Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

Part of paid clinical trials in Atlanta, Georgia.

Sponsor
Emory University
Study ID
NCT04404530
Status
Recruiting
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Conditions

  • Phenylketonurias

Eligibility Criteria

Sex
ALL
Age
16 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Palynziq — DRUG
    Participants will take Palynziq as prescribed by their genetics doctor. In accordance with the Palynziq Risk Evaluation and Mitigation Strategy (REMS) protocol, patients will have their first injection of Palynziq at the Emory Genetics Clinic under the supervision of a physician. After initiating therapy, patients will continue to mail in blood spot filter papers and 3-day dietary records to their clinic providers as standard components of clinical care.

Study Details

Phenylketonuria (PKU) is an inherited metabolic disorder that impairs the metabolism of the essential amino acid phenylalanine (Phe). Without stringent dietary control, Phe accumulates in the blood and brain of PKU patients, leading to severe cognitive deficits. Achieving metabolic control, defined as blood Phe levels within the range of 120-360 μmol/L, has been a significant challenge for PKU patients using traditional diet therapy. The new FDA approved pharmacologic treatment, Palynziq, offers a new approach that could significantly reduce the burden of PKU by improving blood Phe levels and allowing for a less restrictive diet. As little is known about the global metabolic and physiologic effects of Palynziq, the present study aims to capture changes in diet quality, neurological health, nutritional status, the nutritional metabolome, and patient perceptions of mental and social health with sustained Palynziq therapy.

Key Dates

Start date
Oct 8, 2019
Status verified
Jan 2026
Primary completion
Dec 31, 2028
Completion
Dec 31, 2028

Study Design

Enrollment
45 participants (estimated)

Arms

  • Arm: Palynziq Therapy for PKU
    Participants with PKU who are starting Palynziq therapy, or have recently started Palynziq therapy but have not achieved response.

Primary Outcome Measure

Intra-subject Change in Intact Protein Intake [ Time Frame: Baseline through 12 Months Post-Response (up to 14 months after baseline) ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Emory University Hospital Georgia Clinical Research CenterAtlantaGeorgia30322-
The Emory ClinicAtlantaGeorgia30322-

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By research site
Emory University Hospital Georgia Clinical Research Center· Atlanta, GAThe Emory Clinic· Atlanta, GA

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