Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

University of Minnesota (UMN)

Status: Terminated

Conditions: Maroteaux-Lamy Syndrome

Treatments: Drug: Naglazyme®

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02156674

2014LS014

MT2014-08R (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 1 patient

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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1 participants in 1 patient group

Naglazyme®

Experimental group

Description:

weekly Naglazyme® infusion for 2 years

Treatment:

Drug: Naglazyme®