HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

Phase 1/2 Completed 29 enrolled Northwell Health

NaSingle-groupOpen-labelTreatment

Summary

This study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a routine stem cell transplant in order to assess whether or not it is as effective as myeloablative high dose chemotherapy and transplant.

Timeline

Start: 2008-12
Primary completion: 2019-03
Completion: 2019-12

Publications

King AA, Kamani N, Bunin N, Sahdev I, Brochstein J, Hayashi RJ, Grimley M, Abraham A, Dioguardi J, Chan KW, Douglas D, Adams R, Andreansky M, Anderson E, Gilman A, Chaudhury S, Yu L, Dalal J, Hale G, Cuvelier G, Jain A, Krajewski J, Gillio A, Kasow KA, Delgado D, Hanson E, Murray L, Shenoy S. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015 Dec;90(12):1093-8. doi: 10.1002/ajh.24183. Epub 2015 Oct 6.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Alemtuzumab	Monoclonal antibody	10 mg	Intravenous
Subject	Alemtuzumab	Monoclonal antibody	15 mg	Intravenous
Subject	Fludarabine	Small molecule	35 mg/m2	—
Subject	Melphalan	Small molecule	70 mg/m2	—
Background	Cyclosporine	Peptide	—	—
Background	Mycophenolate Mofetil	Small molecule	—	—
Background	Tacrolimus	Small molecule	—	—

HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

Summary

Timeline

Publications

Drugs

Indications