Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

• Status: Terminated
• Trial end date: 2019-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota
• Collaborator: BioMarin Pharmaceutical

Criteria

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