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Lower Dose Cyclophosphamide After Stem Cell Transplant

This study is for people with certain blood cancers who are undergoing a stem cell transplant. After a transplant, a common problem called graft-versus-host disease (GVHD) can occur. The study is testing if a lower dose of a drug called cyclophosphamide, given after the transplant, works better to prevent GVHD. Participants will also receive sirolimus and mycophenolate mofetil. Researchers want to find the lowest effective dose of cyclophosphamide that still protects against GVHD, while also reducing potential side effects. The study will look at how many people are free from GVHD and relapse one year after the transplant. You may be able to join if you are between 12 and 120 years old and have a confirmed hematologic malignancy (blood cancer) that requires a stem cell transplant.

Study design
This is an interventional study with a planned enrollment of 260 participants. It has both a Phase I to find the best dose and a Phase II to evaluate its effectiveness.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will follow participants for at least one year to assess GVHD-free relapse-free survival and for 60 days to assess primary graft failure and severe acute GVHD.

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

NCT05436418

The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation

Recruiting
PHASE1Ages 12+InterventionalTreatment
National Cancer Institute (NCI)
~260 participants
Updated 2026-09-11 on ClinicalTrials.gov
What's tested:MelphalanSirolimusTotal Body Irradiation (TBI)CyclophosphamideMycophenolate MofetiFludarabine

At a glance

Recruiting sites
1 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Phase II: Evaluate the efficacy of PTCy, at the lowest dose determined for each HLA-matching arm from phase I, as assessed by 1-year GVHD-free relapse-free survival (GRFS) rate.
Measured over 1 year
+1 more outcome measured

Conditions

Peripheral Blood Stem Cell Transplantation
Hematopoietic Stem Cell Transplantation

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NCT05436418

Where you'd take part

This study runs at 2 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • City of Hope

    Duarte, Californiastudy coordinator listed

    Not yet recruiting

  • National Institutes of Health Clinical Center

    Bethesda, Marylandstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Christopher G Kanakry, M.D. · PRINCIPAL_INVESTIGATOR · National Cancer Institute (NCI)

Who to contact

Amy H Chai
[email protected]
(240) 858-3755
Email the study team

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Eligibility criteria

Inclusion

Participants must have a histologically or cytologically confirmed hematologic malignancy with standard indication for allogeneic hematopoietic cell transplantation limited to one of the following:
Acute myeloid leukemia (AML) of intermediate or adverse risk disease by the 2017 European LeukemiaNet criteria in first morphologic complete remission (\<5% blasts in the bone marrow, no detectable abnormal peripheral blasts, and no extramedullary disease)
AML of any risk in second or subsequent morphologic complete remission
Acute lymphoblastic leukemia in first or subsequent complete remission
Myelodysplastic syndrome of intermediate or higher score by the Revised International Prognostic Scoring System (IPSS-R)
Primary myelofibrosis of intermediate-2 or higher risk by the DIPSS
Chronic myelomonocytic leukemia
Chronic myelogenous leukemia resistant to or intolerant of \>= 3 tyrosine kinase inhibitors or with history of accelerated phase or blast crisis
B-cell lymphoma including Hodgkin lymphoma that has relapsed within 1 year of completion of primary treatment, relapsed after autologous transplantation, or has progressed through at least 2 lines of therapy
Chronic lymphocytic leukemia with 17p deletion and/or unmutated IgHV or refractory to or intolerant of both BTK and PI3K inhibitors
Mature T or NK neoplasms as defined in the WHO guidelines of sufficient type and severity for allogeneic HCT based on the Prognostic Index for T-cell lymphoma (PIT) score of low-intermediate risk or higher or on recently published clinical practice guidelines
Hematologic malignancy of dendritic cell or histiocytic cell type
Multiple myeloma, stage III, relapsing after therapy with both a proteasome inhibitor and an immunomodulatory drug (IMiD)
Age \>= 50 years or age 18-49 years and also meeting one of the following criteria:
Prior myeloablative HCT
Prior exposure to inotuzumab, gemtuzumab, or other agent that increases the risk for sinusoidal obstruction syndrome.
Hematopoietic Cell Transplantation- Comorbidity Index (HCT-CI) \>= 3
Karnofsky performance score \<80
Co-morbidity considered by the treating physician to be exclusionary of myeloablative conditioning
At least one potentially suitable HLA-haploidentical or 10/10 (HLA-A, B, C, DR, DQ) related or unrelated donor for HCT
Karnofsky performance score \>= 70
Adequate organ function defined as possessing all of the following:
Cardiac ejection fraction \>= 45% by 2D ECHO;
Forced expiratory volume-1 (FEV-1), forced vital capacity (FVC), and diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) all of \>= 50% predicted;
Estimated serum creatinine clearance of \>= 60 ml/minute/1.73m\^2 calculated using eGFR in the clinical lab;
Total bilirubin \<= 2X the upper limit of normal;
Alanine aminotransferase and aspartate aminotransferase \<= 3X the upper limit of normal.
Individuals of child-bearing potential (IOCBP) and participants who can father children must agree to use highly effective contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for at least one year post-transplant.
IOCBP must have a negative serum or urine pregnancy test within 7 days prior to initiation of conditioning regimen.
Ability of participant to understand and the willingness to sign a written informed consent document.
Related (age \>=12) and unrelated (age \>=18) donors deemed eligible (i.e., evaluated at NIH, COH, and FHCC in accordance with existing institutional Standard Policies and Procedures or evaluated per the standards required by the IRB of the National Marrow Donor Program or applicable registry), and willing to donate research samples will be included.
Ability of participant or parent/legal guardian to understand and the willingness to sign a written informed consent document.

Exclusion

Participants who are receiving any other investigational agents. Prior experimental therapies must have been completed at least 2 weeks prior to the date of beginning conditioning.
Active nursing.
Active malignancy of non-hematopoietic type (excluding non-melanoma skin cancers) which is: metastatic, or relapsed/refractory to treatment, or locally advanced and not amenable to curative treatment, or limited disease treated with curative intent treatment within the last 2 years. This excludes non-melanoma skin cancers.
History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agents.
Uncontrolled intercurrent illness (e.g., severe endocrinopathy, disseminated intravascular coagulation, profound electrolyte disturbance, active infectious hepatitis, uncontrolled dental infection) that in the opinion of the Site PI would make it unsafe to proceed with transplantation.

What this trial measures

  • Phase II: Evaluate the efficacy of PTCy, at the lowest dose determined for each HLA-matching arm from phase I, as assessed by 1-year GVHD-free relapse-free survival (GRFS) rate.1 year

    1-year GRFS and 95% CI per arm will be estimated using Kaplan-Meier curves.

  • Phase I: Determine the lowest effective dose of PTCy in combination with sirolimus and mycophenolate mofetil as GVHD prophylaxis after reduced intensity conditioning and PBSCT, as assessed by primary graft failure AND Grade III-IV acute GVHD as ...60 days

    Number of evaluable subjects and DLT will be summarized per dose level in each arm.