Safety, Tolerability, and Efficacy of SRX246, a Vasopressin 1a Receptor Antagonist, in Aggressive Participants With Fragile X Syndrome
概览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- Azevan Pharmaceuticals
- 入组人数
- 80
- 试验地点
- 3
- 主要终点
- Aberrant Behavior Checklist
概览
简要总结
The goal of this clinical trial is to learn if drug SRX246 works to treat irritability, agitation, aggression and self-injury (IAAS) behaviors in adult males with Fragile X Syndrome (FXS). It will also learn about the safety of drug SRX246. The main questions it aims to answer are:
- Does drug SRX246 lower the number of times participants experience IAAS behaviors?
- What medical problems do participants have when taking drug SRX246? Researchers will compare drug SRX246 to a placebo (a look-alike substance that contains no drug) to see if drug SRX246 works to treat IAAS behaviors.
Participants will:
- Take drug SRX246 or a placebo every day for up to 8 months
- Have weekly checkups by phone or video to answer study questions
- Have periodically scheduled home visits by nurses to conduct medical check-ups and tests
- Keep a diary of their symptoms
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 45 Years（Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Biological male, age 18 to 45 years inclusive
- •Molecular genetic confirmation of the full Fragile X messenger ribonucleoprotein mutation (≥200 CGG repetitions)
- •IAAS symptoms leading to a CGI-S score for Disruptive Behavior of 5 or more
- •Reported history of aggression based on the CMAI-Agg subscale (average score \>22) and an average associated disruptiveness scale of at least
- •Stable medication doses for the last month
- •Has a caregiver who lives with or spends extensive time with the participant most days and can report behavioral symptoms
- •Must be able to swallow study drug capsules whole
排除标准
- •Medical condition contraindicating study participation
- •SGPT and SGOT values 2 or more times the upper limit of normal
- •History of psychosis
- •Suicidal behavior
研究组 & 干预措施
SRX246
SRX246 will be provided as 120mg capsule to be taken orally, twice per day, once in the morning and once in the evening.
干预措施: SRX246 (Drug)
Placebo
Placebo will be provided as a matching capsule to be taken orally, twice per day, once in the morning and once in the evening.
干预措施: Placebo (Drug)
结局指标
主要结局
Aberrant Behavior Checklist
时间窗: Over an 8 month period
The Aberrant Behavior Checklist-Community Edition (ABC-C) Irritability subscale - Caregiver rated and then scored using the using the FXS-specific factoring system (ABCFX). Responses will be rated using a scale ranging from 0=not a problem to 3=severe problem.
次要结局
未报告次要终点