EUCTR2021-000497-28-PT进行中（未招募）1 期
Phase 3 Open-Label Extension Study of PTC923 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
- •and/or subjects that are mentally impaired secondary to disease]) with parental/legal
- •guardian consent
- •2. Completed a PTC-sponsored Phase 3 PKU clinical study
- •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
- •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 sterilized surgically (eg, hysterectomy, bilateral
- •tubal ligation, 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: 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)
研究者
相似试验
进行中（未招募）
1 期
A Phase 3 Extension Study of PTC923 in Subjects with PhenylketonuriaMetabolic Disorders - PhenylketonuriaMedDRA version: 20.0Level: LLTClassification code 10034873Term: Phenylketonuria (PKU)System Organ Class: 100000004850EUCTR2021-000497-28-NLPTC Therapeutics, Inc.200
招募中
3 期
A PHASE 3 OPEN-LABEL STUDY OF PTC923 (SEPIAPTERIN) IN PHENYLKETONURIAPhenylketonuriaJPRN-jRCT2041230069KOBAYASHI Tomoyo15
进行中（未招募）
1 期
A Phase 3 Study of PTC923 (SEPIAPTERIN) in Subjects with PhenylketonuriaMetabolic Disorders - PhenylketonuriaMedDRA version: 20.0Level: LLTClassification code 10034873Term: Phenylketonuria (PKU)System Organ Class: 100000004850EUCTR2021-000497-28-DKPTC Therapeutics, Inc.200
招募中
1 期
A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in PhenylketonuriaMetabolic Disorders - PhenylketonuriaMedDRA version: 20.0Level: LLTClassification code: 10034873Term: Phenylketonuria (PKU) Class: 10010331CTIS2023-509229-31-00PTC Therapeutics Inc.200
进行中（未招募）
1 期
A Phase 3 Study of PTC923 (Sepiapterin) in Subjects with PhenylketonuriaMetabolic Disorders - PhenylketonuriaMedDRA version: 20.0Level: LLTClassification code 10034873Term: Phenylketonuria (PKU)System Organ Class: 100000004850EUCTR2021-000497-28-DEPTC Therapeutics, Inc.200