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Most of the real world evidence data related to efficacy of cysteamine therapy is retrospective. This study is a ambispective study to investigate the impact of cystine depletion therapy on the quality of life of patients and their parents.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Patients on extended release cysteamine treatment at study enrollment |
| |
| Cohort 2 | Patients switching from immediate release cysteamine to extended release cysteamine during the study |
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| Cohort 3 | Patients remaining on immediate release cysteamine treatment |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cysteamine Bitartrate | Drug | ERT |
|
| Measure | Description | Time Frame |
|---|---|---|
| to descriptively assess the change from baseline and percent change from baseline in the quality of life overall summary sores at 12 months in switch cohort | 12 months |
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Inclusion criteria:
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All Belgian nephropathic cystinosis patients.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UZA | Antwerp | Belgium | ||||
| HUDERF |
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| ID | Term |
|---|---|
| D003554 | Cystinosis |
| ID | Term |
|---|---|
| D016464 | Lysosomal Storage Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D003543 | Cysteamine |
| ID | Term |
|---|---|
| D008624 | Mercaptoethylamines |
| D005021 | Ethylamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
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| Brussels |
| Belgium |
| UCL Saint-Luc | Brussels | Belgium |
| UZ Gent | Ghent | Belgium |
| UZ Leuven | Leuven | Belgium |
| CHC | Liège | Belgium |
| CHU | Liège | Belgium |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D013438 |
| Sulfhydryl Compounds |
| D013457 | Sulfur Compounds |