Retrospective Observational Safety Effectiveness With Kuvan in hpA (ROSEKA)

• ClinicalTrials.gov Identifier: NCT03864029
• Recruitment Status: Completed
• First Posted: March 6, 2019
• Last Update Posted: March 7, 2019
• Sponsor: BioMarin Pharmaceutical
• Collaborator: Quintiles, Inc.
• Information provided by (Responsible Party): BioMarin Pharmaceutical

Tracking Information

• First Submitted Date: February 22, 2019
• First Posted Date: March 6, 2019
• Last Update Posted Date: March 7, 2019
• Actual Study Start Date: October 10, 2017
• Actual Primary Completion Date: March 9, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 4, 2019)

• 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) [ Time Frame: 2010-2015 ]

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Retrospective Observational Safety Effectiveness With Kuvan in hpA
• 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
• Brief 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.
• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Retrospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Chinese subjects with HPA caused by BH4 deficiency
• Condition: 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

Other Name: Diagnostic Tests

• Study Groups/Cohorts: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 4, 2019): 26
• Original Actual Enrollment: Same as current
• Actual Study Completion Date: July 25, 2018
• Actual Primary Completion Date: March 9, 2018 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: China

Administrative Information

• NCT Number: NCT03864029
• Other Study ID Numbers: BMN 162-504
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Responsible Party: BioMarin Pharmaceutical
• Study Sponsor: BioMarin Pharmaceutical
• Collaborators: Quintiles, Inc.

Investigators

• Study Director: Medical Director, MD; Medical Director

• PRS Account: BioMarin Pharmaceutical
• Verification Date: March 2019