RECRUITING

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Palynziq and PKU: Treatment Impacts on Diet Quality, Neurological Health, Nutritional Status, and the Metabolome

Quick Facts

Study Start:2019-10-08
Study Completion:2027-11
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04404530

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:16 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * 16 years of age or older
  2. * diagnosed with PKU through newborn screening or via diagnosis later in life
  3. * capable of providing consent for medical tests and procedures
  4. * prescription for Palynziq and be enrolled in the Palynziq Risk Evaluation and Mitigation Strategy (REMS) program
  5. * Substudy: Participants must have completed visit 2 of the main study
  1. * unable to provide consent
  2. * a diagnosis of, or take medication for psychiatric, behavioral, or other inherited metabolic disorders

Contacts and Locations

Study Contact

Rani Singh, PhD, RDN, LD
CONTACT
778-404-8519
rsingh@emory.edu

Principal Investigator

Rani Singh, PhD, RDN, LD
PRINCIPAL_INVESTIGATOR
Emory University

Study Locations (Sites)

Emory University Hospital Georgia Clinical Research Center
Atlanta, Georgia, 30322
United States
The Emory Clinic
Atlanta, Georgia, 30322
United States

Collaborators and Investigators

Sponsor: Emory University

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2019-10-08
Study Completion Date2027-11

Study Record Updates

Study Start Date2019-10-08
Study Completion Date2027-11

Terms related to this study

Additional Relevant MeSH Terms

  • Phenylketonurias