A phase 3 study of PTC923 in subjects with phenylketonuria - PTC923-MD-003-PK

PTC Therapeutics Inc.0 个研究点目标入组 5 人开始时间: 待定最近更新: 2024年4月15日

适应症

试验速览

阶段: 3 期
状态: 已完成
发起方: PTC Therapeutics Inc.
入组人数: 5

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional

入排标准

年龄范围: 0 至 99（—）

入选标准

•Informed consent and assent (if necessary, at the investigator*s discretion
•children and/or subjects who are mentally impaired secondary to disease]) with
•parental/legal guardian consent
•2. Male or female subjects of any age
•3. Uncontrolled blood Phe level >=360 µmol/L on current therapy anytime during
•Screening and uncontrolled blood Phe level >=360 µmol/L on current therapy when
•taking the average of the 3 most recent Phe levels from the subject*s medical
•(inclusive of the Screening value)
•4. Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by past
•medical history of at least 2 blood Phe measurements >=600 µmol/L
•5. Women of childbearing potential, as defined in (CTFG 2020), must have a
•pregnancy test at Screening and agree to abstinence or the use of at least one
•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:
•* Intravaginal
•* Transdermal
•Progestogen-only hormonal contraception associated with inhibition of
•* 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
•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).
•6. Males who are sexually active with women of childbearing potential who have
•a vasectomy must agree to use a barrier method of birth control during the
•for up to 90 days after the last dose of study drug. Males must also refrain
•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.
•7. Willing and able to comply with the protocol and study procedures
•8. Willing to continue current diet unchanged while participating in the study

排除标准

•1. The individual, in the opinion of the investigator, is unwilling or unable
•to adhere to
•the requirements of the study
•2. Gastrointestinal disease (such as irritable bowel syndrome, inflammatory
•disease, chronic gastritis, and peptic ulcer disease, etc.) that could affect
•the absorption
•of study drug
•3. History of gastric surgery, including Roux-en-Y gastric bypass surgery or an
•antrectomy with vagotomy, or gastrectomy
•4. Inability to tolerate oral medication
•5. History of allergies or adverse reactions to synthetic BH4 or sepiapterin
•6. Current participation in any other investigational drug study or use of any
•investigational agent within 30 days prior to Screening
•7. Any clinically significant laboratory abnormality as determined by the
•investigator. In
•general, each laboratory value from Screening and baseline chemistry and
•panels should fall within the limits of the normal laboratory reference range,
•deemed not clinically significant by the investigator
•8. A female who is pregnant or breastfeeding, or considering pregnancy
•9. Serious neuropsychiatric illness (eg, major depression) not currently under
•control, that in the opinion of the investigator or sponsor, would interfere
•subject*s ability to participate in the study or increase the risk of
•participation for that
•10. Past medical history and/or evidence of renal impairment and/or condition
•moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60
•and/or under care of a nephrologist
•11. Any abnormal physical examination and/or laboratory findings indicative of
•symptoms of renal disease, including calculated GFR <60 mL/min/1.73m2.
•In subjects >=18 years of age, the Modification of Diet in Renal Disease Equation
•should be used to determine GFR.
•In subjects <18 years, the Bedside Schwartz Equation should be used to determine
•12. Requirement for concomitant treatment with any drug known to inhibit folate
•(eg, methotrexate)
•13. Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic
•mutations in 6-pyruvoyltetrahydropterin synthase, recessive GTP cyclohydrolase
•sepiapterin reductase, quinoid dihydropteridine reductase, or
•pterin-4-alphacarbinolamine
•dehydratase genes
•14. Major surgery within the prior 90 days of screening
•15. Concomitant treatment with BH4 supplementation (eg, sapropterin
•dihydrochloride,
•KUVAN) or pegvaliase-pqpz (PALYNZIQ)
•16. Unwillingness to washout from BH4 supplementation (eg, sapropterin
•dihydrochloride,
•KUVAN) or pegvaliase-pqpz (PALYNZIQ)

研究者

发起方

PTC Therapeutics Inc.

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