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Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
NCT04404530 · View on ClinicalTrials.gov ↗
Study Summary
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.
Conditions Studied
Interventions
- DRUG Palynziq
Study Locations (2)
Georgia
- Emory University Hospital Georgia Clinical Research Center — Atlanta
- The Emory Clinic — Atlanta
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 45 participants |
| Start Date | 2019-10-08 |
| Est. Completion | 2028-12 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04404530
The ClinicalTrials.gov registry entry for NCT04404530 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 45 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Emory University, which has 1,434 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Phenylketonurias appearing as the primary indexed condition, and to 1 intervention — of which Palynziq is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04404530 reports 2 study locations spanning 1 distinct geographic area — top geographies include Georgia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04404530 about?
NCT04404530 is a clinical study titled "Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)". 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 ...
What is the current status of trial NCT04404530?
This trial is currently recruiting. The enrollment target is 45 participants. The study started on 2019-10-08. Estimated completion is 2028-12.
What conditions does trial NCT04404530 study?
This clinical trial studies the following conditions: Phenylketonurias. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04404530?
The interventions under investigation include: Palynziq (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04404530?
This trial is sponsored by Emory University, which has 1,434 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04404530 being conducted?
This trial has 2 study locations across Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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