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Nutritional Impacts of Palynziq for PKU

This study is looking at how the medication Palynziq affects the diet of people with Phenylketonuria (PKU). PKU is a condition where your body can't properly break down an amino acid called phenylalanine (Phe), which can cause health problems if not managed. Palynziq is a new treatment that may help lower Phe levels and allow for a less strict diet. Researchers want to see how much intact protein (from regular food) and medical food protein (special PKU formulas) you can eat after starting Palynziq. You can join if you are 16 or older, have PKU, and have a prescription for Palynziq. The study will track changes in your protein intake for at least 12 months, and for some, up to 6 years.

Study design
This is an observational study, meaning researchers will watch and collect information about 45 participants who are already taking Palynziq.
What's involved
You will take Palynziq as prescribed by your doctor. Your first injection will be supervised at the Emory Genetics Clinic. The study will measure your protein intake at the beginning and then at 12 months, with some participants followed for up to 6 years.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for changes in protein intake for 12 months after starting Palynziq, with a sub-study extending follow-up for an additional five years (6 years total).

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NCT04404530

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

Recruiting
Not specifiedAges 16+Observational
Emory University
~45 participants
Updated 2026-01-14 on ClinicalTrials.gov
What's tested:Palynziq

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Intra-subject Change in Intact Protein Intake
Measured over Baseline through 12 Months Post-Response (up to 14 months after baseline)
+2 more outcomes measured

Conditions

Phenylketonurias

Explore related trials

Phenylketonuria

Where it's being run

2 sites across 1 states
Georgia2

Study leadership

  • Rani Singh, PhD, RDN, LD · PRINCIPAL_INVESTIGATOR · Emory University

Who to contact

Rani Singh, PhD, RDN, LD
[email protected]
778-404-8519
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

16 years of age or older
diagnosed with PKU through newborn screening or via diagnosis later in life
capable of providing consent for medical tests and procedures
prescription for Palynziq and be enrolled in the Palynziq Risk Evaluation and Mitigation Strategy (REMS) program
Substudy: Participants must have completed visit 2 of the main study

Exclusion

unable to provide consent
a diagnosis of, or take medication for psychiatric, behavioral, or other inherited metabolic disorders

What this trial measures

  • Intra-subject Change in Intact Protein IntakeBaseline through 12 Months Post-Response (up to 14 months after baseline)

    Intra-subject change in intact protein intake will be assessed by participant-reported 3-day dietary records.

  • Intra-subject Change in Medical Food Protein IntakeBaseline through 12 Months Post-Response (up to 14 months after baseline)

    Intra-subject change in medical food protein intake will be assessed by participant-reported 3-day dietary records.

  • Sub study: Intra-subject change in intact protein and medical food protein intake.12 Months Post-Response through additional five years (6 years post-response)

    Intra-subject change in medical food protein intake will be assessed by participant-reported 3-day dietary records.