Clinical Trial: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation

Brief Summary: The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell transplantation (HCT).

Detailed Summary: This has been an ongoing area of interest by our group at the Univ. of Minnesota, but this is a new protocol to take the place of several older protocols. While survival has been very good on the prior protocols over the past decade, incomplete engraftment has remained somewhat problematic. Therefore, we have modified the preparative regimen somewhat to increase engraftment by replacing anti-thymocyte globulin (ATG) with Campath-1H, a drug that is more immune suppressive. In addition, we have modified the supportive care regimen. Based on this, we will monitor levels of an anti-oxidant therapy (N-acetylcysteine) and biomarkers of inflammation and oxidative stress for the families that consent to these research studies.
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Number of Patients Achieving Engraftment [ Time Frame: Day 100 ]

Rate of successful engraftment - patients who achieved and sustained donor engraftment; donor chimerism by day 100 of at least 90% after undergoing hematopoietic stem cell transplantation.


Original Primary Outcome: Survival [ Time Frame: at 100 days, 1 year and 3 years ]

Current Secondary Outcome: Overall Survival [ Time Frame: Day 100, 1 Year, 3 Years ]

Number of patients alive at timepoints.


Original Secondary Outcome:

  • Incidence and severity of graft-versus-host disease (GVHD) [ Time Frame: at day 100 ]
  • Incidence of viral and fungal infections [ Time Frame: at day 180 ]
  • Incidence of pulmonary complications [ Time Frame: at day 180 ]
  • Changes in neuropsychometric function [ Time Frame: at 1 year, 2 years and 3 years in comparison to baseline studies ]
  • Changes in the central nervous system in regards to progression using imaging studies (MRI) [ Time Frame: Through 3 years post BMT ]
  • time to CD4 recovery [ Time Frame: Through 1 year ]


Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: April 25, 2008
Date Started: March 2008
Date Completion:
Last Updated: November 6, 2012
Last Verified: November 2012