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ROSEKA: Retrospective Observational Safety Effectiveness With Kuvan in hpA

Sponsor
BioMarin Pharmaceutical (Industry)
Overall Status
Completed
CT.gov ID
NCT03864029
Collaborator
Quintiles, Inc. (Industry)
26
Enrollment
1
Location
9.5
Actual Duration (Months)
2.7
Patients Per Site Per Month

Study Details

Study Description

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 Phase

Study Design

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 :
Oct 10, 2017
Actual Primary Completion Date :
Mar 9, 2018
Actual Study Completion Date :
Jul 25, 2018

Outcome Measures

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) [2010-2015]

  2. Retrospective KUVAN treatment history - dose (mg/kg/day), [2010-2015]

  3. Retrospective KUVAN treatment history - length of exposure (days) [2010-2015]

  4. Retrospective Baseline Phe concentration ≥ 450 μmol/L - laboratory testing results (μmol/L) [2010-2015]

  5. Number of participants with AE reported (% of patient with AE reported) [2010-2015]

  6. Incidents and severity of AE reported (% of incident / % of each severity) [2010-2015]

  7. Retrospective blood Phe level as indicated in laboratory testing results (μmol/L) [2010-2015]

  8. Retrospective body height as indicated in medical note (cm) [2010-2015]

  9. Retrospective body weight as indicated in medical note (kg) [2010-2015]

  10. Retrospective occipital / frontal circumferences as indicated in medical note (cm) [2010-2015]

  11. Retrospective intelligence development status via China local standard development assessment method (score) [2010-2015]

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 West China Hospital of Sichuan University Chengdu Sichuan China 610041

Sponsors and Collaborators

  • BioMarin Pharmaceutical
  • Quintiles, Inc.

Investigators

  • Study Director: Medical Director, MD, Medical Director

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT03864029
Other Study ID Numbers:
  • BMN 162-504
First Posted:
Mar 6, 2019
Last Update Posted:
Mar 7, 2019
Last Verified:
Mar 1, 2019
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Phenylketonurias

Study Results

No Results Posted as of Mar 7, 2019