Retrospective Observational Safety Effectiveness With Kuvan in hpA

Tetrahydrobiopterin Deficiency Clinical Trial

— ROSEKA  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03864029
• Other study ID #: BMN 162-504
• Status: Completed
• Start date: October 10, 2017
• Est. completion date: July 25, 2018

Study information

• Verified date: March 2019
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 26
• Est. completion date: July 25, 2018
• Est. primary completion date: March 9, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Phenylketonurias
• Tetrahydrobiopterin Deficiency

Intervention

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

Locations

Country	Name	City	State
China	West China Hospital of Sichuan University	Chengdu	Sichuan

Sponsors (2)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical	Quintiles, Inc.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Retrospective KUVAN treatment history - dose (mg/kg/day),		2010-2015
Primary	Retrospective KUVAN treatment history - length of exposure (days)		2010-2015
Primary	Retrospective Baseline Phe concentration = 450 µmol/L - laboratory testing results (µmol/L)		2010-2015
Primary	Number of participants with AE reported (% of patient with AE reported)		2010-2015
Primary	Incidents and severity of AE reported (% of incident / % of each severity)		2010-2015
Primary	Retrospective blood Phe level as indicated in laboratory testing results (µmol/L)		2010-2015
Primary	Retrospective body height as indicated in medical note (cm)		2010-2015
Primary	Retrospective body weight as indicated in medical note (kg)		2010-2015
Primary	Retrospective occipital / frontal circumferences as indicated in medical note (cm)		2010-2015
Primary	Retrospective intelligence development status via China local standard development assessment method (score)		2010-2015