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

Not Applicable

Completed

• Conditions: Phenylketonurias
• Interventions: Other: PKU Synergy

• Registration Number: NCT03777826
• Lead Sponsor: Nutricia Research

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-11-19
• Study First Submitted QC: 2018-12-14
• Study First Posted: 2018-12-17
• Study Start: 2019-06-26
• Primary Completion: 2021-05-27
• Study Completion: 2021-05-27
• Status Verified: 2021-12
• Last Update Submitted: 2021-12-08
• Last Update Posted: 2021-12-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 13

Inclusion Criteria

Not provided

Exclusion Criteria

1. For women: Currently pregnant or lactating
2. Current or prior use of the test product within six weeks prior to entry into the study
3. Concurrent conditions (including renal failure and severe hepatic failure) and medication that could interfere with participation, outcome parameters or safety (as determined by Investigator)
4. Psychotropic medication (i.e. medication affecting the nervous system) and inotropic medication
5. Any condition creating high risk of poor compliance with study
6. Participation in any other studies involving investigational or marketed products concomitantly or within six weeks prior to entry into the study. Except for studies related to Kuvan® (synthetic tetrahydrobiopterin (BH4)) without diagnostic, therapeutic or experimental intervention.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open label (1 arm)	PKU Synergy	Open label use of study product (post-marketing): PKU Synergy

Primary Outcome Measures

Name	Time	Method
Product acceptability	24 weeks	Product acceptability questionnaire \[category/score\] \[0-10\] and \[0-5\]
Nutrient intake	24 weeks	Change in nutrient intake after 24 weeks by analysis of 3 day diet diary. Nutrients (energy, micronutrients, essential amino acids and DHA) in \[(m)/(micro)gram/day\] or \[mg/day or mg/kg/day\]

Secondary Outcome Measures

Name	Time	Method
Blood chemistry: nutritional status	24 weeks	Blood chemistry: levels measured in blood: Full Fatty acid profile \[For each FA Erythrocyte Membranes level (%FA)\], full Amino acid profile \[µmol/L\], Vitamin B12 \[pM\], Vitamin D \[ng/ml /nmol/L\]; Folic acid \[nM\]; Selenium \[µM\]; Iodine \[µg/L/ ng/mL\]; Calcium \[mg/dL\]; Zinc \[µmol/L\]; Iron \[µg/L\] analyzed in blood samples by central laboratories
Anthropometrics: weight	24 weeks	Anthropometrics: Weight \[kg\]
Anthropometrics: BMI	24 weeks	Anthropometrics: BMI \[kg/m2\], calculated from weight/height
Compliance	24 weeks	Compliance (product consumption) using daily product intake diary \[number of products used per week; per sachet of 33g\]
Anthropometrics: height	24 weeks	Anthropometrics: Height \[cm or m\]
Phenylalanine (Phe) levels	24 weeks	Dried bloodspot Phenylalanine (Phe) levels \[µmol/L\]

Trial Locations

Locations (3)

University hospital Leipzig; 🇩🇪 Leipzig, Germany

University hospital Münster; 🇩🇪 Münster, Germany

University Medical Center Groningen (UMCG); 🇳🇱 Groningen, Netherlands