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A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
EU CTIS ID: 2023-509229-31-00
What this study is testing
To evaluate the long-term safety of PTC923 in subjects with PKU. To evaluate changes from baseline in dietary Phe/protein consumption.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children]) with parental/legal guardian consent
- Male or female subjects of any age
- Clinical diagnosis of PKU with HPA documented by past medical history of at least 2 blood Phe measurements ≥600 μmol/L
- Women of childbearing potential, as defined in the Clinical Trial Facilitation Group guidance (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 • Bilateral tubal occlusion • 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 permanently sterilized surgically (eg, hysterectomy, bilateral salpingectomy, bilateral oophorectomy).
- 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 and able to comply with the protocol and study procedures.
You likely can't join if
- The individual, in the opinion of the investigator, is unwilling or unable to adhere to the requirements of the study
- Additional criteria for subjects who did not participate in a feeder study: History of gastric surgery, including Roux-en-Y gastric bypass surgery or an antrectomy with vagotomy, or gastrectomy
- Additional criteria for subjects who did not participate in a feeder study: History of allergies or adverse reactions to synthetic BH4 or sepiapterin
- Additional criteria for subjects who did not participate in a feeder study: Current participation in any other investigational drug study or use of any investigational agent within 30 days prior to Screening
- Additional criteria for subjects who did not participate in a feeder study: Any clinically significant laboratory abnormality as determined by the investigator. In general, each laboratory value from Screening and baseline chemistry and hematology panels should fall within the limits of the normal laboratory reference range, unless deemed not clinically significant by the investigator
- Additional criteria for subjects who did not participate in a feeder study: Any abnormal physical examination and/or laboratory findings indicative of signs or symptoms of renal disease, including calculated GFR <60 mL/min/1.73 m2. In subjects ≥18 years of age, the Modification of Diet in Renal Disease Equation should be used to determine GFR. In subjects <18 years of age, the Bedside Schwartz Equation should be used to determine GFR.
See the full eligibility criteria
Who can join
- Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children]) with parental/legal guardian consent
- Male or female subjects of any age
- Clinical diagnosis of PKU with HPA documented by past medical history of at least 2 blood Phe measurements ≥600 μmol/L
- Women of childbearing potential, as defined in the Clinical Trial Facilitation Group guidance (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 • Bilateral tubal occlusion • 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 permanently sterilized surgically (eg, hysterectomy, bilateral salpingectomy, bilateral oophorectomy).
- 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 and able to comply with the protocol and study procedures.
- Willing to continue current diet unchanged while participating in the study (unless specifically instructed to change diet during the study by the investigator).
- 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.
What rules you out
- The individual, in the opinion of the investigator, is unwilling or unable to adhere to the requirements of the study
- Additional criteria for subjects who did not participate in a feeder study: History of gastric surgery, including Roux-en-Y gastric bypass surgery or an antrectomy with vagotomy, or gastrectomy
- Additional criteria for subjects who did not participate in a feeder study: History of allergies or adverse reactions to synthetic BH4 or sepiapterin
- Additional criteria for subjects who did not participate in a feeder study: Current participation in any other investigational drug study or use of any investigational agent within 30 days prior to Screening
- Additional criteria for subjects who did not participate in a feeder study: Any clinically significant laboratory abnormality as determined by the investigator. In general, each laboratory value from Screening and baseline chemistry and hematology panels should fall within the limits of the normal laboratory reference range, unless deemed not clinically significant by the investigator
- Additional criteria for subjects who did not participate in a feeder study: Any abnormal physical examination and/or laboratory findings indicative of signs or symptoms of renal disease, including calculated GFR <60 mL/min/1.73 m2. In subjects ≥18 years of age, the Modification of Diet in Renal Disease Equation should be used to determine GFR. In subjects <18 years of age, the Bedside Schwartz Equation should be used to determine GFR.
- Additional criteria for subjects who did not participate in a feeder study: Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase, recessive GTP cyclohydrolase I, sepiapterin reductase, quinoid dihydropteridine reductase, or pterin-4-alpha-carbinolamine dehydratase genes
- Additional criteria for subjects who did not participate in a feeder study: Major surgery within the prior 90 days of screening
- Additional criteria for subjects who did not participate in a feeder study: Unwillingness to washout from BH4 supplementation (eg, sapropterin dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ)
- Inability to tolerate oral medication
- A female who is pregnant or breastfeeding, or considering pregnancy
- Serious neuropsychiatric illness (eg, major depression) not currently under medical control, that in the opinion of the investigator or PTC, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject
- 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
- Any other condition that in the opinion of the investigator or PTC, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject
- Requirement for concomitant treatment with any drug known to inhibit folate synthesis (eg, methotrexate)
- Concomitant treatment with BH4 supplementation (eg, sapropterin dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ)
- Additional criteria for subjects who did not participate in a feeder study: Gastrointestinal disease (such as irritable bowel syndrome, inflammatory bowel disease, chronic gastritis, and peptic ulcer disease, etc) that could affect the absorption of study drug
The study team makes the final eligibility decision.
Where it's taking place
- Australia
- Canada
- United States
- Mexico
- Georgia
- Turkey
- Brazil
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Australia; Canada; United States; Mexico; Georgia; Turkey and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.