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KAMPER

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

Ensayo de BIOMARIN PHARMACEUTICAL INC en Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency (del registro, en inglés).

Fase

no aplica

Estado

Completado

Participantes

627

previstos

Centros

69

Fin del objetivo primario

may 2021

fecha real

Qué significa cada fase y cada estado.

Estudio observacional. Comenzó en dic 2009.

Qué mide

Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs) (A maximum of 15 years treatment duration.) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroKuvan® 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.

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Todos los de BMRN, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT01016392), actualizada en abr 2022.