Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

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Trial Status Completed

Trial Identifier

NCT01016392

Condition

Phenylketonuria, Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

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.

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.

Eligibility Criteria

Birth Sex

All

Age

From 4 Years

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

627

Study Started:

2009-12

Study Updated:

2022-04-15

Trial Locations

  • Research Site

    Graz, , Austria

  • Research Site

    Innsbruck, , Austria

  • Research Site

    Salzburg, , Austria

  • Research Site

    Vienna, , Austria

  • Research Site

    Amiens, , France

  • Research Site

    Angers, , France

  • Research Site

    Bordeaux, , France

  • Research Site

    Brest, , France

  • Research Site

    Bron, , France

  • Research Site

    Caen, , France

  • Research Site

    Dijon, , France

  • Research Site

    Lille, , France

  • Research Site

    Limoges, , France

  • Research Site

    Marseille, , France

  • Research Site

    Nantes, , France

  • Research Site

    Nice, , France

  • Research Site - Armand Trousseau

    Paris, , France

  • Research Site - Necker

    Paris, , France

  • Research Site

    Reims, , France

  • Research Site

    Rouen, , France

  • Research Site

    Strasbourg, , France

  • Research Site

    Tarbes, , France

  • Research Site

    Toulouse, , France

  • Research Site

    Tours, , France

  • Research Site

    Vandoeuvre les Nancy, , France

  • Research Site

    Berlin, , Germany

  • Research Site

    Cottbus, , Germany

  • Research Site

    Duesseldorf, , Germany

  • Research Site

    Frankfurt, , Germany

  • Research Site

    Hamburg, , Germany

  • Research Site

    Heidelberg, , Germany

  • Research Site

    Jena, , Germany

  • Research Site

    Leipzig, , Germany

  • Research Site

    Magdeburg, , Germany

  • Research Site

    Muenchen, , Germany

  • Research Site

    Muenster, , Germany

  • Research Site

    Reutlingen, , Germany

  • Research Site

    Bologna, , Italy

  • Research Site

    Catania, , Italy

  • Research Site

    Catanzaro, , Italy

  • Research Site

    Genova, , Italy

  • Research Site

    Milano, , Italy

  • Research Site - Ospedale Annunziata

    Napoli, , Italy

  • Research Site - Policlinico Federico

    Napoli, , Italy

  • Research Site

    Padova, , Italy

  • Research Site

    Roma, , Italy

  • Research Site

    Amsterdam, , Netherlands

  • Research Site

    Groningen, , Netherlands

  • Research Site

    Maastricht, , Netherlands

  • Research Site

    Rotterdam, , Netherlands

  • Research Site

    Utrecht, , Netherlands

  • Research Site

    Porto, , Portugal

  • Research Site

    Banska Bystrica, , Slovakia

  • Research Site

    Kosice, , Slovakia

  • Research Site

    Bilbao, , Spain

  • Research Site

    Esplugues De Llobregat, , Spain

  • Research Site

    Madrid, , Spain

  • Research Site

    Malaga, , Spain

  • Research Site

    Murcia, , Spain

  • Research Site

    Palma De Mallorca, , Spain

  • Research Site

    Pamplona, , Spain

  • Research Site

    Santa Cruz de Tenerife, , Spain

  • Research Site

    Santander, , Spain

  • Research Site

    Santiago de Compostela, , Spain

  • Research Site

    Sevilla, , Spain

  • Research Site

    Zaragoza, , Spain

  • Research Site

    Gothenburg, , Sweden

  • Research Site

    Stockholm, , Sweden

  • Research Site

    Uppsala, , Sweden

Inclusion Criteria

  • 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.

Exclusion Criteria

  • Known hypersensitivity to Kuvan®
  • Legal incapacity or limited legal capacity without legal guardian representation
  • Breast-feedin

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