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Study identifier: NCT05166161 Synced from ClinicalTrials.gov · August 07, 2026
● Study status: Active Not Recruiting

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Condition: Phenylketonuria  ·  Sponsor: PTC Therapeutics

PhasePhase 3
Planned participants200
Who can joinAll sexes, N/A to no upper limit
Healthy volunteersNo

Sponsor, CRO or site team? See where this study is running → — listed sites, countries and recruiting context. Everything below is written for patients and caregivers.

About this study

The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.

This description comes directly from the study's public registry record.

Talk to the study team

No contact information is available for this study as per the public registry record.

Always discuss trial participation with your own doctor first.

Locations (46)

Children's Hospital ColoradoAurora, Colorado, United States
UF College of Medicine, Department of Pediatrics Division of Genetics and MetabolismGainesville, Florida, United States
Indiana University School of Medicine Department of Medical and Molecular GeneticsIndianapolis, Indiana, United States
Boston Children's HospitalBoston, Massachusetts, United States
Icahn School of Medicine at Mount Sinai (ISMMS)New York, New York, United States
UPMC Children's Hospital of PittsburghPittsburgh, Pennsylvania, United States
University of UtahSalt Lake City, Utah, United States
The Queensland Children's HospitalSouth Brisbane, Brisbane, Australia
Westmead HospitalWestmead, New South Wales, Australia
PARC Clinical ResearchAdelaide, South Australia, Australia
Royal Children's HospitalParkville, Victoria, Australia
Hospital de clinicas de Porto AlegrePorto Alegre, Rio Grande do Sul, Brazil

+ 34 more locations — full list on the registry record.

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