Clinical Trial: Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome

Brief Summary: This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.

Detailed Summary:
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome:

  • Change in urinary glycosaminoglycan (GAG) excretion [ Time Frame: 2 years ]
    Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years
  • Change in distance traveled [ Time Frame: 2 years ]
    Change in distance traveled in 6 minute walk and standard tests of range of motion and mobility
  • Change in neurocognitive ability [ Time Frame: 2 years ]
    Subjects will undergo full neuropsychological testing annually and abbreviated testing for attention and adaptive skills at the six-month interval visits


Original Primary Outcome: Same as current

Current Secondary Outcome: development of antibodies to Naglazyme therapy [ Time Frame: 2 years ]

development of antibodies including neutralizing antibodies with weekly Naglazyme therapy and the impact of these antibodies on cardiorespiratory and skeletal parameters as measured by the 6--minute walk test and standard tests of range of motion and mobility, and the change in neurocognitive ability


Original Secondary Outcome: Same as current

Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: June 3, 2014
Date Started: January 2016
Date Completion: March 2020
Last Updated: August 15, 2016
Last Verified: August 2016