Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of MRM-3379 in Male Participants With Fragile X Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 16
- 主要终点
- Incidence and severity of treatment-emergent adverse events and Withdrawal due to adverse events and withdrawal due to adverse events
研究概览
简要总结
This study is a multicenter, double-blind, randomized, placebo-controlled study to assess the safety and tolerability of 3 doses of MRM-3379 in male participants with Fragile X Syndrome ages 16 to 45 (inclusive). In addition, a parallel cohort of participants ages 13 to <16 will receive open-label MRM-3379. All participants will participate for 12 weeks of treatment. The study is also intended as a proof-of-concept investigation to evaluate whether MRM-3379 can improve FXS symptoms
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 13 Years 至 45 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Willing and able to provide signed informed consent/assent. Where local regulations permit inclusion of participants deemed unable to provide informed consent, a legally authorized representative must provide informed consent on the participant's behalf, and the participant must provide assent if applicable.
- •Male, 13-45 years of age (inclusive)
- •Weight ≥30 kg and BMI between 18 and 36 kg/m2 (inclusive) at the screening visit
- •Diagnosis of FXS with a molecular genetic ≥200 CGG repetitions .
- •Able to perform the PVT and ORRT of the NIH-TCB
- •Have a consistent caregiver(s) who is willing and able to be present regularly and reliably with the participant
- •Able to swallow tablets or capsules
排除标准
- •History of or current medical condition other than FXS and related issues that would place the participant at higher risk from study participation
研究组 & 干预措施
Arm 2: Middle dose of MRM-3379
Participants 16-45 years of age randomized to this arm will receive the middle dose of MRM-3379
干预措施: Middle Dose of MRM-3379 (Drug)
Arm 3: High dose of MRM-3379
Participants 16-45 years of age randomized to this arm will receive the high dose of MRM-3379
干预措施: High dose of MRM-3379 (Drug)
Arm 1: Low Dose of MRM-3379
Participants 16-45 years of age randomized to this arm will receive the low dose of MRM-3379
干预措施: Low dose of MRM-3379 (Drug)
Arm 4 :Placebo
Participants 16-45 years of age randomized to this arm will receive Placebo
干预措施: Placebo (Drug)
Arm 5: Low dose of MRM-3379 Open-Label
Participants 13 to < 16 years of age will receive the low dose of MRM-3370 Open-Label
干预措施: Low dose of MRM-3379 Open-Label (Drug)
结局指标
主要结局
Incidence and severity of treatment-emergent adverse events and Withdrawal due to adverse events and withdrawal due to adverse events
时间窗: Baseline to Week 12
次要结局
未报告次要终点