Actively Recruiting
Pilot Study of Emapalumab With Post-Transplant Cyclophosphamide as Graft-Versus-Host Disease Prophylaxis for Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation
Led by City of Hope Medical Center · Updated on 2026-05-11
15
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
C
City of Hope Medical Center
Lead Sponsor
N
National Cancer Institute (NCI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating a treatment approach to prevent graft-versus-host disease (GVHD) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who undergo reduced-intensity donor hematopoietic cell transplantation (HCT). GVHD occurs when donor stem cell graft cells attack the patient's normal tissues, which can complicate recovery after transplant. This phase I trial aims to test the safety, side effects, and effectiveness of combining emapalumab with post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil to prevent GVHD in this patient group. Participants receive chemotherapy drugs such as fludarabine, melphalan, or busulfan before transplant to eliminate cancer cells and prepare the bone marrow. On day 0, patients get an infusion of donor stem cells. Emapalumab is given intravenously on days -8, -7, -1, 14, and 21. Cyclophosphamide is given on days 3 and 4, tacrolimus from days 5 to 95, and mycophenolate mofetil from days 5 to 35 either intravenously or orally. Additional procedures include chest CT scans, echocardiography or multigated acquisition scans, and blood sample collections. Throughout the study, participants are monitored for adverse events and GVHD occurrence up to one year after transplant. Researchers assess immune cell recovery, infection rates, relapse, survival, and patient quality of life using questionnaires at various time points. Biomarkers and the gut microbiome are also studied to understand treatment effects. Follow-up visits occur at 100 days, 6 months, and 1 year post-transplant to evaluate outcomes and safety.
CONDITIONS
Brief Title
Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provided documented informed consent
- Age between 18 and 75 years
- Karnofsky performance status of 70% or higher
- Diagnosis of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) in complete remission with bone marrow blast less than 5% and AML negative for minimal residual disease
- Planned reduced-intensity conditioning with fludarabine/melphalan or busulfan/fludarabine before allogeneic hematopoietic cell transplant using an 8/8 matched related or unrelated donor graft
- Total bilirubin less than or equal to 1.5 times upper limit of normal unless Gilbert's disease
- Aspartate aminotransferase (AST) less than or equal to 3 times upper limit of normal
- Alanine aminotransferase (ALT) less than or equal to 3 times upper limit of normal
- Creatinine clearance of 60 mL/min or higher
- Left ventricular ejection fraction of 50% or higher within 30 days before protocol therapy
- QTcB less than or equal to 480 ms
- Pulmonary function tests (FEV1, FVC, DLCO) at least 50% predicted or oxygen saturation over 92% on room air
- Seronegative for HIV, hepatitis C, and active hepatitis B, or undetectable viral load if positive
- Meet institutional and federal infectious disease titer requirements
- QuantiFERON-TB Gold positive with prophylaxis if at risk
- Negative pregnancy test for women of childbearing potential
- Agreement to use effective birth control or abstain from heterosexual activity during the study and up to 180 days post-transplant
You will not qualify if you...
- Prior allogeneic hematopoietic cell transplant
- Other cancer therapies within two weeks before transplant conditioning, except certain targeted agents allowed until one day before conditioning
- History of allergic reactions to similar study agents
- Psychological issues, lack of appropriate caregivers, or medication non-compliance
- Clinically significant uncontrolled illness
- Active uncontrolled infections (bacterial, viral, fungal)
- Other active malignancies
- Pregnant or breastfeeding women
- Any condition judged by the investigator to contraindicate participation due to safety concerns
- Inability to comply with study procedures due to feasibility or logistics issues
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 screening and enrollment visit
Duration - 7 to 8 days
Participants receive conditioning chemotherapy with fludarabine and melphalan or busulfan and fludarabine before transplantation.
Daily visits for chemotherapy administration from day -8 to day -2
Duration - Up to 100 days post-transplant
Participants receive hematopoietic cell transplantation followed by treatment with emapalumab, cyclophosphamide, tacrolimus, and mycophenolate mofetil as graft-versus-host disease prophylaxis.
Multiple visits including infusion on day 0, emapalumab on days -8, -7, -1, 14, and 21, cyclophosphamide on days 3 and 4, tacrolimus daily from days 5 to 95, and mycophenolate mofetil daily from days 5 to 35
Duration - Up to 1 year post-transplant
Participants are monitored for safety, graft-versus-host disease, immune recovery, infections, and quality of life after treatment completion.
Follow-up visits at day 100, 6 months, and 1 year post-transplant
Trial Site Locations
Total: 1 location
1
City of Hope Medical Center
Duarte, California, United States, 91010
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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