NCT02120157

Myeloablative Haploidentical BMT With Post-transplant Cyclophosphamide for Pediatric Patients With Hematologic Malignancies

Pediatric Blood & Marrow Transplant Consortium (PBMTC) Phase II Myeloablative Haploidentical BMT With Post-transplantation Cyclophosphamide for Pediatric Patients With Hematologic Malignancies

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Academic / Other
• Sex: All
• Age: 6 Months – 25 Years
• Healthy volunteers: Not accepted

Summary

This is a multi-institutional phase II haploidentical T cell replete bone marrow transplant (BMT) study in children with high-risk leukemia. The myeloablative conditioning regimen prescribed will be Total body irradiation (TBI)-based for lymphoid leukemia and busulfan-based for myeloid leukemia. Our goal is to establish an easily exportable, inexpensive platform for haplotransplantation that has a safety profile equivalent to matched related and unrelated BMTs. The primary objective will be to estimate the incidence of 6-month non-relapse mortality (NRM), hypothesizing that NRM is \< 18%.

Detailed description

This is a phase II prospective study designed to evaluate the incidence of 6 month non- relapse mortality, safety, and feasibility of haploidentical bone marrow transplantation (BMT) after myeloablative conditioning with post-transplant Cy. Conditioning regimens include a total body irradiation (TBI)-based prep for lymphoid leukemias and a chemotherapy based prep for myeloid leukemias. To estimate the incidence of non-relapse mortality at 180 days following myeloablative haploidentical BMT for children and young adults with high risk hematologic malignancies.

Conditions

• Myeloablative Conditioning
• HLA-mismatched Bone Marrow Transplantation
• Graft Survival
• Transplantation, Bone Marrow

Interventions

Type	Name	Description
DRUG	Cyclophosphamide	Chemotherapy administration
RADIATION	TBI	Radiation Therapy
DRUG	Busulfan	Chemotherapy Administered
OTHER	Unmanipulated Bone Marrow	Bone Marrow Transplant
DRUG	Tacrolimus	Immunosuppressive Drug Administered
DRUG	Mycophenolate mofetil	Immunosuppressive Drug Administered

Timeline

• Start date: 2015-07-02
• Primary completion: 2018-06-15
• Completion: 2020-10-01
• First posted: 2014-04-22
• Last updated: 2021-11-26
• Results posted: 2019-04-18

Locations

9 sites across 2 countries: United States, Canada