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

  • STATUS
    Recruiting
  • End date
    Feb 7, 2025
  • participants needed
    200
  • sponsor
    PTC Therapeutics
Updated on 7 October 2022
total protein
phenylalanine

Summary

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

All participants who complete a PTC-sponsored Phase 3 phenylketonuria study will be eligible for this Phase 3 open-label extension study, PTC923-MD-004-PKU.

Details
Condition Phenylketonuria
Treatment PTC923
Clinical Study IdentifierNCT05166161
SponsorPTC Therapeutics
Last Modified on7 October 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Completed a PTC-sponsored Phase 3 PKU clinical study
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] <60 milliliters [mL]/minute [min] min as estimated most recently during qualifying participation in a feeder study) and/or under care of a nephrologist
Any other condition 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
Requirement for concomitant treatment with any drug known to inhibit folate synthesis (for example, methotrexate)
Concomitant treatment with tetrahydrobiopterin (BH4) supplementation (for example, sapropterin dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ)
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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