EUCTR2021-000497-28-DK进行中（未招募）1 期
Phase 3 Open-Label Study of PTC923 (SEPIAPTERIN) in Phenylketonuria
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 200
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
- 性别
- All
入选标准
- •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 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:
- •- 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 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).
- •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 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.
- •6. Willing and able to comply with the protocol and study procedure
- •7. 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
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. 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
- •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)
- •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
- •16. Major surgery within the prior 90 days of screening
- •17. Unwillingness to washout from BH4 supplementation (eg, sapropterin dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ)
研究者
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