NL-OMON53510尚未招募3 期
A phase 3 open-label study of PTC923 (Sepiapterin) in Phenylketonuria - PTC923-MD-004-PK
适应症
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 8
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
- •Individuals are eligible to participate if they meet all the following
- •inclusion criteria:
- •1. Informed consent and assent (if necessary, at the investigator*s discretion
- •[ie, for children]) with parental/legal guardian consent
- •2. Male or female subjects of any age.
- •3. Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by
- •past medical history of at least 2 blood Phe measurements >=600 µmol/L
- •4. Women of childbearing potential, as defined in (CTFG 2020), must have a
- •pregnancy test at study entry and agree to abstinence or the use of at least
- •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 ovulation:
- •* 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).
- •5. 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
- •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.
- •6. Willing and able to comply with the protocol and study procedures
- •7. Willing to continue current diet unchanged while participating in the study
- •(unless specifically instructed to change diet during the study by the
- •investigator)
排除标准
- •Individuals are not eligible to participate if they meet any of the following
- •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
- •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
- •5. Past medical history and/or evidence of renal impairment and/or condition
- •moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60
- •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
- •(eg, methotrexate)
- •8. 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:
- •9. 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
- •10. History of gastric surgery, including Roux-en-Y gastric bypass surgery or
- •an antrectomy with vagotomy, or gastrectomy
- •11. History of allergies or adverse reactions to synthetic BH4 or sepiapterin
- •12. Current participation in any other investigational drug study or use of any
- •investigational agent within 30 days prior to Screening
- •13. 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
- •14. 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.
- •15. 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
- 另有 3 项未显示
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