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Emapalumab for Preventing Graft-versus-Host Disease After Transplant

This study is testing a new approach to prevent graft-versus-host disease (GVHD) in people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who are having a donor stem cell transplant. You would receive emapalumab along with standard medications (cyclophosphamide, tacrolimus, and mycophenolate mofetil). Researchers want to see how safe this combination is, looking for serious side effects and infusion reactions within 28 days after transplant. They also want to see how well it prevents GVHD and improves survival. The study aims to enroll 15 participants aged 18 to 75 years old.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It is a Phase I trial, focusing on safety and side effects, and plans to enroll 15 participants.
What's involved
You would undergo blood sample collection, receive medications like Busulfan, Cyclophosphamide, and Emapalumab, and have procedures such as chest CT scans and echocardiograms.
Compensation
Not stated in the trial record.
Follow-up
The study will assess the incidence of acute GVHD at 100 days and chronic GVHD at 1 year post-transplant. Overall survival and progression-free survival will also be estimated at 1 year post-transplant.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06996119

Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant

Recruiting
PHASE1Ages 18–75InterventionalTreatment
City of Hope Medical Center
~15 participants
Updated 2026-07-01 on ClinicalTrials.gov
What's tested:Biospecimen CollectionBusulfanComputed TomographyCyclophosphamideEchocardiography TestEmapalumab

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of grade 3 or higher adverse events (AEs)
Measured over From starting the first emapalumab dose to the first observation of a primary safety endpoint (PSE) event or day 28 post-hematopoietic cell transplantation (HCT), whichever comes first
+3 more outcomes measured

Conditions

Acute Myeloid Leukemia
Graft Versus Host Disease
Myelodysplastic Syndrome

Explore related trials

Acute Myeloid LeukemiaMyelodysplastic Syndrome

Where it's being run

1 sites across 1 states
California1

Study leadership

  • Amandeep Salhotra · PRINCIPAL_INVESTIGATOR · City of Hope Medical Center

Who to contact

Amandeep Salhotra
[email protected]
626-218-2405
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Documented informed consent of the participant and/or legally authorized representative
Assent, when appropriate, will be obtained per institutional guidelines
Age: ≥ 18 years and ≤ 75 years
Note: Patients \> 70 years of age must have Karnofsky performance status ≥ 80% and HCT-comorbidity index (CI) ≤ 2
Karnofsky performance status ≥ 70%
Patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) in complete remission with bone marrow (BM) blast of \< 5%. AML must be negative for minimal residual disease (MRD-)
Planned to undergo reduced-intensity conditioning (RIC) with either fludarabine/melphalan (Flu/Mel) or busulfan/fludarabine (Bu/Flu) regimens prior to an allogeneic HCT using a mobilized peripheral blood stem cell (PBMC) graft from an 8/8 match related/unrelated donor (A, B, C, DR by high resolution typing)
Total bilirubin ≤ 1.5 x upper limit of normal (ULN) (unless has Gilbert's disease)
Aspartate aminotransferase (AST) ≤ 3.0 x ULN
Alanine aminotransferase (ALT) ≤ 3.0 x ULN
Creatinine clearance of ≥ 60 mL/min per 24-hour urine test or the Cockcroft-Gault formula
Left ventricular ejection fraction (LVEF) ≥ 50%
Note: To be performed within 30 days prior to day 1 of protocol therapy
Bazett's correction formula (QTcB) ≤ 480 ms
If able to perform pulmonary function tests: forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC) and diffusion capacity of the lung for carbon monoxide (DLCO) (diffusion capacity) ≥ 50% of predicted (corrected for hemoglobin).
If unable to perform pulmonary function tests: Oxygen (O2) saturation \> 92% on room air
Seronegative for HIV antigen (Ag)/antibody (Ab) combo, hepatitis C virus (HCV), active hepatitis B virus (HBV) (surface antigen negative) OR
If seropositive for HIV, HCV or HBV, nucleic acid quantitation must be performed. Viral load must be undetectable
Meets other institutional and federal requirements for infectious disease titer requirements
Note Infectious disease testing to be performed within 28 days prior to day 1 of protocol therapy
QuantiFERON-TB Gold+
Administer tuberculosis prophylaxis to patients at risk for tuberculosis, or known to have a positive purified protein derivative (PPD) test result, or positive interferon gamma (IFNγ) release assay
Women of childbearing potential (WOCBP): negative urine or serum pregnancy test
If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least 180 days post-HCT
Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for \> 1 year (women only)

Exclusion

Prior allogeneic HCT
Other cancer therapies (chemotherapy, radiation, biologics) are not allowed within two weeks of starting HCT conditioning; however targeted agents for underlying hematologic malignancies may be continued up to one day before conditioning, including, but not limited to:
FLT3 inhibitors
IDH1/2 inhibitors
Menin inhibitors
ABL-BCR inhibitors
BCL-2 inhibitors
Hydroxyurea
History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agent
Psychological issues, no appropriate caregivers identified, or non-compliant to medication
Clinically significant uncontrolled illness
Active uncontrolled infections (bacterial, viral, fungal). Infections are considered controlled if appropriate therapy has been initiated and, at the time of screening, no signs of infection are present
Other active malignancy
Females only: Pregnant or breastfeeding
Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures
Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)

What this trial measures

  • Incidence of grade 3 or higher adverse events (AEs)From starting the first emapalumab dose to the first observation of a primary safety endpoint (PSE) event or day 28 post-hematopoietic cell transplantation (HCT), whichever comes first

    Will be based on Common Terminology Criteria for Adverse Events (CTCAE) version (v) 5.0, with the exception of infections which ill be graded based on the Report of the Bone Marrow Transplant (BMT) Clinical Trials Network (CTN) Infections Disease Technical Committee. The toxicity/AE information recorded on each subject will include type, severity, duration, and attribution/ association with the study regimen. Tables will be constructed to summarize the observed incidence, severity and type of toxicity, including infection.

  • Incidence of severe infusion reactionFrom starting the first emapalumab dose to the first observation of a PSE event or day 28 post-HCT, whichever comes first

    Will be defined as grade 4 per CTCAE v 5.0 after receiving the first or second dose of emapalumab. The toxicity/AE information recorded on each subject will include type, severity, duration, and attribution/ association with the study regimen. Tables will be constructed to summarize the observed incidence, severity and type of toxicity, including infection.

  • Incidence of primary graft failureFrom starting the first emapalumab dose to the first observation of a PSE event or day 28 post-HCT, whichever comes first

    Will be defined as the failure to achieve an absolute neutrophil count of 500/ul or more for 3 days and donor chimerism of less than 5%. Will be calculated using the competing risk method as described by Gooley et al. (1999).

  • Non-relapse mortality (NRM)From starting the first emapalumab dose to the first observation of a PSE event or day 28 post-HCT, whichever comes first

    Will be calculated using the competing risk method as described by Gooley et al. (1999).