Back to resultsNaglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
Primary Purpose
Maroteaux-Lamy Syndrome
Status
Terminated
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Naglazyme®
Sponsored by
About this trial
This is an interventional treatment trial for Maroteaux-Lamy Syndrome focused on measuring Maroteaux-Lamy Syndrome, mucopolysaccharide VI, MPS VI
Eligibility 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
Sites / Locations
- University of Minnesota Medical Center, Fairview
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Naglazyme®
Arm Description
weekly Naglazyme® infusion for 2 years
Outcomes
Primary Outcome Measures
Change in Urinary Glycosaminoglycan (GAG) Excretion
Change in urinary glycosaminoglycan (GAG) excretion from baseline to 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
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
Secondary Outcome Measures
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.
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.
Full Information
NCT ID
NCT02156674
First Posted
June 3, 2014
Last Updated
December 22, 2020
Sponsor
Masonic Cancer Center, University of Minnesota
Collaborators
BioMarin Pharmaceutical
1. Study Identification
Unique Protocol Identification Number
NCT02156674
Brief Title
Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
Official Title
Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
December 2020
Overall Recruitment Status
Terminated
Why Stopped
Slow accrual
Study Start Date
January 26, 2016 (Actual)
Primary Completion Date
December 1, 2019 (Actual)
Study Completion Date
December 1, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Masonic Cancer Center, University of Minnesota
Collaborators
BioMarin Pharmaceutical
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Maroteaux-Lamy Syndrome
Keywords
Maroteaux-Lamy Syndrome, mucopolysaccharide VI, MPS VI
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Naglazyme®
Arm Type
Experimental
Arm Description
weekly Naglazyme® infusion for 2 years
Intervention Type
Drug
Intervention Name(s)
Naglazyme®
Intervention Description
1 mg per kg of body weight administered once weekly as an intravenous infusion
Primary Outcome Measure Information:
Title
Change in Urinary Glycosaminoglycan (GAG) Excretion
Description
Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years
Time Frame
Baseline and 2 years
Title
Change in Distance Traveled
Description
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
Title
Change in Neurocognitive Ability
Description
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 Outcome Measure Information:
Title
Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy
Description
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
Title
Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy
Description
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
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elizabeth Braulin, M.D.
Organizational Affiliation
University of Minnesota
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Minnesota Medical Center, Fairview
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
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