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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03777826
Recruitment Status : Recruiting
First Posted : December 17, 2018
Last Update Posted : June 18, 2020
Sponsor:
Information provided by (Responsible Party):
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.


Condition or disease Intervention/treatment Phase
Phenylketonurias Other: PKU Synergy Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : June 26, 2019
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : March 2021


Arm Intervention/treatment
Open label (1 arm)
Open label use of study product (post-marketing): PKU Synergy
Other: PKU Synergy
PKU Synergy is a citrus flavored, powdered amino-acid mixture (containing traces of Phe, 4,3mg per portion; and 20 gr. Protein Equivalent (PE)) with a tailored amino acid and micronutrient profile adapted for the special requirements of HPA/PKU (Hyperphenylalaninemia/Phenylketonuria) subjects over 10 years of age with an increased Phenylalanine-(Phe) tolerance/intake.




Primary Outcome Measures :
  1. Nutrient intake [ Time Frame: 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]

  2. Product acceptability [ Time Frame: 24 weeks ]
    Product acceptability questionnaire [category/score] [0-10] and [0-5]


Secondary Outcome Measures :
  1. Compliance [ Time Frame: 24 weeks ]
    Compliance (product consumption) using daily product intake diary [number of products used per week; per sachet of 33g]

  2. Phenylalanine (Phe) levels [ Time Frame: 24 weeks ]
    Dried bloodspot Phenylalanine (Phe) levels [µmol/L]

  3. Blood chemistry: nutritional status [ Time Frame: 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

  4. Anthropometrics: weight [ Time Frame: 24 weeks ]
    Anthropometrics: Weight [kg]

  5. Anthropometrics: height [ Time Frame: 24 weeks ]
    Anthropometrics: Height [cm or m]

  6. Anthropometrics: BMI [ Time Frame: 24 weeks ]
    Anthropometrics: BMI [kg/m2], calculated from weight/height


Other Outcome Measures:
  1. Safety Parameters (Incidence, frequency, seriousness, severity and relatedness of (Serious) Adverse Events) [ Time Frame: 24 weeks ]
    Incidence, frequency, seriousness, severity and relatedness of (Serious) Adverse Events

  2. Tolerance (Standard gastrointestinal questionnaire reporting) [ Time Frame: 24 weeks ]
    Tolerance: Standard gastrointestinal questionnaire reporting of diarrhea constipation, abdominal distension, nausea, vomiting, burping, flatulence, regurgitation, and colic/cramps (0, 1, 12 and 24 weeks) [absent, mild, moderate, severe]

  3. Subject characteristics [ Time Frame: week 0 ]
    • Age [years]
    • Sex [male/female]
    • Country [Germany/Netherlands]

  4. Phe tolerance level and/or range [ Time Frame: week 0 ]
    [mg Phe/day]

  5. PKU phenotype [ Time Frame: week 0 ]
    Recording of PKU (Phenylketonuria) genotype from medical history [hyperphenylalaninemia (HPA) or mild/moderate/classic PKU]



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria:

  1. PKU subjects identified by newborn screening and started low-Phe diet before 3 months of age
  2. PKU subjects (with or without current AAM use) with an increased Phe-tolerance/intake due to:

    • Mild PKU phenotype
    • 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:

  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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03777826


Contacts
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Contact: Nutricia Research +31 30 295000 Register.clinicalresearchnutricia@danone.com

Locations
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Germany
University hospital Leipzig Completed
Leipzig, Germany
University hospital Münster Completed
Münster, Germany
Netherlands
University Medical Center Groningen (UMCG) Recruiting
Groningen, Netherlands
Contact: F.J. van Spronsen         
Sponsors and Collaborators
Nutricia Research
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Responsible Party: Nutricia Research
ClinicalTrials.gov Identifier: NCT03777826    
Other Study ID Numbers: MPR16TA09791
First Posted: December 17, 2018    Key Record Dates
Last Update Posted: June 18, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Phenylketonurias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases