Clinical Trial: Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Brief Summary: The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Mucopolysaccharidosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.

Detailed Summary:
Sponsor: Tehran University of Medical Sciences

Current Primary Outcome: Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ]

Original Primary Outcome: Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic HSCT [ Time Frame: 1 year ]

Current Secondary Outcome:

  • One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ]
  • One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ]
  • Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ]
  • Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ]


Original Secondary Outcome:

  • One year overall survival after allogeneic HSCT [ Time Frame: 1 year ]
  • One year Progressive Free Survival after allogeneic HSCT [ Time Frame: 1 year ]
  • Transplantation Related Mortality (TRM) after allogeneic HSCT [ Time Frame: 1 year ]
  • Acute and chronic GVHD rate after allogeneic HSCT [ Time Frame: 1 year ]


Information By: Tehran University of Medical Sciences

Dates:
Date Received: November 8, 2010
Date Started: September 2009
Date Completion: December 2012
Last Updated: May 31, 2012
Last Verified: May 2012