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Completed

NCT Number: NCT03792451

Nutrition Status of Adults Treated With Pegvaliase

Conduct a cross-sectional analysis of the nutritional status of individuals with PKU treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. We will evaluate laboratory markers of nutritional status, including markers of protein and micronutrient nutriture. In addition, a three-day food record and food frequency questionnaire will be obtained to assess protein intake and overall diet quality.

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Key information

Conditions

Phenylketonurias Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of phenylketonuria
  • Aged 18-65 years
  • Currently treated with pegvaliase
  • Have either 1) received treatment with pegvaliase for one or more years or 2) followed an unrestricted diet for 6 or more months while on pegvaliase
  • Currently following an unrestricted diet that meets or exceeds the RDA for protein (0.8 g/kg/day)

Exclusion criteria

  • Unable to consent to study
  • Under age 18 years or over age 65
  • Routine food intake provides less than the RDA for protein (0.8 g/kg/d)
  • Consuming any PKU medical foods

Treatment and study plan

Pegvaliase

Drug

Treated with pegvaliase for one or more years or followed an unrestricted diet for 6 or more months while being treated with pegvaliase.

Other names: Palynziq

Primary outcomes

  1. Mean concentrations of serum marker(s) of protein status

    Time frame: through study completion, an average of 1 year

  2. Mean concentrations of serum marker(s) of micronutrient status

    Time frame: through study completion, an average of 1 year

  3. Mean concentrations of serum marker(s) of essential fatty acid status

    Time frame: through study completion, an average of 1 year

  4. Mean concentrations of serum marker(s) of cardiovascular status

    Time frame: through study completion, an average of 1 year

  5. Mean protein intake (g/day) as reported on a three-day food record

    Time frame: through study completion, an average of 1 year

  6. Mean micronutrient intake (%DRI) as reported on a three-day food record

    Time frame: through study completion, an average of 1 year

Secondary outcomes

  1. Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III

    Time frame: through study completion, an average of 1 year

    "The Healthy Eating Index-2015 (HEI-2015) is… used to assess how well a set of foods aligns with the 2015-2020 Dietary Guidelines for Americans. The HEI-2015 includes 13 components that can be summed to a maximum total score of 100 points. The components capture… adequacy components and… moderation components. For the adequacy components, higher scores reflect higher intakes that meet or exceed the standards. For the moderation components, higher scores reflect lower intakes because lower intakes are more desirable. A higher total score indicates a diet that aligns better with the Dietary Guidelines." - https://www.cnpp.usda.gov/sites/default/files/healthy_eating_index/HEI2015-ScoresForAmericans.pdf

    Adequacy components include total fruits, whole fruits, total vegetables, greens and beans, whole grains, dairy, total protein foods, seafood and plant proteins, fatty acids. Moderation components include refined grains, sodium, added sugars, and saturated fats.

  2. Correlation between serum micronutrient concentrations and average micronutrient intake

    Time frame: through study completion, an average of 1 year

Sponsors and collaborators

Lead sponsor

Boston Children's Hospital

Other

Collaborators

  • BioMarin Pharmaceutical

Registry information

Official study title

Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
Jan 3, 2019
Registry last updated
Mar 4, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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