EUCTR2020-004805-30-BE进行中（未招募）1 期
A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A)
Pharnext SCA0 个研究点目标入组 387 人开始时间: 2021年3月4日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 387
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) Male and non-pregnant female subjects, aged 16 to 65 years with a genetically proven diagnosis of CMT1A Notes: a) A report of a genetic
- •test confirming PMP22 duplication and therefore a diagnosis of CMT1A
- •must be available in the subject's record at the clinical site. b) In the
- •absence of a report of a genetic test confirming PMP22 duplication in the
- •subject's medical record, a confirmatory genetic test must be conducted
- •via the central laboratory as part of Screening. c) In the exceptional
- •case wherein subject was randomized into the study without meeting (a)
- •or (b), an unscheduled confirmatory genetic test will be performed. In
- •the event of a negative genetic test result, the subject will be withdrawn
- •from the study
- •2) Able to provide written informed consent/assent and comply with study procedures
- •3) Mild-to-moderate severity assessed by CMTNS-v2 score >2 and =18
- •4) Muscle weakness in at least foot dorsiflexion on clinical assessment
- •5) Ulnar nerve motor conduction time of at least 15m/s
- •6) If taking prescribed psychoactive drug(s) (eg, antidepressants, stimulants, tranquilizers, anti-epileptics), should be on a stable dose for at least 4 weeks prior to randomization, which is not planned to be changed
- •7) If taking prescribed or ‘over-the-counter’ analgesic medication(s) (eg, paracetamol/acetaminophen, nonsteroidal anti-inflammatory drugs), should be on a stable dose for at least 2 weeks prior to randomization, which is not planned to be changed
- •8) If female, subject must be: (a) Surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation; or (b) Of childbearing potential and using a birth control method such as:
- •Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
- •o Intravaginal
- •o Transdermal
- •Progestogen-only hormonal contraception associated with inhibition of ovulation:
- •o Injectable
- •o Implantable
- •Intrauterine device (IUD)
- •Intrauterine hormone-releasing system (IUS)
- •Bilateral tubal occlusion
- •Vasectomized partner
- •Sexual abstinence;
- •or (c) Of non-childbearing potential (i.e., postmenopausal for at least 1 year)
- •9) If male, the subject must have had a vasectomy or must use a reliable method of birth control with their partner or total abstinence from sexual intercourse. The subject must agree to continue using their selected method of birth control with their sexual partner during the study and for 120 days after study completion.
- •For subjects consenting to enter the OLE Period, the following Inclusion
- •criteria will be confirmed/reassessed on Screening Day (SV6) of the OLE
- •1. Able to provide written informed consent/assent and comply with
- •study procedures.
- •2. If female, subject must be (a) surgically sterilized via hysterectomy,
- •bilateral oophorectomy, or bilateral tubal ligation; or (b) of childbearing
- •potential and using a birth control method such as:
- •Combined (estrogen and progestogen containing) hormonal
- •contraception
- •associated with inhibition of ovulation:
- •o Intravaginal
- •o Transdermal
- •Progestogen-only hormonal contraception associated with inhibition
- •of ovulation:
- •o Injectable
- •o Implantable
- •Bilateral tubal occlusion
- •Vasectomized partner
- •Sexual abstinence
- •or (c) of non-childbearing potential (ie, no menses for =12 consecutive
- 另有 2 项未显示
排除标准
- •1) Subjects previously enrolled in any PXT3003 study
- •2) Subjects living in the same household and enrolled in a PXT3003 study (due to potential lack of adequate storage for study material, risk of mixing treatments and potential unblinding)
- •3) CMT of any subtype other than 1A
- •4) ONLS score of 0
- •5) Known clinically significant motor or sensory abnormalities secondary to a different neurological cause.Note: subjects with diagnosis of
- •unilateral carpal tunnel syndrome at least 1 year prior to Screening Visit,
- •that is asymptomatic at the time of Screening Visit, will not be excluded
- •from participating in this study
- •6) Subjects who have had any surgery or have a concomitant disorder that reduces the mobility of the ankle making it, in the opinion of the investigator, difficult to assess the efficacy of the treatment. Note:
- •subjects with surgical repair of unilateral carpal tunnel syndrome will
- •not be excluded from participating in this study
- •7) Known peripheral neuropathy, myopathy, or neuromuscular disorder of any other kind
- •8) Any other clinically significant and/or uncontrolled medical condition that, in the opinion of the investigator, could be a confound, may increase subject’s risk, or may preclude successful participation or completion of the study
- •9) Known hypersensitivity or intolerance to PXT3003 (or matching placebo), including any of its active ingredients and/or any of its excipients 10) Concomitant treatments including but not limited to baclofen, naltrexone, sorbitol (pharmaceutical form), opioids, and potentially neurotoxic drugs such as amiodarone, chloroquine, and chemotherapeutics capable of inducing peripheral neuropathy. Subjects able to stop these medications at least 2 weeks before randomization and for the study duration may be included
- •11) History of porphyria
- •12) Diagnosis or history of substance use disorder by DSM-V criteria within the past 12 months
- •13) Medical or recreational use of marijuana in the 3 months prior to the Screening Visit
- •14) Active suicidality (e.g. any suicide attempts within the past 12 months or any current suicide intent, including a plan, as assessed by the Columbia Suicide Severity Rating Scale (C-SSRS) score of YES” on questions 4 or 5; and/or based on clinical evaluation by the investigator)
- •15) Currently active major depression, as determined by a BDI-II score = 20
- •16) Currently lactating, pregnant, or planning on becoming pregnant during the study
- •17) ALT or AST levels greater than 2 times the ULN
- •18) Significant renal impairment as determined by GFR of less than 50 mL/min
- •19) Subject has participated in an investigational drug or device study within 30 days prior to the Screening Visit or plans to participate in an investigational drug or device study during the course of this study
- •20) Subject is a dependent and/or relative of the Sponsor or investigator.
- •For subjects consenting to enter the OLE Period, the following criteria
- •will be confirmed/reassessed on SV6 of the OLE Period
- •1.Any clinically significant and/or uncontrolled medical condition that, in the opinion of the investigator, could be a confound factor, may increase
- •subject's risk, or may preclude successful participation or completion of
- •2.Concomitant treatments including but not limited to baclofen,
- •naltrexone, sorbitol (pharmaceutical form) other than PXT3003 taken in
- •the Double-blind Treatment Period of this study, opioids, potent CNS
- •depressants, and potentially neurotoxic drugs such as amiodarone,
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