NCT03777826

Effects of Synergy on Nutrient Intake and Acceptability in Phenylketonuria (PKU)

An Open-label, Explorative, Post Launch, Multicenter, Multi-country Intervention Study of PKU Synergy (an Amino-acid Mixture) to Evaluate Change in Nutrient Intake in PKU Subjects With an Increased Phe-tolerance/Intake

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Nutricia Research · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). Patients with mild PKU or partly responsive to the drug synthetic tetrahydrobiopterin (BH4) (Kuvan®) can change to a more relaxed diet. However due to difficulty to adapt their diet, these patients are at risk of an imbalanced nutritional status and an insufficient intake of specific micronutrients, essential amino acids and DHA (Docosahexaenoic acid). The study product is designed to improve the nutritional status of the patients. The study investigates if the nutritional status is indeed improved following 24 week use of the study product, and also the study aims to evaluate product acceptability.

Conditions

• Phenylketonurias

Interventions

Type	Name	Description
OTHER	PKU Synergy	PKU Synergy is a citrus flavored, powdered amino-acid mixture (containing traces of Phe, 4,3mg per portion; and 20 gr. Protein Equivalent (PE)) with a tailored amino acid and micronutrient profile adapted for the special requirements of HPA/PKU (Hyperphenylalaninemia/Phenylketonuria) subjects over 10 years of age with an increased Phenylalanine-(Phe) tolerance/intake.

Timeline

• Start date: 2019-06-26
• Primary completion: 2021-05-27
• Completion: 2021-05-27
• First posted: 2018-12-17
• Last updated: 2021-12-09

Locations

3 sites across 2 countries: Germany, Netherlands