Status:

COMPLETED

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

Lead Sponsor:

Nutricia Research

Conditions:

Phenylketonurias

Eligibility:

All Genders

12+ years

Phase:

NA

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 Pheny...

Eligibility Criteria

Inclusion

  • 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:
  • Mild PKU phenotype
  • BH4 treatment
  • 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
  • Willing and able to provide informed consent (and assent in case of minors if required by local law/regulations)
  • For women of childbearing potential: not to have the intention to become pregnant during the study
  • Exclusion Criteria:
  • For women: Currently pregnant or lactating
  • Current or prior use of the test product within six weeks prior to entry into the study
  • Concurrent conditions (including renal failure and severe hepatic failure) and medication that could interfere with participation, outcome parameters or safety (as determined by Investigator)
  • Psychotropic medication (i.e. medication affecting the nervous system) and inotropic medication
  • Any condition creating high risk of poor compliance with study
  • 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.

Exclusion

    Key Trial Info

    Start Date :

    June 26 2019

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ACTUAL

    End Date :

    May 27 2021

    Estimated Enrollment :

    13 Patients enrolled

    Trial Details

    Trial ID

    NCT03777826

    Start Date

    June 26 2019

    End Date

    May 27 2021

    Last Update

    December 9 2021

    Active Locations (3)

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    Page 1 of 1 (3 locations)

    1

    University hospital Leipzig

    Leipzig, Germany

    2

    University hospital Münster

    Münster, Germany

    3

    University Medical Center Groningen (UMCG)

    Groningen, Netherlands