Clinical Trial: Single Patient Expanded Access Protocol: Metabolic Boost

Study Status: No longer available
Recruit Status: No longer available
Study Type: Expanded Access

Official Title: Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Brief Summary: This is a single patient expanded access protocol to investigate the effects of a second dose of facilitating cell-enhanced hematopoietic stem cell product.

Detailed Summary:

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.

Sponsor: University of Louisville

Current Primary Outcome:

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Information By: University of Louisville

Dates:
Date Received: December 19, 2013
Date Started:
Date Completion:
Last Updated: March 17, 2017
Last Verified: March 2017