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PKU Low Calorie Drink Study

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: NCT04272736
Recruitment Status : Not yet recruiting
First Posted : February 17, 2020
Last Update Posted : February 17, 2020
Sponsor:
Information provided by (Responsible Party):
Nutricia UK Ltd

Brief Summary:
This study will evaluate the compliance, acceptability, gastrointestinal (GI) tolerance and safety of a lower calorie amino acid based liquid protein substitute for patients with Phenylketonuria (PKU) or hyperphenylalaninemia (HPA).

Condition or disease Intervention/treatment Phase
Phenylketonurias Hyperphenylalaninaemia Dietary Supplement: Low calorie protein substitute 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: Supportive Care
Official Title: Evaluating the Compliance, Acceptability, Safety and Tolerance of a Lower Calorie Protein Substitute for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Study
Estimated Study Start Date : September 1, 2020
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : December 31, 2023


Arm Intervention/treatment
Experimental: Low calorie protein substitute
Single arm designed, 3 day baseline, 28 day on the lower calorie amino acid based liquid protein substitute.
Dietary Supplement: Low calorie protein substitute
The evaluated dietary supplement is a lower calorie amino acid based liquid protein substitute designed for patients with PKU.




Primary Outcome Measures :
  1. Compliance [ Time Frame: Daily for 31 days ]
    Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded


Secondary Outcome Measures :
  1. Acceptability: Tick-box questionnaire [ Time Frame: Recorded at baseline and end of intervetnion (day 31) ]
    Tick-box questionnaire on overall liking and acceptability of product (ie dislike a lot, dislike moderately, neutral, like moderately, like a lot)

  2. Blood phenylalanine and other amino acid levels [ Time Frame: Recorded at baselien and end of intervention (day 31) ]
    Blood spot test.

  3. Gastro-intestinal tolerance [ Time Frame: recorded at baseline, beginning of intervention and end of intervention(12 out of 31 days in total) ]
    Questionnaire detailing any GI symptoms, severity (none, mild, moderate, severe) and change from usual.

  4. Nutrient intake [ Time Frame: Recorded at basleine and end of intervention (day 31) ]
    24h dietary recall.

  5. Anthropometry [ Time Frame: Recorded at baseline and end of intervention (day 31) ]
    Measurements of height (m) and weight (kg) at baseline and end of study.



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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female
  • 3 years of age or above
  • Diagnosed with classical or variant type phenylketonuria (PKU), or hyperphenylalaninemia (HPA)
  • Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalent daily, for at least 1 month prior to study commencement
  • Have a prescribed daily Phe allowance
  • Written informed consent from patient, or from parent / carer if applicable

Exclusion Criteria:

  • Pregnant or lactating
  • Requiring enteral tube or parenteral nutrition
  • Major hepatic or renal dysfunction
  • Participation in other studies within 1 month prior to entry to this study
  • Allergy to any of the study product ingredients
  • Investigator concern around the patient being underweight (e.g.: BMI lower than 18.5kg/m2 for adults) and/or having an eating disorder
  • Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements

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): NCT04272736


Contacts
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Contact: Gary Hubbard, PhD 07738024720 gary.hubbard@nutricia.com

Sponsors and Collaborators
Nutricia UK Ltd
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Responsible Party: Nutricia UK Ltd
ClinicalTrials.gov Identifier: NCT04272736    
Other Study ID Numbers: PKULite2018
First Posted: February 17, 2020    Key Record Dates
Last Update Posted: February 17, 2020
Last Verified: February 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