NCT01203722

Reduced Intensity, Partially HLA Mismatched BMT to Treat Hematologic Malignancies

Reduced Intensity, Partially HLA Mismatched Allogeneic BMT for Hematologic Malignancies Using Donors Other Than First-degree Relatives

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 87 (actual)
• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Academic / Other
• Sex: All
• Age: 6 Months – 75 Years
• Healthy volunteers: Accepted

Summary

If transplantation using mismatched unrelated donors or non-first-degree relatives could be performed with an acceptable toxicity profile, an important unmet need would be served. Towards this goal, the current study extends our platform of nonmyeloablative, partially HLA-mismatched bone marrow transplant (BMT) and Peripheral Blood Stem Cell Transplant (PBSCT) to the use of such donors, investigating up to several postgrafting immunosuppression regimens that incorporate high-dose Cy. Of central interest is the incorporation of sirolimus into this postgrafting immunosuppression regimen. The primary goal for phase 1 is to identify a transplant regimen associated with acceptable rates of severe acute GVHD and NRM by Day 100 and for phase 2 estimate the 6-month probability of survival without having had acute grade III- IV GVHD or graft failure.

Conditions

• Hematologic Malignancies

Interventions

Type	Name	Description
DRUG	Fludarabine	Fludarabine 30 mg/m2/day
DRUG	Cytoxan	Pre-BMT: Cytoxan 14.5 mg/kg/day administered IV; Post-Transplantation: High-dose Cytoxan 50mg/kg/day
RADIATION	Total Body Irradiation	400 cGy TBI administered in a single fraction
PROCEDURE	Allogeneic Blood or Marrow Transplant
PROCEDURE	Peripheral Blood Stem Cell Transplant
DRUG	Mycophenolate Mofetil	15mg/kg by mouth three times daily
DRUG	Sirolimus	Loading Dose: Sirolimus 6mg by mouth once; Maintenance dose: Sirolimus 2mg by mouth daily
DRUG	Tacrolimus	Tacrolimus 1mg intravenously, daily

Timeline

• Start date: 2010-09-01
• Primary completion: 2024-05-28
• Completion: 2024-05-28
• First posted: 2010-09-16
• Last updated: 2026-02-19
• Results posted: 2026-02-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study