Clinical trial NCT03792451: Nutrition Status of Adults Treated With Pegvaliase
Official title: Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Phenylketonurias
Lead sponsor: Boston Children's Hospital
Sponsor class: OTHER
Collaborators: name: BioMarin Pharmaceutical
First posted: 2019-01-03
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
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.

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.


Eligibility 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

Primary outcomes:
measure: Mean concentrations of serum marker(s) of protein status
timeFrame: through study completion, an average of 1 year
measure: Mean concentrations of serum marker(s) of micronutrient status
timeFrame: through study completion, an average of 1 year
measure: Mean concentrations of serum marker(s) of essential fatty acid status
timeFrame: through study completion, an average of 1 year
measure: Mean concentrations of serum marker(s) of cardiovascular status
timeFrame: through study completion, an average of 1 year
measure: Mean protein intake (g/day) as reported on a three-day food record
timeFrame: through study completion, an average of 1 year
measure: Mean micronutrient intake (%DRI) as reported on a three-day food record
timeFrame: through study completion, an average of 1 year
Secondary outcomes:
measure: Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III
timeFrame: through study completion, an average of 1 year
measure: Correlation between serum micronutrient concentrations and average micronutrient intake
timeFrame: through study completion, an average of 1 year
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
Study officials:
affiliation: Boston Children's Hospital
name: Krista Viau, PhD, RD
role: PRINCIPAL_INVESTIGATOR
References: