Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients < 4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial


Muntau A. C. , Burlina A., Eyskens F., Freisinger P., De Laet C., Leuzzi V., ...Daha Fazla

ORPHANET JOURNAL OF RARE DISEASES, cilt.12, 2017 (SCI İndekslerine Giren Dergi) identifier identifier identifier

  • Cilt numarası: 12
  • Basım Tarihi: 2017
  • Doi Numarası: 10.1186/s13023-017-0600-x
  • Dergi Adı: ORPHANET JOURNAL OF RARE DISEASES

Özet

Background: Sapropterin dihydrochloride, a synthetic formulation of BH4, the cofactor for phenylalanine hydroxylase (PAH, EC 1.14.16.1), was initially approved in Europe only for patients >= 4 years with BH4-responsive phenylketonuria. The aim of the SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan r) trial was to assess the efficacy (improvement in daily phenylalanine tolerance, neuromotor development and growth parameters), safety and pharmacokinetics of sapropterin dihydrochloride in children < 4 years.