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.

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. - ESSENTIAL

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON48912

Enrollment

Registered

2020-02-26

Start date

2020-09-24

Completion date

Unknown

Last updated

2024-04-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Phenylketonuria / PKU

Interventions

PKU Synergy is intended for oral use only. The daily indicated dosage is one 33g stick pack of test product, taken once per day at a fixed time for 24 weeks, e.g. at breakfast or supper.

Eligibility

Age

12 Years to 99 Years

Inclusion criteria

Inclusion criteria: Inclusion criteria: 1. PKU subjects identified by newborn screening and started low-Phe diet before 3 months of age. 2. PKU subjects with an increased Phe-tolerance/intake due to: mild PKU phenotype or BH4 treatment. 3. 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. 4. Age*12 years. 5. 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 6. 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 7. 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 8. Willing and able to comply with study procedures, 9. Willing and able to provide informed consent (and assent in case of minors if required by local law/regulations). 10. For women of childbearing potential: not to have the intention to become pregnant during the study.

Exclusion criteria

Exclusion criteria: 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.

Design outcomes

Primary

Measure	Time frame
Main outcome parameters: Nutrient intake (three-day diet diary) Product acceptability (questionnaire)	—

Secondary

Measure	Time frame
Other parameters: Compliance (weekly) Blood chemistry: Full fatty acid profile, full amino acid profile, Vitamins B1, B6, B12 & D; Folic acid; Selenium; Iodine; Calcium; Zinc; Iron; Copper; Manganese *Biweekly dried bloodspot Phe-levels	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)