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

NCT03856203 | Completed DMC

• 1 other identifier: IRB-P00029889
• Study Type: observational
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Phenylketonurias

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (7)

• 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

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Boston Children's Hospital: lead
• BioMarin Pharmaceutical: collaborator

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 14, 2019
• First Posted: February 27, 2019
• Study Start: March 8, 2019
• Primary Completion: December 31, 2022
• Study Completion: October 20, 2023
• Last Updated: November 18, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)