Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Masonic Cancer Center, University of Minnesota1 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Maroteaux-Lamy Syndrome

Interventions

Naglazyme®DRUG

1 mg per kg of body weight administered once weekly as an intravenous infusion

Eligibility

Sex: ALLMin age: 3 Years

Medical Language ↔ Plain English

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

Locations (1)

University of Minnesota Medical Center, Fairview

Minneapolis, Minnesota, United States

Outcomes

Primary Outcomes

Change in Urinary Glycosaminoglycan (GAG) Excretion

Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years

Time frame: 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

Time frame: 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

Time frame: Baseline and 2 years

Secondary Outcomes

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.

Time frame: 6 months

Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy

Time frame: 2 years

Data from ClinicalTrials.gov

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