Skip to main content
Update Location

My Location

Update your location to show providers, locations, and services closest to you.

Enter a zip code
Or
Select a campus/region
(352) 733-0111

PTC923-004 extension study

  • Status
    Accepting Candidates
  • Age
    N/A - N/A
  • Sexes
    All
  • Healthy Volunteers
    No
I'm interested
Share this study
Facebook
Twitter
LinkedIn

Objective

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.

Description

Eligible participants are:

Feeder participants: those who have completed a Phase 3 Ptc Therapeutics (Ptc) sponsored feeder study (including Study PTC923-MD-003-PKU).

Non-feeder controlled participants: those who have not completed a feeder study and have blood Phe levels

Details

Full study title A Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria
Protocol number OCR42332
ClinicalTrials.gov ID NCT05166161
Phase Phase 3

Eligibility

Inclusion Criteria:

  • Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by past medical history of at least 2 blood Phe measurements ≥600 μmol/L.

  • Women of childbearing potential must have a negative pregnancy test at screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for up to 90 days after the last dose of the study drug.

  • Males who are sexually active with women of childbearing potential who have not had a vasectomy must agree to use a barrier method of birth control during the study and for up to 90 days after the last dose of study drug. Males must also refrain from sperm donations during this time period.

  • Willing to continue current diet unchanged while participating in the study (unless specifically instructed to change diet during the study by the investigator).

Exclusion Criteria:

  • Inability to tolerate oral medication.

  • A female who is pregnant or breastfeeding, or considering pregnancy.

  • Serious neuropsychiatric illness (for example, major depression) not currently under medical control, that in the opinion of the investigator or sponsor, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.

  • Past medical history and/or evidence of renal impairment and/or condition including moderate/severe renal insufficiency (glomerular filtration rate [GFR]

Lead researcher

  • Roberto T Zori, MD
    Clinical Cytogeneticist (Genetic Diagnostic Specialist)
    Roberto T Zori

Participate in a study

Here are some general steps to consider when participating in a research study:

  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.