Effects of Palynziq on diet and health in patients with PKU
Palynziq and PKU: Treatment Impacts on Diet Quality, Neurological Health, Nutritional Status, and the Metabolome
This study is testing how the medication Palynziq affects the diet and overall health of people with PKU who are 16 and older.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 45 (estimated) |
| Ages | 16 Years and up |
| Sex | All |
| Sponsor | Emory University Academic / other |
| Locations | 2 sites (Atlanta, Georgia and 1 other locations) |
| Trial ID | NCT04404530 on ClinicalTrials.gov |
What this trial studies
This observational study investigates the nutritional impacts of Palynziq, a newly approved medication for patients with Phenylketonuria (PKU). It aims to assess changes in diet quality, neurological health, nutritional status, and patient perceptions of mental and social health among individuals receiving Palynziq therapy. By capturing these effects, the study seeks to provide insights into how this treatment can improve metabolic control and overall well-being for PKU patients. Participants must be 16 years or older and enrolled in the Palynziq Risk Evaluation and Mitigation Strategy program.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 16 and older diagnosed with PKU who are prescribed Palynziq.
Not a fit: Patients unable to provide consent or those with psychiatric or other inherited metabolic disorders may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved dietary flexibility and better health outcomes for patients with PKU.
How similar studies have performed: While there have been studies on dietary management in PKU, the specific impacts of Palynziq on nutritional and health outcomes are novel and not extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Criteria: * unable to provide consent * a diagnosis of, or take medication for psychiatric, behavioral, or other inherited metabolic disorders
Where this trial is running
Atlanta, Georgia and 1 other locations
- Emory University Hospital Georgia Clinical Research Center — Atlanta, Georgia, United States (Recruiting)
- The Emory Clinic — Atlanta, Georgia, United States (Recruiting)
Study contacts
- Principal investigator: Rani Singh, PhD, RDN, LD — Emory University
- Study coordinator: Rani Singh, PhD, RDN, LD
- Email: rsingh@emory.edu
- Phone: 778-404-8519
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.