Actively Recruiting
Comparing Post-Transplant Cyclophosphamide with Calcineurin Inhibitors As a GVHD Prophylaxis to Standard Care of Methotrexate and Calcineurin Inhibitors for Acute Leukemia Incorporating Patient Pharmacogenomics Profiling
Led by King Faisal Specialist Hospital & Research Center · Updated on 2025-03-13
264
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating two different approaches to prevent graft-versus-host disease (GVHD) in patients with acute leukemia undergoing allogeneic hematopoietic stem cell transplantation (allo-HCT). This phase 3 randomized clinical trial compares the current standard GVHD prevention regimen using methotrexate and calcineurin inhibitors to a newer strategy using post-transplant cyclophosphamide combined with calcineurin inhibitors. The study includes patients aged 14 to 65 with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) in remission who have a matched related donor for stem cell transplantation. Participants are randomly assigned to one of two treatment groups. The experimental group receives a myeloablative conditioning regimen followed by matched related donor peripheral blood stem cell transplant, with cyclophosphamide given intravenously at 50 mg/kg/day on days +3 and +5 post-transplant, along with oral tacrolimus or another calcineurin inhibitor. The standard care group receives a similar transplant and conditioning regimen but with methotrexate given on days +1, +3, and +6 alongside calcineurin inhibitors for GVHD prevention. Both groups use myeloablative conditioning tailored to leukemia type, such as IV Busulfan/Fludarabine for AML or VP16/TBI for ALL. During the study, participants are monitored for graft-versus-host disease occurrence, survival without relapse, and other health outcomes for at least one year post-transplant. Researchers assess acute GVHD within 100 days and chronic GVHD within one year after transplant. Evaluations include cardiac, pulmonary, liver, and kidney function tests at baseline, as well as ongoing clinical monitoring. The study measures GVHD-free and relapse-free survival at one year to compare the effectiveness of the two prophylaxis strategies. The total duration of participation varies based on transplant and follow-up schedules.
CONDITIONS
Brief Title
Comparing Post-Transplant Cyclophosphamide As GVHD Prophylaxis to Standard of Care for Acute Leukemia Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with acute leukemias (AML, ALL) in morphologic complete remission with or without hematologic recovery
- Patients must have a fully matched (8/8) related donor willing to donate peripheral blood stem cells and must meet institutional criteria for donation
- Planned myeloablative conditioning regimen
- Cardiac function: ejection fraction at rest 250% by MUGA or TTE
- Estimated creatinine clearance greater than 50 mL/minute
- Pulmonary function: DLCO 250% (adjusted for hemoglobin), and FVC and FEV1 250%
- Liver function: total bilirubin less than 2x the upper limit of normal (unless elevated bilirubin is attributed to Gilbert's Syndrome) and ALT/AST less than 2.5x the upper normal limit
- Signed informed consent
You will not qualify if you...
- Karnofsky or Lansky Performance Score less than 70%
- Active disease
- Patients with uncontrolled bacterial, viral, or fungal infections
- Presence of fluid collection (ascites, pleural or pericardial effusion) that interferes with methotrexate clearance or makes methotrexate use contraindicated
- Patients seropositive for HIV-1 or -2
- Patients seropositive for HTLV-I or -II
- Patients with active Hepatitis B or C viral replication by PCR
- Women who are pregnant (positive serum or urine 2bHCG) or breastfeeding
- Females with childbearing potential or men who have sexual contact with them unwilling to use effective birth control or abstinence for one year after transplantation
- History of uncontrolled autoimmune disease or on active treatment
- Patients with prior malignancies, except resected non-melanoma skin cancer or treated cervical carcinoma in situ; cancer treated with curative intent 5 years or more ago will be allowed; cancer treated with curative intent less than 5 years ago will not be allowed.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 week starting from transplant day
Participants receive a hematopoietic stem cell transplant with a myeloablative conditioning regimen followed by GVHD prophylaxis. Depending on the assigned group, participants receive either cyclophosphamide on day +3 and day +5 or methotrexate on days +1, +3, and +6, along with tacrolimus or another calcineurin inhibitor.
Daily visits during the first week post-transplant
Duration - Up to 1 year post-transplant
Participants are monitored for graft-versus-host disease (GVHD), relapse, and overall health outcomes for up to one year after transplant.
Regular follow-up visits up to 12 months
Trial Site Locations
Total: 1 location
1
King Faisal Specialist Hospital & Research Center
Riyadh, Saudi Arabia
Actively Recruiting
Research Team
R
Riad O El Fakih, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
PREVENTION
Number of Arms
2
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