Tetrahydrobiopterin DeficiencyROSEKANCT03864029
Retrospective Observational Safety Effectiveness With Kuvan in hpA
An observational study of Tetrahydrobiopterin Deficiency, run by BioMarin Pharmaceutical at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Tetrahydrobiopterin Deficiency
- What is being tested?
- KUVAN
- How many people
- 26 participants
- Where
- 1 siteChina
- Study dates
- 10 Oct 2017to 25 Jul 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Chinese subjects with HPA caused by BH4 deficiency
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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
- Being tested
- KUVAN
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KUVANDrug
Also called Diagnostic Tests
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
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 2010-2015
Retrospective KUVAN treatment history - dose (mg/kg/day),
Measured: 2010-2015
Retrospective KUVAN treatment history - length of exposure (days)
Measured: 2010-2015
Retrospective Baseline Phe concentration ≥ 450 μmol/L - laboratory testing results (μmol/L)
Measured: 2010-2015
Number of participants with AE reported (% of patient with AE reported)
Measured: 2010-2015
Incidents and severity of AE reported (% of incident / % of each severity)
Measured: 2010-2015
Retrospective blood Phe level as indicated in laboratory testing results (μmol/L)
Measured: 2010-2015
Retrospective body height as indicated in medical note (cm)
Measured: 2010-2015
Retrospective body weight as indicated in medical note (kg)
Measured: 2010-2015
Retrospective occipital / frontal circumferences as indicated in medical note (cm)
Measured: 2010-2015
Retrospective intelligence development status via China local standard development assessment method (score)
Measured: 2010-2015
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2019
- Study start10 Oct 2017
- Primary completion9 Mar 2018
- Study completion25 Jul 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tetrahydrobiopterin DeficiencyStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Quintiles, Inc.
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Medical Director
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03864029
- Sponsor's protocol ID
- BMN 162-504
- Acronym
- ROSEKA
- Status last verified
- Mar 2019
- First submitted
- 22 Feb 2019
- Last updated
- 7 Mar 2019
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03864029
- Acronym / sponsor ID
- ROSEKA / BMN 162-504
- Conditions
- Tetrahydrobiopterin Deficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Collaborators
- Quintiles, Inc.
- Study start
- 10 Oct 2017 (actual)
- Primary completion
- 9 Mar 2018 (actual)
- Study completion
- 25 Jul 2018 (actual)
- First posted
- 6 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Chinese subjects with HPA caused by BH4 deficiency
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KUVANAlso: Diagnostic Testsretrospective 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 | Drug | – |
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)
Time frame: 2010-2015
Retrospective KUVAN treatment history - dose (mg/kg/day),
Time frame: 2010-2015
Retrospective KUVAN treatment history - length of exposure (days)
Time frame: 2010-2015
Retrospective Baseline Phe concentration ≥ 450 μmol/L - laboratory testing results (μmol/L)
Time frame: 2010-2015
Number of participants with AE reported (% of patient with AE reported)
Time frame: 2010-2015
Incidents and severity of AE reported (% of incident / % of each severity)
Time frame: 2010-2015
Retrospective blood Phe level as indicated in laboratory testing results (μmol/L)
Time frame: 2010-2015
Retrospective body height as indicated in medical note (cm)
Time frame: 2010-2015
Retrospective body weight as indicated in medical note (kg)
Time frame: 2010-2015
Retrospective occipital / frontal circumferences as indicated in medical note (cm)
Time frame: 2010-2015
Retrospective intelligence development status via China local standard development assessment method (score)
Time frame: 2010-2015
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2019.
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Active, not recruitingObservational study
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Spinal Muscular Atrophy, Fragile X Syndrome
Recruiting
Phenylalanine-free Diet for Patients With Secondary Hyperphenylalaninemia in ICU
Non-Phenylketonuric Hyperphenylalaninemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director, MD study director
Medical Director
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Tetrahydrobiopterin Deficiency, run by BioMarin Pharmaceutical at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Mar 2019.