Retrospective Observational Safety Effectiveness With Kuvan in hpA

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.
    



Study Type

Observational


Primary Outcome

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)


Condition

Tetrahydrobiopterin Deficiency

Intervention

KUVAN


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

26

Start Date

October 10, 2017

Completion Date

July 25, 2018

Primary Completion Date

March 9, 2018

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.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Medical Director, MD, , 

Location Countries

China

Location Countries

China

Administrative Informations


NCT ID

NCT03864029

Organization ID

BMN 162-504


Responsible Party

Sponsor

Study Sponsor

BioMarin Pharmaceutical

Collaborators

 Quintiles, Inc.

Study Sponsor

Medical Director, MD, Study Director, Medical Director


Verification Date

March 2019