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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

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.

Condition or disease Intervention/treatment
Tetrahydrobiopterin Deficiency Drug: KUVAN

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Study Type : Observational
Actual Enrollment : 26 participants
Observational Model: Cohort
Time Perspective: Retrospective
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
Actual Study Start Date : October 10, 2017
Actual Primary Completion Date : March 9, 2018
Actual Study Completion Date : July 25, 2018



Intervention Details:
  • 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


Primary Outcome Measures :
  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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Chinese subjects with HPA caused by BH4 deficiency
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03864029


Locations
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China, Sichuan
West China Hospital of Sichuan University
Chengdu, Sichuan, China, 610041
Sponsors and Collaborators
BioMarin Pharmaceutical
Quintiles, Inc.
Investigators
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Study Director: Medical Director, MD Medical Director
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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT03864029    
Other Study ID Numbers: BMN 162-504
First Posted: March 6, 2019    Key Record Dates
Last Update Posted: March 7, 2019
Last Verified: March 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Phenylketonurias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases