Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Not Applicable

Terminated

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.

Recruitment & Eligibility

Inclusion Criteria

Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) treated with a prior allogeneic transplant >2 years previously
Persons currently receiving Naglazyme may be accepted into the study
Age > 2 years
>10% engrafted based on most recent testing
Willing to commit to traveling to the University of Minnesota every 6 months
Written informed consent with parent/guardian consent for children < 18 years of age or persons unable to consent with minor assent if appropriate

Exclusion Criteria

History of cardiac or pulmonary insufficiency or those requiring continuous supplemental oxygen
Pregnant or breastfeeding
Any condition that, in the view of the investigator, places the patient at high risk of poor treatment compliance or of not completing the study

Arm && Interventions

Group	Intervention	Description
Naglazyme®	Naglazyme®	weekly Naglazyme® infusion for 2 years

Primary Outcome Measures

Name	Time	Method
Change in Urinary Glycosaminoglycan (GAG) Excretion	Baseline and 2 years	Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years
Change in Distance Traveled	Baseline and 2 years	Change in distance traveled in 6 minute walk and standard tests of range of motion and mobility. Improvement in distance walked from the start to study to end of the study is reported
Change in Neurocognitive Ability	Baseline and 2 years	Subjects will undergo full neuropsychological testing annually and abbreviated testing for attention and adaptive skills at the six-month interval visits

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy	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. Anti-rhASB antibodies in patient's sera were tested in the Biomarin Laboratory using an in-house test of ELISA.