not Recruiting
8
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
9
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Active, Not Recruiting
10
Memorial Sloan Kettering Nassau
Rockville Centre, New York, United States, 11553
Active, Not Recruiting
11
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
12
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
13
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
Research Team
J
Joseph Oved, MD
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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