Using Methotrexate to Prevent Graft Versus Host Disease in Haploidentical Bone Marrow Transplants

GVHD Prophylaxis With Methotrexate and Cyclosporine in Haploidentical Stem Cell Transplantation Using Posttransplant Cyclophosphamide in Hematologic Malignancies: Phase I/II Trial

Phase1; Phase2 Interventional University of Sao Paulo General Hospital · NCT04622956

This study is testing if using methotrexate can help prevent graft-versus-host disease in patients receiving bone marrow transplants from partially matched donors.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment47 (estimated)
Ages18 Years to 70 Years
SexAll
SponsorUniversity of Sao Paulo General Hospital Academic / other
Drugs / interventionsalemtuzumab, methotrexate, cyclophosphamide
Locations4 sites (Rio De Janeiro, RJ and 3 other locations)
Trial IDNCT04622956 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the use of methotrexate as a prophylactic treatment to prevent graft-versus-host disease (GVHD) in patients undergoing haploidentical hematopoietic cell transplantation (HCT). The study compares methotrexate with the commonly used combination of posttransplant cyclophosphamide and cyclosporine. Patients with specific hematologic malignancies who have a haploidentical donor will be enrolled, and their outcomes will be monitored to assess the effectiveness of methotrexate in reducing GVHD incidence. The trial aims to provide insights into the optimal GVHD prophylaxis strategy in this patient population.

Who should consider this trial

Good fit: Ideal candidates for this study include patients with specific hematologic malignancies in remission who have a haploidentical related donor.

Not a fit: Patients who have previously undergone allogeneic transplants or have uncontrolled infections may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly reduce the incidence of GVHD and improve long-term outcomes for patients undergoing haploidentical HCT.

How similar studies have performed: Previous studies have shown mixed results regarding the use of methotrexate for GVHD prophylaxis, indicating that while some approaches have been successful, this specific application in haploidentical HCT is still being explored.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Diagnosis of acute myeloid leukemia and chronic myeloid leukemia in complete morphologic remission, myelodysplastic syndrome with less than 10% in bone marrow or peripheral blood, Ph-negative acute lymphoblastic leukemia in complete morphologic remission, chemosensitive Hodgkin lymphoma or non-Hodgkin lymphoma in at least partial remission
* Donor type: haploidentical related donor
* Graft source: bone marrow or peripheral blood
* Recipients of non-myeloblative or myeloablative intensity conditioning
* Left Ventricle Ejection fraction \> 40%
* Estimated creatinine clearance \> 40 mL/min
* Adjusted DLCO ≥ 40% and FEV1 ≥ 40%
* Total bilirubin \< 2x ULN e ALT/AST \< 2.5x ULN

Exclusion Criteria:

* Prior allogeneic transplant
* Ex-vivo graft manipulation (T-cell-depleted or CD34-selected grafts)
* Use of alemtuzumab or anti-thymocyte globulin
* KPS \< 70%
* Patients with uncontrolled bacterial, viral or fungal infections (currently taking medication and with progression or no clinical improvement) at time of enrollment
* Pregnant or lactating women
* Patients seropositive for human immunodeficiency virus (HIV) or active hepatitis B or C infection by PCR
* Presence of fluid collection (ascites, pleural or pericardial effusion) that may interfere with methotrexate clearance or make methotrexate use contraindicated
* Patients with a serious medical or psychiatric illness likely to interfere with participation in this study

Where this trial is running

Rio De Janeiro, RJ and 3 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Graft Vs Host DiseaseHematopoietic NeoplasmMethotrexatePost-transplant cyclophosphamideCyclosporineHaploidenticalHematopoietic cell transplantation
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.