Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Study Overview
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Brief Summary
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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.

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

Conditions
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Maroteaux-Lamy Syndrome

Intervention / Treatment
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* Drug: Naglazyme®


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 3 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Naglazyme®<br>weekly Naglazyme® infusion for 2 years | Drug: Naglazyme®<br>* 1 mg per kg of body weight administered once weekly as an intravenous infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Urinary Glycosaminoglycan (GAG) Excretion | Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years | Baseline and 2 years | 
| Change in Distance Traveled | 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 | Baseline and 2 years | 
| Change in Neurocognitive Ability | Subjects will undergo full neuropsychological testing annually and abbreviated testing for attention and adaptive skills at the six-month interval visits | Baseline and 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy | 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. | 6 months | 
| Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy | 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. | 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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mucopolysaccharide VI, Maroteaux-Lamy Syndrome, MPS VI