Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Retrospective Observational Safety Effectiveness With Kuvan in hpA (ROSEKA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03864029
Recruitment Status : Completed
First Posted : March 6, 2019
Last Update Posted : March 7, 2019
Sponsor:
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
Change History
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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
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
Removed Location Countries  
 
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