Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Maroteaux-Lamy Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02156674
• Other study ID #: 2014LS014
• Secondary ID: MT2014-08R
• Status: Terminated
• Phase: N/A
• Start date: January 26, 2016
• Est. completion date: December 1, 2019

Study information

• Verified date: December 2020
• Source: Masonic Cancer Center, University of Minnesota
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 1
• Est. completion date: December 1, 2019
• Est. primary completion date: December 1, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Maroteaux-Lamy Syndrome
• Mucopolysaccharidosis VI
• Syndrome

Intervention

Drug:
• Naglazyme®
1 mg per kg of body weight administered once weekly as an intravenous infusion

Locations

Country	Name	City	State
United States	University of Minnesota Medical Center, Fairview	Minneapolis	Minnesota

Sponsors (2)

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

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Urinary Glycosaminoglycan (GAG) Excretion	Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years	Baseline and 2 years
Primary	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
Primary	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	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
Secondary	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