Inherited Metabolic Disorders IMD Clinical Trial
Official title:
A Single-arm, Open-label, Study to Evaluate the Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Undergoing Stem Cell Transplantation After Reduced Intensity Conditioning
This study is designed to assess the safety and exploratory efficacy of using HSC835 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
This phase II study is designed to assess the safety of the Novartis product HSC835 and its ability to achieve donor blood stem cell engraftment in patients with certain Inherited Metabolic Disorders who undergo stem cell transplantation. A reduced intensity conditioning will be used prior to transplantation. Patients with Hurler syndrome, MLD, Krabbe or cALD could be eligible for this study. ;