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临床试验/EUCTR2021-000497-28-DE
EUCTR2021-000497-28-DE进行中（未招募）1 期

Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria

PTC Therapeutics, Inc.0 个研究点目标入组 200 人开始时间: 2021年12月22日最近更新:
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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 clinical trial of medicinal product

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
  • •and/or subjects that are mentally impaired secondary to disease]) 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
  • •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:
  • •- 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).
  • •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: 200
  • •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
  • •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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