Status:
COMPLETED
Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
Lead Sponsor:
Boston Children's Hospital
Collaborating Sponsors:
BioMarin Pharmaceutical
Conditions:
Phenylketonurias
Eligibility:
All Genders
18-65 years
Brief Summary
Conduct a prospective, longitudinal study to evaluate nutritional status in adults with phenylketonuria (PKU) before and during treatment with pegvaliase (Palynziq™).
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...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
March 8 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
October 20 2023
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT03856203
Start Date
March 8 2019
End Date
October 20 2023
Last Update
November 18 2023
Active Locations (1)
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1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115