Retrospective Observational Safety Effectiveness With Kuvan in hpA

NCT03864029 | Completed FDA Drug

• 1 other identifier: BMN 162-504
• Study Type: observational
• Target: 26
• Locations: 1 country
• Sites: 1

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.

Conditions

Tetrahydrobiopterin Deficiency

Outcome Measures

Primary Outcomes (11)

• 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

• Retrospective KUVAN treatment history - dose (mg/kg/day),

  2010-2015

• Retrospective KUVAN treatment history - length of exposure (days)

  2010-2015

• Retrospective Baseline Phe concentration ≥ 450 μmol/L - laboratory testing results (μmol/L)

  2010-2015

• Number of participants with AE reported (% of patient with AE reported)

  2010-2015

• Incidents and severity of AE reported (% of incident / % of each severity)

  2010-2015

• Retrospective blood Phe level as indicated in laboratory testing results (μmol/L)

  2010-2015

• Retrospective body height as indicated in medical note (cm)

  2010-2015

• Retrospective body weight as indicated in medical note (kg)

  2010-2015

• Retrospective occipital / frontal circumferences as indicated in medical note (cm)

  2010-2015

• Retrospective intelligence development status via China local standard development assessment method (score)

  2010-2015

Interventions

KUVAN DRUG

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

Also known as: Diagnostic Tests

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Chinese subjects with HPA caused by BH4 deficiency

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead
• Quintiles, Inc.: collaborator

Study Sites (1)

West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

Location

MeSH Terms

Conditions

Phenylketonurias

Interventions

sapropterin

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director, MD

  Medical Director

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2019
• First Posted: March 6, 2019
• Study Start: October 10, 2017
• Primary Completion: March 9, 2018
• Study Completion: July 25, 2018
• Last Updated: March 7, 2019

Record last verified: 2019-03

Locations

CN (1)