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.
- Conditions
- Phenylketonuria / PKU10021605
- Registration Number
- NL-OMON48912
- Lead Sponsor
- utricia Research
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
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.
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
- Not specified
- Primary Outcome Measures
Name Time Method <p>Main outcome parameters:<br /><br>*Nutrient intake (three-day diet diary)<br /><br>*Product acceptability (questionnaire)</p><br>
- Secondary Outcome Measures
Name Time Method <p>Other parameters:<br /><br>*Compliance (weekly)<br /><br>*Blood chemistry: Full fatty acid profile, full amino acid profile, Vitamins<br /><br>B1, B6, B12 & D; Folic acid; Selenium; Iodine; Calcium; Zinc; Iron; Copper;<br /><br>Manganese<br /><br>*Biweekly dried bloodspot Phe-levels </p><br>