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 ]
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