What is emapalumab studying for primary immune regulatory disorders and autoinflammatory conditions?

Emapalumab is being studied to see if it can reduce inflammation and help prepare patients with primary immune regulatory disorders or autoinflammatory conditions for stem cell transplant. The study compares emapalumab to fludarabine and dexamethasone to improve transplant success. This research is led by Joseph Oved and funded by the FDA Office of Orphan Products Development.

Who may be eligible for this primary immune disorder clinical trial?

People of any age with a primary immune regulatory disorder or autoinflammatory condition may be eligible. Participants must be receiving their first stem cell transplant and have adequate organ function. Both males and females can join, but pregnant or breastfeeding women cannot participate. Please review eligibility criteria for more details.

Is there any cost to participate in this primary immune disorder clinical trial?

Study-related care, including the study drugs and all trial visits, is provided at no cost. However, personal expenses such as travel or lodging may vary and are not guaranteed to be covered. Participants should plan accordingly for any additional costs.

Actively Recruiting

Phase 2

All Genders

ID05787574

Phase 2 Trial of Emapalumab or Fludarabine and Dexamethasone to Reduce Inflammation Before Stem Cell Transplant in People with Primary Immune Regulatory Disorders or Autoinflammatory Conditions

Led by Joseph Oved · Updated on 2026-06-02

Participants Needed

Research Sites

N/A

Total Duration

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 andor 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

All Genders

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

Research Team

Joseph Oved, MD

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