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| ID | Type | Description | Link |
|---|---|---|---|
| MT2014-08R | Other Identifier | University of Minnesota Blood and Marrow Transplant Program |
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Slow accrual
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| Name | Class |
|---|---|
| BioMarin Pharmaceutical | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Naglazyme® | Experimental | weekly Naglazyme® infusion for 2 years |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Naglazyme® | Drug | 1 mg per kg of body weight administered once weekly as an intravenous infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Urinary Glycosaminoglycan (GAG) Excretion | Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years | 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 | 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 | Baseline and 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Elizabeth Braulin, M.D. | University of Minnesota | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Minnesota Medical Center, Fairview | Minneapolis | Minnesota | 55455 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Naglazyme® | weekly Naglazyme® infusion for 2 years Naglazyme®: 1 mg per kg of body weight administered once weekly as an intravenous infusion |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Naglazyme® | weekly Naglazyme® infusion for 2 years Naglazyme®: 1 mg per kg of body weight administered once weekly as an intravenous infusion |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Urinary Glycosaminoglycan (GAG) Excretion | Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years | Posted | Number | mg/mm Cr | Baseline and 2 years |
|
|
2 years
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Naglazyme® | weekly Naglazyme® infusion for 2 years Naglazyme®: 1 mg per kg of body weight administered once weekly as an intravenous infusion |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Elizabeth Braunlin MD, PhD | Masonic Cancer Center, University of Minnesota | 612 626 2755 | braun002@umn.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Dec 4, 2015 | Oct 28, 2020 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D009087 | Mucopolysaccharidosis VI |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| ID | Term |
|---|---|
| C508864 | galsulfase |
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| 6 months |
| 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 |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
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| 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 | Posted | Number | Foot | 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 | Not Evaluated | Posted | 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. | Posted | Number | participants | 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. | No data available | Posted | 2 years |
|
|
| 0 |
| 1 |
| 0 |
| 1 |
| 0 |
| 1 |
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| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |