ISRCTN79102999进行中（未招募）3 期
A Phase III, randomized, crossover, open-label, active-controlled study of sepiapterin versus sapropterin in participants with phenylketonuria =2 years of age
PTC Therapeutics (United States)0 个研究点目标入组 142 人开始时间: 2024年1月29日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 142
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
- •Participants with any PAH mutation are permitted to screen and enroll into the study. However, participants with biochemically diagnosed classical PKU will be capped at 30% of the total study population. Biochemically diagnosed classical PKU includes those with =2 historical blood Phe concentrations =1200 µmol/L in their medical history. Furthermore, participants with initial newborn screen performed >3 days after birth demonstrating values =1200 µmol/L may have these values excluded from the =2 historical blood Phe concentration requirement. Genotyping will not be required for study eligibility; however, all participants will undergo genotyping unless documented in their medical history, and these data will be collected for analysis.
- •Participants are eligible to be included in the study only if all the following criteria apply:
- •1. Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children]) with parental/legal guardian consent
- •2. Male or female participants =2 years of age
- •3. Uncontrolled blood Phe level =360 µmol/L on current therapy at any time 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 participant’s medical history (inclusive of the Screening value)
- •4. Clinical diagnosis of PKU with hyperphenylalaninemia 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 negative pregnancy test at Screening 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):
- •5.1. Combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation:
- •5.1.1. Oral
- •5.1.2. Intravaginal
- •5.1.3. Transdermal
- •5.2. Progestogen-only hormonal contraception associated with inhibition of ovulation:
- •5.2.1. Oral
- •5.2.2. Injectable
- •5.2.3. Implantable
- •5.3. Intrauterine device
- •5.4. Intrauterine hormone-releasing system
- •5.5. Bilateral tubal occlusion
- •5.6. 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).
- •6. 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 the 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.
- •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 bowel disease, chronic gastritis, 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 hematology panels should fall within the limits of the normal laboratory reference range, unless 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 medical control, that in the opinion of the investigator or sponsor would interfere with the participant’s ability to participate in the study or increase the risk of participation for that participant
- •10. Past medical history and/or evidence of renal impairment and/or condition including moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60 mL/min) and/or under care of a nephrologist
- •11. Any abnormal physical examination and/or laboratory findings indicative of signs or symptoms of renal disease, including calculated GFR <60 mL/min/1.73m2
- •In participants =18 years of age, the Modification of Diet in Renal Disease Equation should be used to determine GFR.
- •In participants <18 years, the Bedside Schwartz Equation should be used to determine GFR.
- •12. Requirement for concomitant treatment with levodopa or with any drug known to inhibit folate synthesis (eg, methotrexate)
- •13. Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase, recessive guanosine triphosphate (GTP) cyclohydrolase I, sepiapterin reductase, quinoid dihydropteridine reductase, or pterin 4-alpha-carbinolamine dehydratase genes
- •14. Major surgery within the prior 90 days of Screening
- •15. Unwillingness to washout from BH4 supplementation (eg, sapropterin dihydrochloride, KUVAN)
- •16. Use of pegvaliase-pqpz (PALYNZIQ) concurrently or within the 60 days prior to Screening
- •17. Greater than 20% variance in dietary Phe consumption as measured by mandatory weekly 3-day diet record collection for 4 consecutive weeks (Dietary Observation Period during Screening)
研究者
相似试验
进行中（未招募）
1 期
Testing a new targeted therapy trastuzumab deruxtecan (DS-8201a) against ado-trastuzumab emtansine (T-DM1) for patients with advanced stage breast cancer previously treated with trastuzumab and taxane.MedDRA version: 23.0Level: PTClassification code: 10065430Term: HER2 positive breast cancer Class: 100000004864MedDRA version: 20.0Level: LLTClassification code: 10027475Term: Metastatic breast cancer Class: 10029104MedDRA version: 20.1Level: PTClassification code: 10055113Term: Breast cancer metastatic Class: 100000004864Metastatic breast cancerCTIS2024-511204-16-00Daiichi Sankyo Inc.240
进行中（未招募）
1 期
Testing a new targeted therapy (DS-8201a) against ado-trastuzumab emtansine (T-DM1) for patients with advanced-stage breast cancer previously treated with trastuzumab and taxane.nresectable/metastatic breast cancer with human epidermal growth factor receptor 2 (HER2)-positive expressionMedDRA version: 20.0Level: LLTClassification code 10027475Term: Metastatic breast cancerSystem Organ Class: 100000004864EUCTR2018-000222-61-FRDaiichi Sankyo Inc.524
招募中
3 期
Testing a new targeted therapy trastuzumab deruxtecan (DS-8201a) against ado-trastuzumab emtansine (T-DM1) for patients with advanced stage breast cancer previously treated with trastuzumab and taxane.Metastatic breast cancer2024-511204-16-00Daiichi Sankyo Inc.63
进行中（未招募）
1 期
Testing a new targeted therapy (DS-8201a) against ado-trastuzumab emtansine (T-DM1) for patients with advanced-stage breast cancer previously treated with trastuzumab and taxane.EUCTR2018-000222-61-ESDaiichi Sankyo Inc.500
进行中（未招募）
1 期
Testing a new targeted therapy (DS-8201a) against other targeted and chemotherapy (selected by your doctor) for patients with advanced-stage breast cancer.nresectable/metastatic breast cancer with human epidermal growth factor receptor 2 (HER2)-positive expressionEUCTR2018-000221-31-ITDaiichi Sankyo Inc.600