Metachromatic Leukodystrophy Clinical Trial
Official title:
Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
This is a single patient expanded access protocol to investigate the effects of a second dose of facilitating cell-enhanced hematopoietic stem cell product.
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.
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