Clinical Trial: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Brief Summary: The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.

Detailed Summary: The objective for the study is to establish chimerism following reduced intensity conditioning with no grade III/IV GVHD. The primary endpoint we will follow is production of the missing enzyme at ≥ 10% of the normal level at day 180 post-transplant in > 90% of patients.
Sponsor: University of Louisville

Current Primary Outcome: Production of missing enzyme at levels greater than or equal to 10% of normal [ Time Frame: Day 180 post transplant to three years ]

Original Primary Outcome: Production of missing enzyme at levels greater than or equal to 10% of normal [ Time Frame: At day 180 post transplant in 90% of patients ]

Current Secondary Outcome: Enriched Hematopoetic Stem Cell Engraftment [ Time Frame: One month to three years ]

Original Secondary Outcome:

Information By: University of Louisville

Dates:
Date Received: June 10, 2011
Date Started: April 2011
Date Completion: April 2028
Last Updated: November 30, 2016
Last Verified: November 2016