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

NCT02120157 ↗

Phase 2 Completed 35 enrolled Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

NaSingle-groupOpen-labelTreatment

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%.

Timeline

Start: 2015-07-02
Primary completion: 2018-06-15
Completion: 2020-10-01

Publications

Fierro-Pineda JC, Tsai HL, Blackford A, Cluster A, Caywood E, Dalal J, Davis J, Egeler M, Huo J, Hudspeth M, Keating A, Kelly SS, Krueger J, Lee D, Lehmann L, Madden L, Oshrine B, Pulsipher MA, Fry T, Symons HJ. Prospective PTCTC trial of myeloablative haplo-BMT with posttransplant cyclophosphamide for pediatric acute leukemias. Blood Adv. 2023 Sep 26;7(18):5639-5648. doi: 10.1182/bloodadvances.2023010281.

Drugs

Evaluation	Drug	Modality	Dose	Route
Background	Busulfan	Small molecule	—	Intravenous
Background	Cyclophosphamide	Other / unclassified	50 mg/kg	Intravenous
Background	Mycophenolate Mofetil	Small molecule	15 mg/kg	Oral
Background	Tacrolimus	Small molecule	0.015 mg/kg	Intravenous

Indications

No indication recorded.