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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	ClinicalTrials.gov
Last refreshed on:	12 December 2020
Main ID:	NCT03864029
Date of registration:	22/02/2019
Prospective Registration:	No
Primary sponsor:	BioMarin Pharmaceutical
Public title:	Retrospective Observational Safety Effectiveness With Kuvan in hpA ROSEKA
Scientific 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
Date of first enrolment:	October 10, 2017
Target sample size:	26
Recruitment status:	Completed
URL:	https://clinicaltrials.gov/show/NCT03864029
Study type:	Observational
Study design:
Phase:

Countries of recruitment
China

Contacts

Name:	Medical Director, MD
Address:
Telephone:
Email:
Affiliation:	Medical Director

Key inclusion & exclusion criteria

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.

Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Tetrahydrobiopterin Deficiency

Intervention(s)

Drug: KUVAN

Primary Outcome(s)

Retrospective KUVAN treatment history - dose (mg/kg/day), [Time Frame: 2010-2015]

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 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]

Incidents and severity of AE reported (% of incident / % of each severity) [Time Frame: 2010-2015]

Number of participants with AE reported (% of patient with AE reported) [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 KUVAN treatment history - length of exposure (days) [Time Frame: 2010-2015]

Retrospective intelligence development status via China local standard development assessment method (score) [Time Frame: 2010-2015]

Retrospective Baseline Phe concentration = 450 µmol/L - laboratory testing results (µmol/L) [Time Frame: 2010-2015]

Secondary Outcome(s)

Secondary ID(s)

BMN 162-504

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Quintiles, Inc.

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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