Comparing two methods to prevent GVHD in leukemia patients after transplant
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
PHASE3 · King Faisal Specialist Hospital & Research Center · NCT04314219
This study is testing whether a new method using cyclophosphamide can better prevent graft-versus-host disease in leukemia patients after they receive a stem cell transplant compared to the usual treatment.
Quick facts
| Phase | PHASE3 |
|---|---|
| Study type | Interventional |
| Enrollment | 264 (estimated) |
| Ages | 14 Years to 65 Years |
| Sex | All |
| Sponsor | King Faisal Specialist Hospital & Research Center (other) |
| Drugs / interventions | methotrexate, cyclophosphamide |
| Locations | 1 site (Riyadh) |
| Trial ID | NCT04314219 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the effectiveness of post-transplant cyclophosphamide compared to the standard GVHD prophylaxis regimen in patients with acute leukemia who are in complete remission. It is a randomized, open-label trial involving patients aged 14 to 65 years undergoing allogeneic hematopoietic cell transplantation with a matched sibling donor. Participants will be assigned to either the intervention group receiving cyclophosphamide or the control group receiving standard care. The study aims to determine which approach better prevents graft-versus-host disease (GVHD).
Who should consider this trial
Good fit: Ideal candidates are patients aged 14 to 65 with acute leukemia in complete remission who have a fully matched sibling donor.
Not a fit: Patients with active disease or those with significant comorbidities may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved GVHD prevention strategies, enhancing patient outcomes after leukemia treatment.
How similar studies have performed: Other studies have shown promise with similar GVHD prophylaxis approaches, indicating potential for success in this trial.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 ≥ 50% by MUGA or TTE * Estimated creatinine clearance greater than 50 mL/minute * Pulmonary function: DLCO ≥ 50% (adjusted for hemoglobin), and FVC and FEV1 ≥ 50% * Liver function: total bilirubin \< 2x the upper limit of normal (unless elevated bilirubin is attributed to Gilbert's Syndrome) and ALT/AST \< 2.5x the upper normal limit * Signed informed consent Exclusion Criteria: * Karnofsky or Lansky Performance Score \< 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 βHCG) or breastfeeding * Females with childbearing potential (FCBP) or men who have sexual contact with FCBP unwilling to use effective forms of 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 previously will be allowed; cancer treated with curative intent \< 5 years previously will not be allowed.
Where this trial is running
Riyadh
- King Faisal Specialist Hospital & Research Center — Riyadh, Saudi Arabia (RECRUITING)
Study contacts
- Principal investigator: Riad O El Fakih, MD — KFSH&RC
- Study coordinator: Riad O El Fakih, MD
- Email: relfakih1@kfshrc.edu.sa
- Phone: + 966114647272
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Acute Lymphoblastic Leukemia in Complete Remission, Acute Myeloid Leukemia in Remission, Sibling Donor Transplant, Allogeneic hematopoietic cell transplantation, GvHD Prophylaxis, Myeloablative regimen