Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

Phenylketonurias Clinical Trial

Official title:

Longitudinal Assessment of Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03856203
• Other study ID #: IRB-P00029889
• Status: Completed
• Start date: March 8, 2019
• Est. completion date: October 20, 2023

Study information

• Verified date: November 2023
• Source: Boston Children's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: October 20, 2023
• Est. primary completion date: December 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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 ±90 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

Related Conditions & MeSH terms

• Phenylketonurias

Locations

Country	Name	City	State
United States	Boston Children's Hospital	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Boston Children's Hospital	BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Intra-subject change in serum markers of protein status at months 0, 9, and 15.		15 months
Primary	Intra-subject difference in lean body mass (%LBM) from month 0 to 15.		15 months
Primary	Intra-subject difference in bone mineral density from month 0 to 15.		15 months
Secondary	Intra-subject change in concentrations of serum markers of micronutrient status at months 0, 9, and 15.		15 months
Secondary	Intra-subject change in serum concentrations of essential fatty acid nutriture at months 0, 9, and 15.		15 months
Secondary	Intra-subject change in BMI at months 0, 9, and 15.		15 months
Secondary	Intra-subject change in serum markers of cardiovascular status at months 0, 9, and 15.		15 months
Secondary	Intra-subject change in emotional eating, cognitive restraint, uncontrolled eating at months 0, 9, and 15.	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.	15 months
Secondary	Intra-subject change in food neophobia at months 0, 9, and 15.	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).	15 months
Secondary	Intra-subject change in Epicurean eating tendencies at months 0, 9, and 15.	Epicurean eating tendencies questionnaire includes seven items measured with a seven-point scale (range 7-49). Higher scores indicative of greater Epicurean eating tendencies.	15 months