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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-000321-31 | EudraCT Number |
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The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial.
This protocol only concern subjects where compassionate use program was not accepted. Efficacy will be evaluated once yearly to follow the subject's progress in clinical parameters.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Lamazym | Experimental | 1 mg Lamazym/kg body weight |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Lamazym | Drug | ERT, i.v. infusions weekly |
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| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in Adverse events | AE documented at all visits | 3 year |
| Change from baseline in Lamazym antibodies | AB measured every 12th week | 3 year |
| Measure | Description | Time Frame |
|---|---|---|
| progress from baseline in number of steps climbed in 3 minutes | 1 year, 2 year and 3 year | |
| Progress from baseline in equivalent age | 1 year, 2 year and 3 year | |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9 | Copenhagen | DK-2100 | Denmark |
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| Label | URL |
|---|---|
| 12th international symposium on MPS and related diseases | View source |
| Results posted in EudraCT | View source |
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| ID | Term |
|---|---|
| D008363 | alpha-Mannosidosis |
| ID | Term |
|---|---|
| D044904 | Mannosidase Deficiency Diseases |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| Progress from baseline in Forced Vital Capacity |
| 1 year, 2 year and 3 year |
| Progress from baseline in distance walked in 6 minutes | 1 year, 2 year and 3 year |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |