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Trial record 4 of 11 for:    Boston Children's | pku

Nutrition Status of Adults With PKU Before and During Treatment 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: NCT03856203
Recruitment Status : Recruiting
First Posted : February 27, 2019
Last Update Posted : April 16, 2019
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Krista Viau, Boston Children’s Hospital

Brief Summary:
Conduct a prospective, longitudinal study to evaluate nutritional status in adults with phenylketonuria (PKU) before and during treatment with pegvaliase (Palynziq™).

Condition or disease
Phenylketonurias

Detailed Description:
The investigators will compare intra-subject change in adults with PKU who follow a protein-restricted diet (protein intake less than the RDA for protein based on weight (0.8 g/kg/day) with or without a PKU medical food) within 60 days of starting pegvaliase (baseline) and 9 and 15 months later. The investigators will collect laboratory measures of nutrition status, anthropometrics, Dual-Energy X-Ray Absorptiometry (DXA) to measure body composition and bone mineral density (BMD), and indirect calorimetry to measure resting energy expenditure (REE). In addition, three-day food records will be obtained to assess protein intake and overall diet quality.

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Study Type : Observational
Estimated Enrollment : 30 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Longitudinal Assessment of Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
Actual Study Start Date : March 8, 2019
Estimated Primary Completion Date : March 2022
Estimated Study Completion Date : September 2022





Primary Outcome Measures :
  1. Intra-subject change in intact and medical food protein intake (grams/day) as reported on three-day food records at months 0, 9, and 15. [ Time Frame: 15 months ]
  2. Intra-subject change in serum markers of protein status at months 0, 9, and 15. [ Time Frame: 15 months ]
  3. Intra-subject difference in lean body mass (%LBM) from month 0 to 15. [ Time Frame: 15 months ]
  4. Intra-subject difference in bone mineral density from month 0 to 15. [ Time Frame: 15 months ]

Secondary Outcome Measures :
  1. Intra-subject change in concentrations of serum markers of micronutrient status at months 0, 9, and 15. [ Time Frame: 15 months ]
  2. Intra-subject change in serum concentrations of essential fatty acid nutriture at months 0, 9, and 15. [ Time Frame: 15 months ]
  3. Intra-subject change in BMI at months 0, 9, and 15. [ Time Frame: 15 months ]
  4. Intra-subject change in serum markers of cardiovascular status at months 0, 9, and 15. [ Time Frame: 15 months ]
  5. Intra-subject change in emotional eating, cognitive restraint, uncontrolled eating at months 0, 9, and 15. [ Time Frame: 15 months ]
    Three-Factor Eating Questionnaire (TFEQ)-18 is an 18-item questionnaire measured on a four-point scale cognitive restraint (6 items), uncontrolled eating (9 items) and emotional eating (3 items). Responses are measured on a four-point scale and the raw scores for each subscale are transformed to correspond to a 0-100 point scale. Higher scores in the respective scales are indicative of greater cognitive restraint, emotional eating, and uncontrolled eating.

  6. Intra-subject change in food neophobia at months 0, 9, and 15. [ Time Frame: 15 months ]
    Food Neophobia Scale, which was adapted for a PKU population, includes nine questions measured on a seven-point scale with a higher total score indicative of greater food neophobia (score range 9-63).

  7. Intra-subject change in Epicurean eating tendencies at months 0, 9, and 15. [ Time Frame: 15 months ]
    Epicurean eating tendencies questionnaire includes seven items measured with a seven-point scale (range 7-49). Higher scores indicative of greater Epicurean eating tendencies.



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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 study will recruit adults with PKU who are starting therapy with pegvaliase and currently following a protein-restricted diet with or without medical food.
Criteria

Inclusion Criteria:

  • Diagnosis of phenylketonuria
  • Aged 18-65 years
  • Following a protein-restricted diet with or without medical food within the last 30 days
  • Routine natural protein intake from food is less than the RDA for protein based on weight (0.8 g/kg/day).
  • If not consuming animal proteins, recommended protein intake is 115% RDA due to lower bioavailability of plant proteins.
  • Within ±60 days of starting treatment with pegvaliase at time of study enrollment

Exclusion Criteria:

  • Unable to consent to study
  • Under age 18 or over age 65 years
  • Routine natural protein intake is greater than the RDA for protein (0.8 g/kg/d) within the last 30 days.
  • Females who are currently pregnant or lactating

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): NCT03856203


Contacts
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Contact: Krista Viau, PhD, RD 617-355-6516 krista.viau@childrens.harvard.edu
Contact: Eorna Maguire Eorna.Maguire@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    617-355-6516    krista.viau@childrens.harvard.edu   
Sponsors and Collaborators
Boston Children’s Hospital
BioMarin Pharmaceutical

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Responsible Party: Krista Viau, Principal Investigator, Boston Children’s Hospital
ClinicalTrials.gov Identifier: NCT03856203    
Other Study ID Numbers: IRB-P00029889
First Posted: February 27, 2019    Key Record Dates
Last Update Posted: April 16, 2019
Last Verified: April 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
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