2025-523738-21-00招募中2 期
Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of MRM-3379 in Male Participants with Fragile X Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 15
- 试验地点
- 3
- 主要终点
- To evaluate the safety and tolerability of MRM-3379 in male participants with FXS on the bases of the following endpoints: -Incidence and severity of TEAEs; - Withdrawal due to AEs.
研究概览
简要总结
To evaluate the safety and tolerability of MRM-3379 in male participants with FXS on the basis of the following endpoints:
- Incidence and severity of TEAEs
- Withdrawal due to AEs
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 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 ≥18 and <36 kg/m2 (inclusive)
- •Diagnosis of FXS with a molecular genetic ≥200 CGG repetitions.
- •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
Placebo for MRM-3379
Placebo
干预措施: Placebo for MRM-3379 (Drug)
MRM-3379
Test
干预措施: MRM-3379 (Drug)
结局指标
主要结局
To evaluate the safety and tolerability of MRM-3379 in male participants with FXS on the bases of the following endpoints: -Incidence and severity of TEAEs; - Withdrawal due to AEs.
To evaluate the safety and tolerability of MRM-3379 in male participants with FXS on the bases of the following endpoints: -Incidence and severity of TEAEs; - Withdrawal due to AEs.
次要结局
- To evaluate the preliminary efficacy of MRM-3379 in male participants with FXS on the basis of the following endpoints: - Key secondary endpoint: Change from baseline on the NIH-TCB Crystallized Cognition Composite (CCC): Composed from the Picture Vocabulary Test (PVT) and Oral Reading Recognition Test (ORRT) change sensitive scores; - Other secondary endpoint: Clinician Global Impression-Change (CGI-C) score
- To describe the PK properties of MRM-3379 on the basis of the following endpoint: Steady-state MRM-3379 and metabolite plasma concentrations
研究者
Mirum Pharmaceuticals, Inc.
Scientific
Mirum Pharmaceuticals Inc.
研究点 (3)
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