A Phase 3 Extension Study of PTC923 in Subjects with Phenylketonuria

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 200

Inclusion Criteria

1. Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children
and/or subjects that are mentally impaired secondary to disease]) with parental/legal
guardian consent
2. Completed a PTC-sponsored Phase 3 PKU clinical study
3. Women of childbearing potential, as defined in (CTFG 2020), must have a negative pregnancy test at study entry and agree to abstinence or the use of at least one highly effective form of contraception (with a failure rate of <1% per year when used consistently and correctly):
• Combined (estrogen and progestogen containing) hormonal contraception associated
with inhibition of ovulation:
- Oral
- Intravaginal
- Transdermal
• Progestogen-only hormonal contraception associated with inhibition of ovulation:
- Oral
- Injectable
- Implantable
• Intrauterine device
• Intrauterine hormone-releasing system
• Vasectomized partner with confirmed azoospermia
Highly effective contraception or abstinence must be continued for the duration of the
study, and for up to 90 days after the last dose of the study drug.
All females will be considered of childbearing potential unless they are postmenopausal
(at least 12 months consecutive amenorrhea in the appropriate age group without other
known or suspected cause) or have been sterilized surgically (eg, hysterectomy, bilateral
tubal ligation, bilateral oophorectomy).
4. 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.
Males who are abstinent will not be required to use a contraceptive method unless they
become sexually active. Males who have undergone a vasectomy are not required to use a
contraceptive method if at least 16 weeks post procedure.
5. Willing and able to comply with the protocol and study procedure
6. Willing to continue current diet unchanged while participating in the study (unless
specifically instructed to change diet during the study by the investigator)
Are the trial subjects under 18? yes
Number of subjects for this age range: 80
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 100
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

1. The individual, in the opinion of the investigator, is unwilling or unable to adhere to the
requirements of the study
2. Inability to tolerate oral medication
3. A female who is pregnant or breastfeeding, or considering pregnancy
4. Serious neuropsychiatric illness (eg, major depression) not currently under medical
control, that in the opinion of the investigator or sponsor, would interfere with the
subject’s ability to participate in the study or increase the risk of participation for that
subject
5. Past medical history and/or evidence of renal impairment and/or condition including
moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60 mL/min)
as estimated most recently during qualifying participation in a feeder study) and/or under care of a nephrologist
6. Any other condition that in the opinion of the investigator or sponsor, would interfere
with the subject’s ability to participate in the study or increase the risk of participation for
that subject
7. Requirement for concomitant treatment with any drug known to inhibit folate synthesis
(eg, methotrexate)
8. Concomitant treatment with BH4 supplementation (eg, sapropterin dihydrochloride,
KUVAN) or pegvaliase-pqpz (PALYNZIQ)

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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