Single Patient Expanded Access Protocol: Metabolic Boost (Boost)
|Metachromatic Leukodystrophy||Biological: Enriched Hematopoetic Stem Cell Infusion|
|Study Type:||Expanded Access What is Expanded Access?|
|Official Title:||Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders|
Biological: Enriched Hematopoetic Stem Cell Infusion
Hematopoietic stem cell transplantation (HSCT) has been established as an important therapeutic option for patients with a variety of inherited metabolic disorders (IMD). The potential life-threatening complications of conventional myeloablative HSCT have limited its application. Additionally, conventional HSCT is only available to the small minority of medically suitable candidates who have histocompatibility leukocyte antigen (HLA)-identical siblings to donate bone marrow or mobilized peripheral blood stem cells.
Donor mobilized peripheral blood stem cells or bone marrow will be processed via a novel technology to deplete mature immune cells while retaining hematopoietic stem cells (HSC) and graft facilitating cells (FC).
A now-standard reduced intensity, nonmyeloablative recipient conditioning regimen will be used to promote mixed allogeneic chimerism, thereby significantly reducing morbidity and mortality.
These two enhancements are intended to significantly improve the benefit:risk ratio of HSCT for patients with IMDs. If successful, transplantation will become a more feasible option for patients without HLA-identical siblings to donate stem cells, and could be offered to patients early in disease progression.
The objective for the study is to establish chimerism following reduced intensity conditioning with no grade III/IV graft-versus-host disease (GVHD). The primary endpoint we will follow is production of the missing enzyme at ≥ 10% of the normal level at day 180 post-transplant as for investigational new drug (IND) 14070. This expanded access protocol details the approach to providing a second dose of the product.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02021266
|United States, North Carolina|
|Durham, North Carolina, United States, 27705|
|Study Director:||Suzanne T Ildstad, MD||University of Louisville|