EUCTR2020-004805-30-IT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) - na
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Pharnext SA
- 入组人数
- 350
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
- •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) Stable dose of prescribed psychoactive drugs (e.g. antidepressants, stimulants, tranquilizers, anti-epileptics) for at least 4 weeks prior to randomization, which is not planned to be changed
- •7) Stable dose of prescribed or 'over-the-counter' (OTC) analgesic medications (e.g. paracetamol/acetaminophen, nonsteroidal antiinflammatory drugs [NSAIDs]) 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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 271
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 23
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 (e.g. diabetes, alcohol, vascular, autoimmune, neoplastic, neurodegenerative, HIV, etc.)
- •6) Subjects who have had any surgery or have a concomitant disorder (e.g. severe arthrosis) that reduces the mobility of the ankle making it, in the opinion of the investigator, difficult to assess the efficacy of the treatment.
- •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 or may preclude successful participation or completion of the study
- •9) Known hypersensitivity or intolerance to baclofen, naltrexone, or sorbitol
- •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 Beck Depression Inventory (BDI-II) score = 20
- •16) Currently lactating, pregnant, or planning on becoming pregnant during the study
- •17) Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels greater than 2 times the upper limit of normal (ULN)
- •18) Significant renal impairment as determined by Glomerular Filtration Rate (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.
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