CompletedNot Applicable

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

Germany, Netherlands13 participantsStarted 2019-06-26

Plain-language 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.

Who can participate

Age range12 Years

SexALL

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Inclusion criteria

✓. PKU subjects identified by newborn screening and started low-Phe diet before 3 months of age
✓. PKU subjects (with or without current AAM use) with an increased Phe-tolerance/intake due to:
✓. If treated with BH4, PKU subjects should be on a stable BH4 treatment for at least 26 consecutive weeks up to start test product intake
✓. Age≥12 years
✓. If subjects (irrespective whether BH4 users or mild PKU) use amino-acid mixture(s; AAM), then a maximum of 25 Protein Equivalents (PE) derived from the AAM per day applies and usage on a daily basis for at least 26 consecutive weeks up to Visit 1
✓. If subjects (irrespective whether BH4 users or mild PKU) use AAMs they should be capable and willing to substitute their current AAM(s; maximum of 25 PE per day) with one portion of the test product per day
✓. If subjects (irrespective whether BH4 users or mild PKU) use omega-3, antioxidant, and/or vitamin supplements, to stop usage of the supplements and be able and willing to substitute with the test product
✓. Willing and able to comply with study procedures

Exclusion criteria

What they're measuring

Nutrient intake

Timeframe: 24 weeks

Product acceptability

Timeframe: 24 weeks

Trial details

NCT IDNCT03777826

SponsorNutricia Research

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-05-27

View on ClinicalTrials.gov More Phenylketonurias trials