Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) DeficiencyKAMPERNCT01016392
Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
An observational study of Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency, run by BioMarin Pharmaceutical at 69 sites in 9 countries.
Trial summary
Kuvan® is a synthetic copy of a body's own substance called tetrahydrobiopterin (BH4). BH4 is required by the body to use an amino acid called phenylalanine in order to build another substance called tyrosine. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency
- How many people
- 627 participants
- Where
- 69 sitesin 9 countries
- Study dates
- Dec 2009to 31 May 2021
- How long will I take part?
- 5 Years
- 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 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All subjects with HPA due to PKU or BH4 deficiency followed at participating centres. And specific groups (elderly, pediatric, pregnant women and subjects with renal or hepatic insufficiency).
Who may be able to join
- Adult or pediatric subject (no age limit) of either gender with HPA due to PKU or BH4 deficiency.
- Have been shown to be responsive to BH4 or Kuvan. (Note: For Spain only-Have been shown to be responsive to BH4 or for the newly diagnosed subjects to be responsive to Kuvan as defined in the Summary of Product Characteristics [SmPC]).
- Currently being treated with Kuvan® at a participating centre.
- Subject or parent/legal guardian willing and able to provide written signed informed consent and given before any data collection. If a child is old enough to read and write, a separate assent form will be given.
Who cannot join
- Known hypersensitivity to Kuvan®
- Legal incapacity or limited legal capacity without legal guardian representation
- Breast-feeding
Trial locations and contacts
69 sites in 9 countries.
69 sites in 9 countries
France21 sites
Research Site
Amiens
Research Site
Angers
Research Site
Bordeaux
Research Site
Brest
Research Site
Bron
Research Site
Caen
Research Site
Dijon
Research Site
Lille
Research Site
Limoges
Research Site
Marseille
Research Site
Nantes
Research Site
Nice
Research Site - Armand Trousseau
Paris
Research Site - Necker
Paris
Research Site
Reims
Research Site
Rouen
Research Site
Strasbourg
Research Site
Tarbes
Research Site
Toulouse
Research Site
Tours
Research Site
Vandœuvre-lès-Nancy
Germany12 sites
Research Site
Berlin
Research Site
Cottbus
Research Site
Düsseldorf
Research Site
Frankfurt
Research Site
Hamburg
Research Site
Heidelberg
Research Site
Jena
Research Site
Leipzig
Research Site
Magdeburg
Research Site
München
Research Site
Münster
Research Site
Reutlingen
Spain12 sites
Research Site
Bilbao
Research Site
Esplugues de Llobregat
Research Site
Madrid
Research Site
Málaga
Research Site
Murcia
Research Site
Palma de Mallorca
Research Site
Pamplona
Research Site
Santa Cruz de Tenerife
Research Site
Santander
Research Site
Santiago de Compostela
Research Site
Seville
Research Site
Zaragoza
Italy9 sites
Research Site
Bologna
Research Site
Catania
Research Site
Catanzaro
Research Site
Genova
Research Site
Milan
Research Site - Ospedale Annunziata
Naples
Research Site - Policlinico Federico
Naples
Research Site
Padova
Research Site
Roma
Netherlands5 sites
Research Site
Amsterdam
Research Site
Groningen
Research Site
Maastricht
Research Site
Rotterdam
Research Site
Utrecht
Austria4 sites
Research Site
Graz
Research Site
Innsbruck
Research Site
Salzburg
Research Site
Vienna
Sweden3 sites
Research Site
Gothenburg
Research Site
Stockholm
Research Site
Uppsala
About this study
Official title: Kuvan® Adult Maternal Pediatric European Registry
Kuvan® is a synthetic copy of a body's own substance called tetrahydrobiopterin (BH4). BH4 is required by the body to use an amino acid called phenylalanine in order to build another substance called tyrosine.
Kuvan® received marketed authorisation in Europe in December 2008 and is now available in several European countries for the treatment of Hyperphenylalaninemia (HPA).
Read more
The primary objective is to assess the long-term safety in subjects treated with Kuvan®.
Secondary objectives are to provide additional information regarding:
- Safety in specific subject groups (elderly, pediatric, pregnant women and subjects with renal or hepatic insufficiency).
- Growth and neurocognitive outcomes for subjects with hyperphenylalaninemia (HPA) who are receiving treatment with Kuvan®.
- Progress and outcome of pregnancy for women with HPA who become pregnant while receiving treatment with Kuvan® (these women will be enrolled in a dedicated sub-registry).
- Assessment of adherence to diet and to Kuvan®.
- Assessment of long-term sensitivity to Kuvan®treatment.
Read the detailed description
This is an observational, multicenter, drug registry Study. The study will have a total duration of 15 years, including a 10-year inclusion period. No diagnostic, therapeutic or experimental intervention is involved. Subjects will receive clinical assessments, medications and treatments solely as determined by their study physician.
- Conditions
- Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency
- Keywords
- Observational post-authorization safety study on Kuvan®.
- Medical subject headings
- Phenylketonurias
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs)
Measured: A maximum of 15 years treatment duration.
Secondary outcomes (9)
Incidence of AEs/SAEs in specific population (elderly, children, subjects with renal or hepatic insufficiency)
Measured: A maximum of 15 years treatment duration.
Description on somatic growth (in BH4 deficient children < 3 years)
Measured: A maximum of 15 years treatment duration.
Neurocognitive outcomes
Measured: A maximum of 15 years treatment duration.
Neurological and psychiatric assessment
Measured: A maximum of 15 years treatment duration.
Diet and Kuvan® treatment adherence
Measured: A maximum of 15 years treatment duration.
Long-term sensitivity to Kuvan® treatment
Measured: A maximum of 15 years treatment duration.
Blood Phe levels
Measured: A maximum of 15 years treatment duration.
Tyrosine (Tyr) levels
Measured: A maximum of 15 years treatment duration.
Pregnancy and delivery outcomes
Measured: A maximum of 15 years treatment duration.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2009 (estimated)
- Study startDec 2009
- Primary completion31 May 2021
- Study completion31 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) 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
- Prospective
- Enrolment
- 627 participants (actual)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ece Kucuksayrac, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01016392
- Sponsor's protocol ID
- EMR700773-001
- Acronym
- KAMPER
- Status last verified
- Apr 2022
- First submitted
- 18 Nov 2009
- Last updated
- 15 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Kuvan® Adult Maternal Pediatric European Registry
- ClinicalTrials.gov ID
- NCT01016392
- Acronym / sponsor ID
- KAMPER / EMR700773-001
- Conditions
- Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency
- Keywords
- Observational post-authorization safety study on Kuvan®.
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 627 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Dec 2009
- Primary completion
- 31 May 2021 (actual)
- Study completion
- 31 May 2021 (actual)
- First posted
- 19 Nov 2009 (estimated)
- 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.
Kuvan® is a synthetic copy of a body's own substance called tetrahydrobiopterin (BH4). BH4 is required by the body to use an amino acid called phenylalanine in order to build another substance called tyrosine.
Kuvan® received marketed authorisation in Europe in December 2008 and is now available in several European countries for the treatment of Hyperphenylalaninemia (HPA).
The primary objective is to assess the long-term safety in subjects treated with Kuvan®.
Secondary objectives are to provide additional information regarding:
- Safety in specific subject groups (elderly, pediatric, pregnant women and subjects with renal or hepatic insufficiency).
- Growth and neurocognitive outcomes for subjects with hyperphenylalaninemia (HPA) who are receiving treatment with Kuvan®.
- Progress and outcome of pregnancy for women with HPA who become pregnant while receiving treatment with Kuvan® (these women will be enrolled in a dedicated sub-registry).
- Assessment of adherence to diet and to Kuvan®.
- Assessment of long-term sensitivity to Kuvan®treatment.
Detailed description
This is an observational, multicenter, drug registry Study. The study will have a total duration of 15 years, including a 10-year inclusion period. No diagnostic, therapeutic or experimental intervention is involved. Subjects will receive clinical assessments, medications and treatments solely as determined by their study physician.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All subjects with HPA due to PKU or BH4 deficiency followed at participating centres. And specific groups (elderly, pediatric, pregnant women and subjects with renal or hepatic insufficiency).
Key inclusion criteria (4 listed)
- Adult or pediatric subject (no age limit) of either gender with HPA due to PKU or BH4 deficiency.
- Have been shown to be responsive to BH4 or Kuvan. (Note: For Spain only-Have been shown to be responsive to BH4 or for the newly diagnosed subjects to be responsive to Kuvan as defined in the Summary of Product Characteristics [SmPC]).
- Currently being treated with Kuvan® at a participating centre.
- Subject or parent/legal guardian willing and able to provide written signed informed consent and given before any data collection. If a child is old enough to read and write, a separate assent form will be given.
Key exclusion criteria (3 listed)
- Known hypersensitivity to Kuvan®
- Legal incapacity or limited legal capacity without legal guardian representation
- Breast-feeding
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs)
Time frame: A maximum of 15 years treatment duration.
9 secondary outcomes
Incidence of AEs/SAEs in specific population (elderly, children, subjects with renal or hepatic insufficiency)
Time frame: A maximum of 15 years treatment duration.
Description on somatic growth (in BH4 deficient children < 3 years)
Time frame: A maximum of 15 years treatment duration.
Neurocognitive outcomes
Time frame: A maximum of 15 years treatment duration.
Neurological and psychiatric assessment
Time frame: A maximum of 15 years treatment duration.
Diet and Kuvan® treatment adherence
Time frame: A maximum of 15 years treatment duration.
Long-term sensitivity to Kuvan® treatment
Time frame: A maximum of 15 years treatment duration.
Blood Phe levels
Time frame: A maximum of 15 years treatment duration.
Tyrosine (Tyr) levels
Time frame: A maximum of 15 years treatment duration.
Pregnancy and delivery outcomes
Time frame: A maximum of 15 years treatment duration.
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 15 Apr 2022.
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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.
Ece Kucuksayrac, MD study director
BioMarin Pharmaceutical
69 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency, run by BioMarin Pharmaceutical at 69 sites in 9 countries.
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 3 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 15 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 69 sites in France, Germany, Spain, Italy, Netherlands and 4 more countries. 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?
Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs) (time frame: A maximum of 15 years treatment duration.). 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 15 Apr 2022.