NCT03246906

Comparison of Triple GVHD Prophylaxis Regimens for Nonmyeloablative or Reduced Intensity Conditioning Unrelated Mobilized Blood Cell Transplantation

A Randomized Phase II Study to Compare the Net Clinical Benefit of Cyclosporine and Sirolimus Combined With MMF or Post-Transplant Cyclophosphamide as GVHD Prophylaxis After HLA-Matched or HLA-Mismatched Unrelated G-CSF Mobilized Blood Cell Transplantation Using Nonmyeloablative or Reduced Intensity Conditioning for Patients With Hematologic Malignancies

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 150 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This randomized phase II trial includes a blood stem cell transplant from an unrelated donor to treat blood cancer. The treatment also includes chemotherapy drugs, but in lower doses than conventional (standard) stem cell transplants. The researchers will compare two different drug combinations used to reduce the risk of a common but serious complication called "graft versus host disease" (GVHD) following the transplant. Two drugs, cyclosporine (CSP) and sirolimus (SIR), will be combined with either mycophenolate mofetil (MMF) or post-transplant cyclophosphamide (PTCy). This part of the transplant procedure is the main research focus of the study.

Detailed description

OUTLINE: Patients are randomized to 1 of 2 arms. ARM I: Patients undergo allogeneic hematopoietic stem cell transplant (HCT) at day 0. Patients with an HLA-matched unrelated donor receive mycophenolate mofetil orally (PO) on days 0 to 40, cyclosporine PO every 12 hours twice daily (BID) on days -3 to 96 then tapered to day 150, and sirolimus PO once daily (QD) on days -3 to day 150 then tapered to day 180. Patients with an HLA-mismatched donor receive mycophenolate mofetil PO on days 0-100 then tapered to day 150, cyclosporine PO BID on days -3 to 150 then tapered to day 180, and sirolimus PO QD on days -3 to 180 then tapered to day 365. ARM II: Patients undergo HCT at day 0. Patients with an HLA-matched unrelated donor receive cyclosporine PO BID on days 5-96 then tapered to day 150, sirolimus PO QD on days 5-150 then tapered to day 180, and cyclophosphamide intravenously (IV) on days 3 and 4. Patients with an HLA-mismatched donor receive cyclosporine PO BID on days 5-150 then tapered to day 180, sirolimus PO QD on days 5-180 then tapered to day 365, and cyclophosphamide IV on days 3 and 4. After completion of study treatment, patients are followed up at 6 months and every year thereafter.

Conditions

• Acute Lymphoblastic Leukemia
• Acute Myeloid Leukemia
• Aggressive Non-Hodgkin Lymphoma
• Chronic Lymphocytic Leukemia
• Diffuse Large B-Cell Lymphoma
• Mantle Cell Lymphoma
• Myelodysplastic Syndrome
• Myelodysplastic/Myeloproliferative Neoplasm
• Prolymphocytic Leukemia
• Recurrent Chronic Lymphocytic Leukemia
• Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive
• Recurrent Hodgkin Lymphoma
• Recurrent Plasma Cell Myeloma
• Recurrent Small Lymphocytic Lymphoma
• Recurrent Waldenstrom Macroglobulinemia
• Hematologic and Lymphocytic Disorder
• Blastic Plasmacytoid Dendritic Cell Neoplasm

Interventions

Type	Name	Description
PROCEDURE	Allogeneic Hematopoietic Stem Cell Transplantation	Undergo HCT
DRUG	Cyclophosphamide	Given IV
DRUG	Cyclosporine	Given PO
DRUG	Mycophenolate Mofetil	Given PO
DRUG	Sirolimus	Given PO

Timeline

• Start date: 2017-09-11
• Primary completion: 2025-05-03
• Completion: 2025-05-03
• First posted: 2017-08-11
• Last updated: 2025-06-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study