Clinical Trial: Quantitative Requirements of Docosahexaenoic Acid for Neural Function in Children With Phenylketonuria

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Quantitative Requirements of Docosahexaenoic Acid for Neural Function in Children With Phenylketonuria

Brief 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 15 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 i

Detailed Summary:
Sponsor: Ludwig-Maximilians - University of Munich

Current Primary Outcome: latency of visually evoked potentials [ Time Frame: assessed basally (before intervention start) and at the end of the 6 month intervention period ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • fatty acid composition of plasma phospholipids [ Time Frame: assessed basally (before intervention start) and at the end of the 6 month intervention period ]
  • fine motor skills [ Time Frame: assessed basally (before intervention start) and at the end of the 6 month intervention period ]
  • test of reaction time [ Time Frame: assessed basally (before intervention start) and at the end of the 6 month intervention period ]


Original Secondary Outcome: Same as current

Information By: Ludwig-Maximilians - University of Munich

Dates:
Date Received: April 22, 2009
Date Started: May 2009
Date Completion:
Last Updated: June 19, 2014
Last Verified: June 2014