Quantitative Requirements of Docosahexaenoic Acid for Neural Function in Children With Phenylketonuria
Condition: Phenylketonuria
MeSH terms: Phenylketonurias
Study type: Interventional

Summary: 
      Patients with phenylketonuria (PKU) have an inborn error in the metabolism of the amino acid
      phenylalanine (Phe) and thus must follow a strictly controlled protein-restricted diet from
      early infancy. This protein-restricted diet is devoid of natural dietary sources of n-3 long
      chain polyunsaturated fatty acids (LC-PUFA), such as eggs, meat, milk or fish. Therefore,
      blood concentrations of n-3 LC-PUFA, especially of docosahexaenoic acid (DHA) are reduced in
      PKU children compared to healthy controls. DHA availability is considered important for
      optimal neurological function. Previous studies have shown that neural function of PKU
      children is improved by high dose supplementation of fish oil providing DHA, as shown by
      significant improvements of both visual evoked potential latencies and of fine motor skills
      and coordination, but no dose response relationship has been established so far.

      This multicentric double-blind randomized trial aims at determining quantitative DHA
      requirements for optimal neural function in PKU children. Patients with classical PKU from
      several major treatment centers in Europe will be randomized to receive between 0 and 8 mg of
      DHA per kg body weight daily for a duration of 6 months. Biochemical (fatty acid composition
      of plasma phospholipids, lipoprotein metabolism and metabolic profiles), and functional
      testing (visual evoked potentials, fine motor skills, cognitive function and markers of
      immune function) will be performed at baseline and after 6 months. Intake per kg body weight
      will be related to outcome parameters and thus a possible dose response relationship will be
      defined. The results from this study are expected to contribute to the improvement of the
      diet of PKU patients, but they also have the potential to help defining quantitative DHA
      needs of healthy children.

      The primary hypothesis is that supplementation with DHA improves visual function in children
      with PKU.
    


Intervention name(s): high oleic sunflower oil; microalgal oil; microalgal oil; microalgal oil; microalgal oil
Intervention type(s): Dietary Supplement; Dietary Supplement; Dietary Supplement; Dietary Supplement; Dietary Supplement
Intervention description(s): placebo, which does not provide DHA; the supplement provides 20 mg DHA per capsule (1 or 2 are consumed per day, depending on body weight); the supplement provides 40 mg DHA per capsule (1 or 2 are consumed per day, depending on body weight); the supplement provides 80 mg DHA per capsule (1 or 2 are consumed per day, depending on body weight); the supplement provides 130 mg DHA per capsule (1 or 2 are consumed per day, depending on body weight)
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Basic Science
Keywords: phenylketonuria; docosahexaenoic acid; visually evoked potential; choice-reaction time