Mobile

ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 da...

Mise à jour : Il y a 5 ans
Référence : EUCTR2009-018168-81

ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days

Femme et Homme

  • | Pays :
    • -
  • | Organes :
    • -
  • | Spécialités :
    • -

Extrait

The primary objective of the study is to evaluate the proportion of responders (≥30% reduction from baseline in blood Phe level) to 20 mg/kg/day Sapropterin dihydrochloride treatment at several time points during 28 +/- 1 days.

Critère d'inclusion

  • Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA))

Liens