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NCT04404530 · ClinicalTrials.gov registry record
Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
A clinical trial of Phenylketonurias, sponsored by Emory University.
- Recruiting
- Registry status
- 45
- Enrollment target
- 2
- Study locations
NCT04404530: Recruiting study of Phenylketonurias, sponsored by Emory University.
NCT04404530 is a study of Phenylketonurias that is actively recruiting participants, run by Emory University. The registered enrollment target is 45 participants, above the 37-participant average among 5 other Phenylketonurias trials with a reported enrollment target (22% higher). The trial reports 2 study locations across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04404530, a study of Phenylketonurias, is actively recruiting participants, sponsored by Emory University.
- RECRUITING
- Registry status
- 45 participants
- Enrollment target
- 2
- Study locations
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.
Primary Outcome
Intra-subject change in intact protein intake will be assessed by participant-reported 3-day dietary records.
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 |
What NCT04404530 shows while recruiting
NCT04404530 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 45 participants, a relatively small participant target, above the 37-participant average among 5 other Phenylketonurias trials with a reported enrollment target (22% higher).
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.
NCT04404530 reports a single indexed study location in Georgia.
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.
What interventions are being tested in trial NCT04404530?
The interventions under investigation include: Palynziq (DRUG).
Who is sponsoring clinical trial NCT04404530?
This trial is sponsored by Emory University, which has 1,208 total clinical trials registered on ClinicalTrials.gov.
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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