An observational study in Phenylketonurias, sponsored by Boston Children's Hospital. Completed at 1 site in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-06-02.
Sponsored by Boston Children's Hospital · Observational
Conduct a prospective, longitudinal study to evaluate nutritional status in adults with phenylketonuria (PKU) before and during treatment with pegvaliase (Palynziq™).
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.
183 studies on the registry are indexed under Phenylketonurias; 38 are open to participants now.
This study's enrollment of 12 is below the median of 40 across 60 observational studies indexed under Phenylketonurias.
Browse Phenylketonurias studies →Boston Children's Hospital is the lead sponsor of 598 studies on the registry; 151 are open to participants now.
Of its 31 completed or terminated interventional studies of FDA-regulated products, 18 (58%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Exclusion Criteria:
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
Intra-subject change in serum markers of protein status at months 0, 9, and 15.
Time frame: 15 months
Intra-subject difference in lean body mass (%LBM) from month 0 to 15.
Time frame: 15 months
Intra-subject difference in bone mineral density from month 0 to 15.
Time frame: 15 months
Intra-subject change in concentrations of serum markers of micronutrient status at months 0, 9, and 15.
Time frame: 15 months
Intra-subject change in serum concentrations of essential fatty acid nutriture at months 0, 9, and 15.
Time frame: 15 months
Intra-subject change in BMI at months 0, 9, and 15.
Time frame: 15 months
Intra-subject change in serum markers of cardiovascular status at months 0, 9, and 15.
Time frame: 15 months
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.
Time frame: 15 months
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).
Time frame: 15 months
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.
Time frame: 15 months
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Jun 2024. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Boston Children's Hospital