A Phase 2 interventional study of Emapalumab and Fludarabine and Dexamethasone in Primary Immune Regulatory Disorder, Autoimmune Lymphoproliferative and Immune System Diseases, sponsored by Joseph Oved. Recruiting at 13 sites in United States. Per ClinicalTrials.gov, last updated 2026-06-02.
Sponsored by Joseph Oved · Phase 2, Interventional, and Treatment
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"
Defects in the immune cells can hinder the immune system's ability to fight infection. A stem cell transplant can replace non-working immune system cells with a donor's fully functioning immune cells, but if inflammation is present, the immune cells from the donor may not graft successfully. The proposed trial tests whether emapalumab or fludarabine and dexamethasone can help prepare the body to receive a stem cell transplant by reducing inflammation, so that the immune system will be able to produce fully functioning immune cells. Participants will receive emapalumab or a combination of fludarabine and dexamethasone over the course of four days in the hospital prior to a planned transplant procedure.
This study has two study groups:
All study participants are eligible to receive one additional emapalumab 3mg/kg in the first 30 days post-transplant if they begin to have CXCL9 levels that are trending up. Participants will receive personalized rATG dosing. Participants will remain in the hospital according to the usual standard of care guidelines for stem cell transplant. Subject status and follow-up examination/data collection will occur on days 0, 7, 14, 21,30, 45, 60, 7, 100, 180, 270, and 365, then quarterly until 3 years post-transplant.
279 studies on the registry are indexed under Immune System Diseases; 81 are open to participants now.
This study's planned enrollment of 39 is below the median of 65 across 188 interventional studies indexed under Immune System Diseases.
Browse Immune System Diseases studies →This is the only study on the registry with Joseph Oved as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Patients receiving first allo-HCT for the following immunologic conditions:
Adequate organ function is required, defined as follows:
Adequate performance status:
Donor Inclusion Criteria:
Related Donors:
Unrelated Donors:
Exclusion Criteria:
Participants in this group will receive emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1.
Drug: Emapalumab · Procedure: Allogeneic hematopoietic stem cell transplant (allo-HCT)
Participants in this group will receive fludarabine and dexamethasone for 5 days in a row on Days -22 through -18.
Drug: Fludarabine and Dexamethasone · Procedure: Allogeneic hematopoietic stem cell transplant (allo-HCT)
Emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1.
Also known as: Gamifant
Fludarabine and dexamethasone for 5 days in a row on Days -22 through -18.
Participants in both groups will receive their standard-of-care stem cell transplant on Day 0.
Also known as: stem cell transplant
Engraftment
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.
Time frame: Day 100 post-alloHCT
Overall Survival (OS)
Overall Survival (OS) is defined as the duration of time between allo-HCT and death due to any cause.
Time frame: 1 year
Event Free Survival (EFS)
Event-Free Survival (EFS) is the length of time from the start of a treatment or study until a patient experiences a defined negative "event" (such as disease recurrence, progression, or death).
Time frame: Up to 2 years post-alloHCT
Non-Relapse Mortality (TRM)
Participants who die for any reason after disease recurrence are considered to have experienced disease related mortality, and will be counted as competing events. Patients alive will be censored at the day of last follow up.
Time frame: 1 year
GVHD free/relapse free survival (GRFS)
Duration of time between transplantation and development of grade III-IV GVHD, chronic GVHD requiring systemic therapy, disease relapse, or death, whichever occurs first.
Time frame: 1 year
Chronic graft vs host disease/relapse free survival (CRFS)
Duration of time between transplantation and development of moderate or severe chronic GVHD (NIH consensus criteria), or death, whichever occurs first.
Time frame: 1 year
All-cause mortality at 1 & 2 years
Time frame: 1 year and 2 years post-alloHCT
Incidence of viral reactivations
Incidence of viral reactivations including: Cytomegalovirus (CMV), Adenovirus, Epstein-Barr Virus (EBV), Human Herpes-6 (HHV6), BK-virus defined as viral reactivations necessitating use of anti-viral treatment.
Time frame: 1 year
Incidence of Acute graft versus host disease (GVHD) at Day 100, 180, and 1-year follow-up
Time frame: Day 100, 180, and 1 year post-alloHCT
Incidence of Chronic GVHD at Day 180 and 1-year follow-up
Time frame: Day 180 and 1 year post-alloHCT
Time to neutrophil engraftment
Neutrophil engraftment is defined as the first of 3 consecutive days of absolute neutrophil count (ANC) ≥ 500 K/µL.
Time frame: 100 days
Time to platelet engraftment
Platelet engraftment is defined as the first of 7 consecutive days with a platelet count exceeding 20,000/µL without transfusion support.
Time frame: 100 days
Incidence of primary engraftment failure
Primary engraftment failure is defined as failure to achieve neutrophil engraftment by Day 30 after transplantation.
Time frame: Day 30 post-alloHCT
Incidence of secondary engraftment failure
Secondary engraftment failure is defined as \<500/µL circulating neutrophils at any time after primary engraftment that is not attributed to disease recurrence or drug therapy.
Time frame: 1 year post-alloHCT
Donor Chimerism
The percentage of donor contribution to hematopoiesis (chimerism) will be determined using short tandem repeat (STR) based analysis of the peripheral blood (Chimerism - all lineages on Day 30, 100, 180, and 365) and bone marrow (all samples obtained post-alloHCT).
Time frame: Day 30, 100, 180, and 1 year post-alloHCT
Incidence of grade ≥ 3 non-hematologic adverse events
Incidence of grade ≥ 3 non-hematologic adverse events as defined by CTCAE version 5.0 at Day 100, 180, and 1 year post allo-HCT.
Time frame: Day 100, 180, and 1 year post-alloHCT
Lymphocyte Reconstitution
CD4+ Immune Reconstitution (CD4+IR) is defined at CD4+ \> 50u/L at two consecutive measures within 100 days post-alloHCT. Lymphocyte subsets (includes CD3, CD4, CD8, CD19, CD56+16, CD45RA, CD45) will be documented by flow cytometry.
Time frame: Day 30, 60, 100, 180, and 1 year and 2 years post-alloHCT
Quality of Life (PROMIS score)
Patient-reported outcomes will be self-reported using the PROMIS Profile Measure Scores. Scores are reported as standardized T-scores based on a U.S. general population average of 50 and a standard deviation of 10.
Time frame: up to 3 years post-alloHCT
Percentage of patients who are eligible to proceed to transplant after receiving an immune suppression prophase
Time frame: 30 days
Plan to share: Yes — The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals.
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