drugset
drugset / Trial / NCT03513328

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328 ↗

Phase 1/2 Completed 6 enrolled University of Florida Live Like Bella Pediatric Cancer Research · collab
RandomizedSequentialOpen-labelTreatment

Summary

In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.

Timeline

Start
2018-06-15
Primary completion
2022-02-17
Completion
2023-02-19

Drugs

EvaluationDrugModalityDoseRoute
Subject Thiotepa Other / unclassified 5 mg/kg Intravenous
Subject Thiotepa Other / unclassified 10 mg/kg Intravenous

Indications

Diamond-Blackfan anemia Hurler syndrome Wiskott-Aldrich syndrome X-linked lymphoproliferative syndrome acquired thrombocytopenia adrenoleukodystrophy bone marrow failure syndrome chronic granulomatous disease common variable immunodeficiency hemolytic anemia hemophagocytic syndrome neutropenia oligosaccharidosis severe combined immunodeficiency sickle cell disease thalassemia