Status:
COMPLETED
Retrospective Observational Safety Effectiveness With Kuvan in hpA
Lead Sponsor:
BioMarin Pharmaceutical
Collaborating Sponsors:
Quintiles, Inc.
Conditions:
Tetrahydrobiopterin Deficiency
Eligibility:
All Genders
Brief 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 med...
Eligibility Criteria
Inclusion
- Willing and able to provide informed consent (unless exemption of obtaining consent is obtained as per local regulation). Assent (if required defined by local regulation and requirement) should also be obtained from the subject and the legal authorized representative.
- Diagnosed with BH4 deficiency per local practice.
- KUVAN® was taken at least 1 dose to treat HPA caused by BH4 deficiency during the period of observation.
- Baseline Phe concentration ≥ 450 µmol/L
Exclusion
- Subject diagnosed to have Phenylketonuria (PKU)
- Has any condition that, in the view of the Investigator, the medical record of the subject in the duration of observation is not reliable, or not accessible.
Key Trial Info
Start Date :
October 10 2017
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
July 25 2018
Estimated Enrollment :
26 Patients enrolled
Trial Details
Trial ID
NCT03864029
Start Date
October 10 2017
End Date
July 25 2018
Last Update
March 7 2019
Active Locations (1)
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1
West China Hospital of Sichuan University
Chengdu, Sichuan, China, 610041