drugset
drugset / Trial / NCT03579875

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

NCT03579875 ↗

Phase 2 Recruiting 48 enrolled Masonic Cancer Center, University of Minnesota
Non-randomizedParallel-groupOpen-labelTreatment

Summary

This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.

Timeline

Start
2018-11-13
Primary completion
2027-01-01
Completion
2029-01-05

Drugs

EvaluationDrugModalityDoseRoute
Subject Alemtuzumab Monoclonal antibody 0.2 mg/kg Intravenous
Subject Busulfan Small molecule 0.6 mg/kg Intravenous
Subject Busulfan Small molecule 0.8 mg/kg Intravenous
Subject Cyclophosphamide Other / unclassified 5 mg/kg Intravenous
Subject Cyclophosphamide Other / unclassified 10 mg/kg Intravenous
Subject Fludarabine Small molecule 35 mg/m2 Intravenous
Subject Melphalan Small molecule 2.35 mg/kg Intravenous
Subject Melphalan Small molecule 70 mg/m2 Intravenous
Subject Methylprednisolone Other / unclassified 1 mg/kg Intravenous
Subject Rituximab Monoclonal antibody 200 mg/m2 Intravenous
Background G-CSF Unknown 5 ug/kg Intravenous

Indications

Fanconi anemia TCR-alpha-beta-positive T-cell deficiency bone marrow failure syndrome dyskeratosis congenita myelodysplastic syndrome telomere syndrome