Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02156674
• Date of registration: 03/06/2014
• Prospective Registration: Yes
• Primary sponsor: Masonic Cancer Center, University of Minnesota
• Public title: Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
• Scientific title: Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome
• Date of first enrolment: January 26, 2016
• Target sample size: 1
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT02156674
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Elizabeth Braulin, M.D.
• Affiliation: University of Minnesota

Key inclusion & exclusion 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



Age minimum: 3 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Maroteaux-Lamy Syndrome

Intervention(s)

Drug: Naglazyme®

Primary Outcome(s)

Change in urinary glycosaminoglycan (GAG) excretion [Time Frame: 2 years]

Change in distance traveled [Time Frame: 2 years]

Change in neurocognitive ability [Time Frame: 2 years]

Secondary Outcome(s)

development of antibodies to Naglazyme therapy [Time Frame: 2 years]

Secondary ID(s)

MT2014-08R

2014LS014

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

BioMarin Pharmaceutical