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| Name | Class |
|---|---|
| Quintiles, Inc. | INDUSTRY |
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A retrospective study to collect the effectiveness and safety data of the past treatment with KUVAN in Chinese patients with HPA caused by BH4 deficiency. The data was collected from relevant past medical history and past clinical and safety assessments.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| KUVAN | Drug | retrospective data on the effectiveness of past treatment with KUVAN®. It includes blood Phe level, physical development status, intelligence development status, etc., during treatment with KUVAN |
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| Measure | Description | Time Frame |
|---|---|---|
| Diagnosis on BH4 deficiency based on medical history - laboratory testing results indicating sub-type (% in each sub-type) • Incidents and severity of AE reported (% of incident / % of each severity) | 2010-2015 | |
| Retrospective KUVAN treatment history - dose (mg/kg/day), | 2010-2015 | |
| Retrospective KUVAN treatment history - length of exposure (days) | 2010-2015 | |
| Retrospective Baseline Phe concentration ≥ 450 μmol/L - laboratory testing results (μmol/L) | 2010-2015 | |
| Number of participants with AE reported (% of patient with AE reported) | 2010-2015 | |
| Incidents and severity of AE reported (% of incident / % of each severity) | 2010-2015 | |
| Retrospective blood Phe level as indicated in laboratory testing results (μmol/L) | 2010-2015 | |
| Retrospective body height as indicated in medical note (cm) | 2010-2015 | |
| Retrospective body weight as indicated in medical note (kg) | 2010-2015 | |
| Retrospective occipital / frontal circumferences as indicated in medical note (cm) |
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Inclusion Criteria:
Exclusion Criteria:
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Chinese subjects with HPA caused by BH4 deficiency
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director, MD | Medical Director | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| West China Hospital of Sichuan University | Chengdu | Sichuan | 610041 | China |
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| ID | Term |
|---|---|
| D010661 | Phenylketonurias |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C003402 | sapropterin |
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| 2010-2015 |
| Retrospective intelligence development status via China local standard development assessment method (score) | 2010-2015 |
| D009422 | Nervous System Diseases |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |