NCT01016392
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.
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.
All
From 4 Years
No
Observational
627
2009-12
2022-04-15
Graz, , Austria
Innsbruck, , Austria
Salzburg, , Austria
Vienna, , Austria
Amiens, , France
Angers, , France
Bordeaux, , France
Brest, , France
Bron, , France
Caen, , France
Dijon, , France
Lille, , France
Limoges, , France
Marseille, , France
Nantes, , France
Nice, , France
Paris, , France
Paris, , France
Reims, , France
Rouen, , France
Strasbourg, , France
Tarbes, , France
Toulouse, , France
Tours, , France
Vandoeuvre les Nancy, , France
Berlin, , Germany
Cottbus, , Germany
Duesseldorf, , Germany
Frankfurt, , Germany
Hamburg, , Germany
Heidelberg, , Germany
Jena, , Germany
Leipzig, , Germany
Magdeburg, , Germany
Muenchen, , Germany
Muenster, , Germany
Reutlingen, , Germany
Bologna, , Italy
Catania, , Italy
Catanzaro, , Italy
Genova, , Italy
Milano, , Italy
Napoli, , Italy
Napoli, , Italy
Padova, , Italy
Roma, , Italy
Amsterdam, , Netherlands
Groningen, , Netherlands
Maastricht, , Netherlands
Rotterdam, , Netherlands
Utrecht, , Netherlands
Porto, , Portugal
Banska Bystrica, , Slovakia
Kosice, , Slovakia
Bilbao, , Spain
Esplugues De Llobregat, , Spain
Madrid, , Spain
Malaga, , Spain
Murcia, , Spain
Palma De Mallorca, , Spain
Pamplona, , Spain
Santa Cruz de Tenerife, , Spain
Santander, , Spain
Santiago de Compostela, , Spain
Sevilla, , Spain
Zaragoza, , Spain
Gothenburg, , Sweden
Stockholm, , Sweden
Uppsala, , Sweden
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