Retrospective Observational Safety Effectiveness With Kuvan in hpA (ROSEKA)

March 5, 2019 updated by: BioMarin Pharmaceutical

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

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 Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

26

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • West China Hospital of Sichuan University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Chinese subjects with HPA caused by BH4 deficiency

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Collaborators

Quintiles, Inc.

Investigators

  • Study Director: Medical Director, MD, Medical Director

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 10, 2017

Primary Completion (Actual)

March 9, 2018

Study Completion (Actual)

July 25, 2018

Study Registration Dates

First Submitted

February 22, 2019

First Submitted That Met QC Criteria

March 4, 2019

First Posted (Actual)

March 6, 2019

Study Record Updates

Last Update Posted (Actual)

March 7, 2019

Last Update Submitted That Met QC Criteria

March 5, 2019

Last Verified

March 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BMN 162-504

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Tetrahydrobiopterin Deficiency

Clinical Trials on KUVAN

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Guatemala

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe