Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

Phase 2 Active not recruiting 75 enrolled Children's Hospital of Philadelphia

Non-randomizedSingle-groupOpen-labelTreatment

Summary

This is a Phase II pilot study to evaluate engraftment and toxicity of patients with non-malignant diseases using a reduced intensity conditioning regimen in the setting of allogeneic transplant for non malignant diseases. Bone Marrow or cord blood will be acceptable as a stem cell source. Recently, reduced intensity conditioning (RIC) regimens have been used for both adult patients with leukemias and pediatric patients with non-malignant diseases. These regimens are better tolerated, resulting in less transplant related morbidity and mortality. Stable mixed chimerism, while insufficient for eradication of leukemias, may be sufficient to cure patients with non-malignant diseases.

Timeline

Start: 2008-01
Primary completion: 2024-07
Completion: 2026-12

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	—	Intravenous
Subject	Busulfan	Small molecule	—	Intravenous
Subject	Fludarabine	Small molecule	—	Intravenous
Subject	Melphalan	Small molecule	140 mg/m2	Intravenous
Background	Cyclosporine	Peptide	—	—
Background	Mycophenolate Mofetil	Small molecule	—	—

Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

Summary

Timeline

Publications

Drugs

Indications