NCT07140614招募中1 期
A First-in-human, Multi-center, Randomized, Participant- and Investigator-blinded, Placebo Controlled, Single Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With Charcot-Marie Tooth Type 1A (CMT1A).
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 28
- 试验地点
- 5
- 主要终点
- Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provide written informed consent before any assessment is performed.
- •Be male or female and 18 to 60 (inclusive) years of age at the time of screening.
- •Participant must have a clinical diagnosis of Charcot-Marie-Tooth Disease Type 1A (CMT1A) including verified documentation of genetic testing showing duplication of the PMP22 gene by an accredited/certified laboratory (according to local regulations)
- •Have detectable upper extremity nerve conduction velocities (sensory and/or motor) in at least one extremity at screening.
- •CMTNSv2R score >2 and ≤20 in at least one assessment, confirmed either at the baseline visit or documented in the medical record within the 6 months prior to baseline.
- •Muscle weakness evidenced by foot dorsiflexion MRC grade <5 in at least one limb, confirmed either at screening assessment or documented in the medical record within the 6 months prior to screening.
排除标准
- •Unable to communicate well with the investigator, to understand and comply with the visits and procedures of the study.
- •History of cardiac, renal, liver, hematological, immune system disorders.
- •Pregnant/nursing female participants. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for the duration of the follow-up period. Sexually active males unless they use a condom during intercourse.
- •Inability or unwillingness to provide serial skin biopsy samples.
- •Inability or unwillingness to undergo repeated venipuncture or in the opinion of the investigator, participant would be at an increased risk for adverse events related to these procedures.
- •Use of any drug intended to modify the course of CMT1A within 6 months from screening, including but not limited to: PTX-3003 (baclofen, sorbitol, and naltrexone in combination).
- •History of compression neuropathy within the last 6 months from screening and/or other conditions that can cause peripheral neuropathy, including but not limited to diabetes, chronic alcoholism, exposure to neurotoxic medications, exposure to environmental neurotoxins, traumatic nerve injury, thyroid disease, or infections.
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
EDK060
Experimental
Doses A; B; C; D
干预措施: EDK060, dose A (Drug)
EDK060
Experimental
Doses A; B; C; D
干预措施: EDK060, dose B (Drug)
EDK060
Experimental
Doses A; B; C; D
干预措施: EDK060, dose D (Drug)
Placebo
Placebo Comparator
Matching placebo
干预措施: Placebo (Other)
EDK060
Experimental
Doses A; B; C; D
干预措施: EDK060, dose C (Drug)
结局指标
主要结局
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: From baseline up to day 140
Incidence of AEs and SAEs by treatment group, including changes in vital signs, laboratory measures, neurological examinations and electrophysiology measures qualifying and reported as AEs.
次要结局
- EDK060 Plasma PK parameters - Cmax(From baseline up to day 140)
- EDK060 Plasma PK parameters - Tmax(From baseline up to day 140)
- EDK060 Plasma PK parameters - AUClast(From baseline up to day 140)
研究者
研究点 (5)
Loading locations...
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