NCT03431090

Haploidentical Hematopoietic Cell Transplantation for Children With Hematologic Malignancies and Myelodysplasia

HAPLEUK17, Haploidentical Hematopoietic Cell Transplantation for Children With Hematologic Malignancies and Myelodysplasia

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Nationwide Children's Hospital · Academic / Other
• Sex: All
• Age: 30 Years
• Healthy volunteers: Accepted

Summary

This is a Phase I/II study designed to evaluate the kinetics of hematopoietic reconstitution and the incidence of acute chronic GVHD after partially matched related donor hematopoietic cell transplantation using an αβTCR/CD19+ cell depleted graft.

Detailed description

Less than 30% of patients undergoing hematopoietic cell transplantation (HCT) will have an HLA-matched sibling donor. There is a high likelihood of being unable to identify a perfect HLA matched unrelated donor, and the time to procure the marrow if such a donor is available is generally \>3 months. An emerging body of literature suggests that related haploidentical HCT with innovative graft engineering may provide equal, or possibly superior, outcomes to conventional unrelated donors. This protocol is designed to test the hypothesis that HCT using an αβT cell / CD19+ B cell depleted graft from partially matched related donors will result in rapid, durable hematopoietic engraftment and rapid immune reconstitution with an acceptably low risk of severe acute and chronic GVHD.

Conditions

• Myelodysplasia
• Hematologic Malignancy

Interventions

Type	Name	Description
DEVICE	CliniMACS® TCRαβ/CD19 Combined Depletion System	The mobilized peripheral blood cell collection (apheresis product) will be processed using a Miltenyi CliniMACS device according to the manufacturing instructions. The processing will deplete the αβTCR+ cells and CD19+ cells from the apheresis product to formulate the graft.

Timeline

• Start date: 2018-03-02
• Primary completion: 2026-12-01
• Completion: 2026-12-01
• First posted: 2018-02-13
• Last updated: 2026-04-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated device study