Nutrition Status of Adults Treated With Pegvaliase

Phenylketonurias Clinical Trial

Official title:

Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03792451
• Other study ID #: P00029611
• Status: Completed
• Start date: January 17, 2019
• Est. completion date: December 31, 2020

Study information

• Verified date: March 2021
• Source: Boston Children's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: December 31, 2020
• Est. primary completion date: December 31, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Phenylketonurias

Intervention

Drug:
• Pegvaliase
Treated with pegvaliase for one or more years or followed an unrestricted diet for 6 or more months while being treated with pegvaliase.

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	Mean concentrations of serum marker(s) of protein status		through study completion, an average of 1 year
Primary	Mean concentrations of serum marker(s) of micronutrient status		through study completion, an average of 1 year
Primary	Mean concentrations of serum marker(s) of essential fatty acid status		through study completion, an average of 1 year
Primary	Mean concentrations of serum marker(s) of cardiovascular status		through study completion, an average of 1 year
Primary	Mean protein intake (g/day) as reported on a three-day food record		through study completion, an average of 1 year
Primary	Mean micronutrient intake (%DRI) as reported on a three-day food record		through study completion, an average of 1 year
Secondary	Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III	"The Healthy Eating Index-2015 (HEI-2015) is… used to assess how well a set of foods aligns with the 2015-2020 Dietary Guidelines for Americans. The HEI-2015 includes 13 components that can be summed to a maximum total score of 100 points. The components capture… adequacy components and… moderation components. For the adequacy components, higher scores reflect higher intakes that meet or exceed the standards. For the moderation components, higher scores reflect lower intakes because lower intakes are more desirable. A higher total score indicates a diet that aligns better with the Dietary Guidelines." - https://www.cnpp.usda.gov/sites/default/files/healthy_eating_index/HEI2015-ScoresForAmericans.pdf; Adequacy components include total fruits, whole fruits, total vegetables, greens and beans, whole grains, dairy, total protein foods, seafood and plant proteins, fatty acids. Moderation components include refined grains, sodium, added sugars, and saturated fats.	through study completion, an average of 1 year
Secondary	Correlation between serum micronutrient concentrations and average micronutrient intake		through study completion, an average of 1 year