CompletedNot Applicable

Retrospective Observational Safety Effectiveness With Kuvan in hpA

China26 participantsStarted 2017-10-10

Plain-language 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.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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)

Timeframe: 2010-2015

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

Timeframe: 2010-2015

Retrospective KUVAN treatment history - length of exposure (days)

Timeframe: 2010-2015

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

Timeframe: 2010-2015

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

Timeframe: 2010-2015

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

Timeframe: 2010-2015

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

Trial details

NCT IDNCT03864029

SponsorBioMarin Pharmaceutical

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2018-03-09

View on ClinicalTrials.gov More Tetrahydrobiopterin Deficiency trials

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Timeframe: 2010-2015

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

Timeframe: 2010-2015

Retrospective KUVAN treatment history - length of exposure (days)

Timeframe: 2010-2015

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

Timeframe: 2010-2015

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

Timeframe: 2010-2015

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

Timeframe: 2010-2015

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