Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

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

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.

Study Overview

• Status: Terminated
• Conditions: Maroteaux-Lamy Syndrome
• Intervention / Treatment: Drug: Naglazyme®

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota Medical Center, Fairview

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Masonic Cancer Center, University of Minnesota
• Collaborators: BioMarin Pharmaceutical
• Investigators: Principal Investigator: Elizabeth Braulin, M.D., University of Minnesota

Study record dates

Study Major Dates

• Study Start (Actual): January 26, 2016
• Primary Completion (Actual): December 1, 2019
• Study Completion (Actual): December 1, 2019

Study Registration Dates

• First Submitted: June 3, 2014
• First Submitted That Met QC Criteria: June 3, 2014
• First Posted (Estimate): June 5, 2014

Study Record Updates

• Last Update Posted (Actual): December 23, 2020
• Last Update Submitted That Met QC Criteria: December 22, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Keywords: MPS VI; Maroteaux-Lamy Syndrome; mucopolysaccharide VI
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Disease; Genetic Diseases, Inborn; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mucopolysaccharidoses; Syndrome; Mucopolysaccharidosis VI
• Other Study ID Numbers: 2014LS014; MT2014-08R (Other Identifier: University of Minnesota Blood and Marrow Transplant Program)