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Terminated No phase listed Observational

Natural History Clinical Study in Adult PKU

ClinicalTrials.gov ID: NCT04768348

Public ClinicalTrials.gov record NCT04768348. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 13, 2026, 12:25 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Prospective Study Investigating the Natural History of Adults With Phenylketonuria (PKU) Due to Phenylalanine Hydroxylase Deficiency

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

The objective of this study is to characterize the natural history of phenylketonuria (PKU) due to phenylalanine hydroxylase (PAH) deficiency in adults through prospective collection of clinical, cognitive, and quality of life assessments.

Study identification

NCT ID
NCT04768348
Recruitment status
Terminated
Study type
Observational
Phase
Not listed
Lead sponsor
Homology Medicines, Inc
Industry
Enrollment
7 participants

Conditions and interventions

Interventions

Not listed

Eligibility (public fields only)

Age range
18 Years to 55 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Apr 19, 2021
Primary completion
Jan 31, 2023
Completion
Jul 31, 2023
Last update posted
Aug 24, 2023

2021 – 2023

United States locations

U.S. sites
8
U.S. states
7
U.S. cities
8
Facility City State ZIP Site status
Kaiser Permanente Los Angeles Medical Center Los Angeles California 90027
Children's Hospital of Orange County Orange California 92868
University of South Florida Tampa Florida 33606
Emory University Hospital Atlanta Georgia 30322
Community Health Clinic Topeka Indiana 46571
University of Pittsburgh Medical Center- CHOP Pittsburgh Pennsylvania 15224
UT Southwestern Medical Center Dallas Texas 75390
University of Utah Health Salt Lake City Utah 84018

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT04768348, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Aug 24, 2023 · Synced Sep 13, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT04768348 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →