Retrospective Observational Safety Effectiveness With Kuvan in hpA

BioMarin Pharmaceutical26 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Tetrahydrobiopterin Deficiency

Interventions

KUVANDRUG

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

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (1)

West China Hospital of Sichuan University

Chengdu, Sichuan, China

Outcomes

Primary Outcomes

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)

Time frame: 2010-2015

Retrospective KUVAN treatment history - dose (mg/kg/day),

Time frame: 2010-2015

Retrospective KUVAN treatment history - length of exposure (days)

Time frame: 2010-2015

Retrospective Baseline Phe concentration ≥ 450 μmol/L - laboratory testing results (μmol/L)

Time frame: 2010-2015

Number of participants with AE reported (% of patient with AE reported)

Time frame: 2010-2015

Incidents and severity of AE reported (% of incident / % of each severity)

Time frame: 2010-2015

Retrospective blood Phe level as indicated in laboratory testing results (μmol/L)

Time frame: 2010-2015

Retrospective body height as indicated in medical note (cm)

Time frame: 2010-2015

Retrospective body weight as indicated in medical note (kg)

Time frame: 2010-2015

Retrospective occipital / frontal circumferences as indicated in medical note (cm)

Time frame: 2010-2015

Retrospective intelligence development status via China local standard development assessment method (score)

Data from ClinicalTrials.gov

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