Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

Phase 1/2 Recruiting 29 enrolled Washington University School of Medicine

NaSingle-groupOpen-labelTreatment

Summary

This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.

Timeline

Start: 2017-03-20
Primary completion: 2028-04
Completion: 2033-04

Publications

Ngwube A, Shah N, Godder K, Jacobsohn D, Hulbert ML, Shenoy S. Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell disease. Blood Adv. 2020 Aug 25;4(16):3894-3899. doi: 10.1182/bloodadvances.2020002236.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Abatacept	Other / unclassified	5 mg/kg	Intravenous
Subject	Abatacept	Other / unclassified	10 mg/kg	Intravenous
Background	Alemtuzumab	Monoclonal antibody	10 mg	Intravenous
Background	Alemtuzumab	Monoclonal antibody	20 mg	Intravenous
Background	Cyclophosphamide	Other / unclassified	50 mg/kg	Intravenous
Background	Fludarabine	Small molecule	30 mg/m2	Intravenous
Background	Hydroxyurea	Other / unclassified	30 mg/kg	Oral
Background	Melphalan	Small molecule	140 mg/m2	—
Background	Mycophenolate Mofetil	Small molecule	—	—
Background	Rituximab	Monoclonal antibody	375 mg/m2	Intravenous
Background	Tacrolimus	Small molecule	—	—
Background	Thiotepa	Other / unclassified	8 mg/kg	Intravenous

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

Summary

Timeline

Publications

Drugs

Indications