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

ABSTRACT:

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®) 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.

Results

In total, 109 male or female children <4 years with confirmed BH₄-responsive phenylketonuria or mild hyperphenylalaninemia and good adherence to dietary treatment were screened. 56 patients were randomly assigned (1:1) to 10 

SUBMITTER: Muntau AC 

PROVIDER: S-EPMC5343543 | biostudies-literature | 2017 Mar

REPOSITORIES: biostudies-literature