Retrospective Observational Safety Effectiveness With Kuvan in hpA (ROSEKA)

BioMarin Pharmaceutical

Status: Completed

Conditions: Tetrahydrobiopterin Deficiency

Treatments: Drug: KUVAN

Study type: Observational

Funder types: Industry

Identifiers

NCT03864029

BMN 162-504

Details and patient eligibility

About

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.

Enrollment: 26 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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 criteria

• 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.