Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
Study Details
Study Description
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 | Intervention/Treatment | Phase |
|---|---|---|
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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 90 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.
Study Design
Outcome Measures
Primary Outcome Measures
- 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. [15 months]
- Intra-subject change in serum markers of protein status at months 0, 9, and 15. [15 months]
- Intra-subject difference in lean body mass (%LBM) from month 0 to 15. [15 months]
- Intra-subject difference in bone mineral density from month 0 to 15. [15 months]
Secondary Outcome Measures
- Intra-subject change in concentrations of serum markers of micronutrient status at months 0, 9, and 15. [15 months]
- Intra-subject change in serum concentrations of essential fatty acid nutriture at months 0, 9, and 15. [15 months]
- Intra-subject change in BMI at months 0, 9, and 15. [15 months]
- Intra-subject change in serum markers of cardiovascular status at months 0, 9, and 15. [15 months]
- Intra-subject change in emotional eating, cognitive restraint, uncontrolled eating at months 0, 9, and 15. [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.
- Intra-subject change in food neophobia at months 0, 9, and 15. [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).
- Intra-subject change in Epicurean eating tendencies at months 0, 9, and 15. [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.
Eligibility Criteria
Criteria
Inclusion Criteria:
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Diagnosis of phenylketonuria
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Aged 18-65 years
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Following a protein-restricted diet with or without medical food within the last 30 days
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Routine natural protein intake from food is less than the RDA for protein based on weight (0.8 g/kg/day).
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If not consuming animal proteins, recommended protein intake is 115% RDA due to lower bioavailability of plant proteins.
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Within ±90 days of starting treatment with pegvaliase at time of study enrollment
Exclusion Criteria:
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Unable to consent to study
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Under age 18 or over age 65 years
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Routine natural protein intake is greater than the RDA for protein (0.8 g/kg/d) within the last 30 days.
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Females who are currently pregnant or lactating
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Boston Children's Hospital | Boston | Massachusetts | United States | 02115 |
Sponsors and Collaborators
- Boston Children's Hospital
- BioMarin Pharmaceutical
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- IRB-P00029889