NCT06001385

HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis

A Phase II Study of Reduced Dose Post Transplantation Cyclophosphamide as GvHD Prophylaxis in Adult Patients With Hematologic Malignancies Receiving HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 313 (estimated)
• Sponsor: Center for International Blood and Marrow Transplant Research · Network
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to determine the effectiveness of Reduced Dose Post-Transplant Cyclophosphamide (PTCy) in patients with hematologic malignancies after receiving an HLA-Mismatched Unrelated Donor (MMUD) . The main question\[s\] it aims to answer are: * Does a reduced dose of PTCy reduce the occurrence of infections in the first 100 days after transplant? * Does a reduced dose of PTCy maintain the same level of protection against Graft Versus Host Disease (GvHD) as the standard dose of PTCy?

Conditions

• Acute Lymphoblastic Leukemia
• Acute Myeloid Leukemia
• Acute Leukemia
• Myelodysplastic Syndromes
• Chronic Myeloid Leukemia
• Chronic Lymphocytic Leukemia
• Myeloproliferative Neoplasm
• Lymphoma
• Chronic Myelomonocytic Leukemia
• Pro-Lymphocytic Leukemia
• Myelofibrosis

Interventions

Type	Name	Description
DRUG	Busulfan	Given IV or PO pre-transplant as part of conditioning regimen
DRUG	Fludarabine	Given IV pre-transplant as part of conditioning regimen
PROCEDURE	PBSC Hematopoietic Stem Cell Transplantation (HSCT)	Peripheral blood stem cell graft is infused from a mismatched unrelated donor on Day 0
DRUG	Post-Transplant Cyclophosphamide	Cyclophosphamide (25mg/kg) is administered on Day 3 and Day 4 post-transplant as an IV infusion over 1-2 hours. First 20 subjects with a 4-6/8 HLA mismatched unrelated donor will receive an intermediate dose of post-transplant cyclophosphamide of 37.5 mg/kg Day 3 and Day 4 post-transplant.
DRUG	Mesna	Mesna is given in divided doses IV 30 min pre- and at 3, 6, and 8 hours post cyclophosphamide.
DRUG	Tacrolimus	Tacrolimus is given at a dose of 0.05 mg/kg PO or an IV dose of 0.03 mg/kg of ideal body weight (IBW) starting on Day +5 post-transplant with taper recommended at Day + 90 and finished by Day +180.
DRUG	Mycophenolate Mofetil	Mycophenolate mofetil (MMF) is given at a dose of 15 mg/kg three times daily IV or PO from Day +5 to Day +35 post-transplant.
OTHER	Patient Reported Outcomes	Survey assessments will be administered to study participants pre transplant, at Day + 100, Day + 180, and Day +365 post transplant.
DRUG	Melphalan	Given IV pre transplant as part of conditioning regimen
RADIATION	Total-body irradiation	Administered pre-transplant as part of conditioning regimen
DRUG	Cyclophosphamide	Given IV pre-transplant as part of conditioning regimen

Timeline

• Start date: 2023-12-08
• Primary completion: 2026-02-01
• Completion: 2026-06-30
• First posted: 2023-08-21
• Last updated: 2026-02-05

Locations

32 sites across 1 country: United States