Actively Recruiting
Allogeneic Hematopoietic Stem Cell Transplantation With Low-Dose Post-Transplant Cyclophosphamide for Graft-versus-Host Disease Prevention in Blood Cancers
Led by Milton S. Hershey Medical Center · Updated on 2026-04-03
41
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of a low-dose post-transplant cyclophosphamide (PTCy) regimen to prevent graft-versus-host disease (GVHD) in patients receiving allogeneic stem cell transplants for blood cancers. This Phase 2, single-arm study focuses on patients undergoing reduced-intensity or non-myeloablative conditioning and receiving stem cells from matched sibling, matched unrelated, or haploidentical donors. The trial aims to see if a lower dose of PTCy can reduce toxicity while maintaining similar outcomes compared to higher doses. Participants will receive 25 mg/kg of PTCy on days +3 and +4 after their transplant, combined with tacrolimus and mycophenolate mofetil (MMF) for GVHD prevention. The study includes transplants from matched sibling, matched unrelated, single allelic mismatched unrelated, and haploidentical donors using peripheral blood stem cells. The conditioning regimens are either reduced-intensity or non-myeloablative. This approach is based on previous studies showing that lower PTCy doses may be effective with less toxicity. During the study, participants will be monitored for one year to assess outcomes such as survival without severe acute GVHD, chronic GVHD requiring treatment, and relapse or disease progression. Evaluations include regular clinical assessments of GVHD status and disease progression. The primary goal is to measure one-year GVHD-Free Relapse-Free Survival (GRFS), with ongoing safety monitoring and follow-up through the study duration ending in 2031.
CONDITIONS
Brief Title
Allogeneic HSCT With Low-Dose Post-Transplant Cyclophosphamide for GVHD Prevention
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 or older at the time of enrollment
- Diagnosis of acute leukemia (acute myeloid leukemia, acute lymphoblastic leukemia, mixed phenotype acute leukemia) or chronic myeloid leukemia with less than 5% blasts in bone marrow and no circulating blasts
- Diagnosis of myelodysplastic syndrome or chronic myelomonocytic leukemia with less than 10% blasts in bone marrow and no circulating blasts
- Diagnosis of secondary acute myeloid leukemia progressing from pre-existing myelodysplastic syndrome, myeloproliferative disease, or overlap syndrome
- Diagnosis of relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma indicated for allogeneic stem cell transplantation
- Diagnosis of lymphoma indicated for allogeneic stem cell transplantation, including follicular lymphoma, Hodgkin lymphoma, diffuse large B cell lymphoma, mantle cell lymphoma, peripheral T cell lymphomas, and Richter's transformation
- Planned reduced intensity or non-myeloablative conditioning regimen
- Availability of a related or unrelated peripheral blood stem cell donor: sibling donor at least haploidentical, children or parent donor at least haploidentical (children donors must be 18 or older), unrelated donor 7/8 or 8/8 HLA match by DNA typing
- Cardiac function with ejection fraction of at least 40%
- Pulmonary function with FEV1 at least 50% predicted and DLCO at least 40% predicted
- Karnofsky performance status of at least 70%
- Women of childbearing potential must have a negative pregnancy test before conditioning
- Female patients not post-menopausal or surgically sterilized must use two effective contraception methods or abstain from heterosexual intercourse from consent through 12 months post-transplant
- Male patients who are partners of women of childbearing potential must use effective barrier contraception or abstain from heterosexual intercourse from consent through 12 months post-transplant
You will not qualify if you...
- Prior allogeneic stem cell transplant
- Active central nervous system involvement by malignant cells
- Uncontrolled infections with progression or no clinical improvement on medication
- HIV, hepatitis B, or hepatitis C with detectable viral load (except hepatitis B surface antibody positive due to vaccination or immunity, or treated hepatitis C in sustained remission)
- Recent myocardial infarction within 6 months, severe heart failure (NYHA class III-IV), uncontrolled angina, severe ventricular arrhythmias, or acute ischemia on ECG
- Pregnant or lactating women (unless feeding via formula); women of childbearing potential must have negative pregnancy test before conditioning
- Serious medical or psychiatric illness likely to interfere with study participation
- Use of investigational agents
- Haploidentical related recipients positive for donor-specific antibodies ≥ 5000 MFI by Luminex
- Use of steroid doses over 10 mg/day within a week before registration
- Autoimmune disorders requiring active immunosuppression therapy
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 visit (in-person)
Duration - Treatment includes transplantation and PTCy administration on days +3 and +4 post-transplant; overall treatment duration depends on clinical management but focuses on early post-transplant period.
Participants receive allogeneic hematopoietic stem cell transplantation followed by low-dose post-transplant cyclophosphamide (PTCy) on days +3 and +4, combined with tacrolimus and mycophenolate mofetil (MMF) for graft-versus-host disease prevention.
Visits around transplantation and PTCy dosing as per clinical care; specific visit schedule depends on clinical management
Duration - Up to 1 year post-transplant
Participants are monitored for graft-versus-host disease, relapse, and overall health status for up to 1 year after transplant to assess outcomes and safety.
Regular follow-up visits during the first year post-transplant as per clinical protocol
Trial Site Locations
Total: 1 location
1
Penn State Cancer Institute
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
Research Team
C
Crystal Sowers
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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