Skip to main content
OpenTrials
Completed

NCT Number: NCT03864029

Retrospective Observational Safety Effectiveness With Kuvan in hpA

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Tetrahydrobiopterin Deficiency Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Phenylketonurias

Sex eligibility

All sexes

Study type

Observational

Primary location

West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

KUVAN

Drug

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

Other names: Diagnostic Tests

Primary outcomes

  1. 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

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

    Time frame: 2010-2015

  3. Retrospective KUVAN treatment history - length of exposure (days)

    Time frame: 2010-2015

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

    Time frame: 2010-2015

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

    Time frame: 2010-2015

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

    Time frame: 2010-2015

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

    Time frame: 2010-2015

  8. Retrospective body height as indicated in medical note (cm)

    Time frame: 2010-2015

  9. Retrospective body weight as indicated in medical note (kg)

    Time frame: 2010-2015

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

    Time frame: 2010-2015

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

    Time frame: 2010-2015

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Collaborators

  • Quintiles, Inc.

Registry information

Official study 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

Acronym: ROSEKA

Important dates

Study start
2017
Primary completion
2018
Study completion
2018
First posted
Mar 6, 2019
Registry last updated
Mar 7, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.