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Trial record 5 of 11 for:    Boston Children's | pku

Nutrition Status of Adults Treated With Pegvaliase

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: NCT03792451
Recruitment Status : Recruiting
First Posted : January 3, 2019
Last Update Posted : August 14, 2019
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Krista Viau, Boston Children’s Hospital

Brief Summary:
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.

Condition or disease Intervention/treatment
Phenylketonurias Drug: Pegvaliase

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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase
Actual Study Start Date : January 17, 2019
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : June 2020



Intervention Details:
  • Drug: Pegvaliase
    Treated with pegvaliase for one or more years or followed an unrestricted diet for 6 or more months while being treated with pegvaliase.
    Other Name: Palynziq


Primary Outcome Measures :
  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 Outcome Measures :
  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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
This cross-sectional study will recruit adults with PKU who have been treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. This may include individuals who participated in the phase III clinical trials for pegvaliase.
Criteria

Inclusion Criteria:

  1. Diagnosis of phenylketonuria
  2. Aged 18-65 years
  3. Currently treated with pegvaliase
  4. 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
  5. Currently following an unrestricted diet that meets or exceeds the RDA for protein (0.8 g/kg/day)

Exclusion Criteria:

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

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): NCT03792451


Contacts
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Contact: Krista Viau, PhD, RD 617-355-6394 krista.viau@childrens.harvard.edu

Locations
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United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Krista Viau, PhD, RD       Krista.Viau@childrens.harvard.edu   
Contact: Eorna Maguire       Eorna.Maguire@childrens.harvard.edu   
Sponsors and Collaborators
Boston Children’s Hospital
BioMarin Pharmaceutical
Investigators
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Principal Investigator: Krista Viau, PhD, RD Boston Children’s Hospital

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Responsible Party: Krista Viau, Clinical Nutrition Specialist II, Boston Children’s Hospital
ClinicalTrials.gov Identifier: NCT03792451    
Other Study ID Numbers: P00029611
First Posted: January 3, 2019    Key Record Dates
Last Update Posted: August 14, 2019
Last Verified: August 2019
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
Product Manufactured in and Exported from the U.S.: 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