Trials / Completed
CompletedNCT03864029
Retrospective Observational Safety Effectiveness With Kuvan in hpA
An Observational Study Research to Collect the Effectiveness and Safety Data of KUVAN® Retrospectively in Chinese Subjects With Hyperphenylalaninemia (HPA) Caused by Tetrahydrobiopterin (BH4) Deficiency
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 26 (actual)
- Sponsor
- BioMarin Pharmaceutical · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | KUVAN | 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 |
Timeline
- Start date
- 2017-10-10
- Primary completion
- 2018-03-09
- Completion
- 2018-07-25
- First posted
- 2019-03-06
- Last updated
- 2019-03-07
Locations
1 site across 1 country: China
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03864029. Inclusion in this directory is not an endorsement.