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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Hematopoietic Stem cell transplantation will be done with the cell therapy product HSC835
Incidence of infusional toxicities
Time frame: 48 hours
Incidence of neutrophil recovery
Time frame: 42 days
Incidence of graft failure
Time frame: 42 days
Time to neutrophil recovery
Time frame: 42 days
Time to platelet recovery
Time frame: 180 days
Number of patients with grade II-IV acute graft versus host disease (aGVHD)
Time frame: 100 days
Number of patients with chronic graft versus host disease (cGVHD)
Time frame: 1 and 2 years
Incidence of death
Time frame: 100 days, 1 year and 2 years
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