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Be Part of Research - Trial Details - A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
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A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Not Recruiting

Open to: ALL

Age: All

London Birmingham Birmingham

Medical Conditions

Phenylketonuria

This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the 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.

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

Feb 2022 Jun 2026

Publications

"van Spronsen F, Peters H, Margvelashvili L, Agladze D, Schwartz IVD, Gizewska M, Hamazaki T, Guilder L, MacDonald A, Vijay S, Inwood A, Minami M, Fjellbirkeland O, Lund A, Lah M, Thomas JA, Longo N, Kiykim E, Ishige M, Burlina A, Belanger-Quintana A, Rutsch F, Opladen T, Mungan H, Ezgu F, Bratkovic D, Chakrapani A, Ito T, Guimas A, Zori R, Tchan M, Sacharow S, Oliveira A, Janeiro P, Guerra IC, Plana JC, Yildiz Y, Canda E, Khan A, Vockley J, Breilyn MS, Manti F, Larkin A, Hughes C, Liu E, Gao L, Ingalls K, Smith N, Muntau AC. Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study. Genet Med. 2026 Jan 12;28(4):101683. doi: 10.1016/j.gim.2026.101683. Online ahead of print."; "41537382"

INTERVENTIONAL

Intervention Type : DRUG
Intervention Description : PTC923 powder for oral use will be suspended in water or apple juice prior to administration.

Intervention Arm Group : PTC923;



You can take part if:



You may not be able to take part if:


This is in the inclusion criteria above


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • Great Ormond Street Hospital
    London
    WC1N 3JH
  • Birmingham Children's Hospital NHS Foundation Trust
    Birmingham
    B4 6NH
  • UNIVERSITY HOSPITALS BIRMINGHAM NHS Foundation Trust
    Birmingham


The study is sponsored by PTC Therapeutics



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Read full details for Trial ID: NCT05166161
Last updated 05 February 2026

This page is to help you find out about a research study and if you may be able to take part

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