NCT04762589已完成2 期
A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Amyotrophic Lateral Sclerosis
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Biojiva LLC
- 入组人数
- 43
- 试验地点
- 4
- 主要终点
- Change from baseline in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks
研究概览
简要总结
RT001-014 is a Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects with Amyotrophic Lateral Sclerosis
详细描述
Forty subjects at 4 EU sites will undergo baseline examination, and then be randomized 1:1 to receive either RT001 or placebo. Repeat visits will occur every 2 months. The final visit will be at 6 months.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Placebo-controlled, double-blind
入排标准
- 年龄范围
- 20 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subject with age 20 years to 75 years at the time of signed consent
- •Patients who are defined as "definite ALS," "probable ALS" or "probable-laboratory-supported ALS," met diagnostic criteria revised EL Escorial for Airlie House.
- •ALSFRS-R > 20
- •Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life
- •Patients of less than 3 years after the onset of ALS
- •Subject has an identified, reliable, study partner (e.g., caregiver, family member, social worker, or friend)
- •If patients are duly capable of study consent but are unable to sign by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation
排除标准
- •Received treatment with other experimental therapies within the last 30 days prior to the first dose
- •Previously received treatment with RT001
- •Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed)
- •SVC < 70 at screening
- •Subject has a feeding tube or the need for a feeding tube is anticipated within the first 24 weeks after enrollment
- •Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period
- •Evidence of any clinically significant neurological disorder other than ALS
- •The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to DSM-V or ICD-10 criteria
- •The subject has a significant pulmonary disorder not attributed to ALS or who require treatments that might complicate the evaluation of the effect of ALS on respiratory function
- •Subject has had a significant illness or infection requiring medical intervention in the past 30 days
- •Female who is breastfeeding or has a positive pregnancy test
- •Male participant or female participant of childbearing potential, who is sexually active and unwilling/unable to use a medically acceptable and effective double barrier birth control method throughout the study
- •Unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to return for visits as scheduled
- •History, within the last 2 years, of alcohol abuse or physical opioid dependence
研究组 & 干预措施
RT001
Experimental
RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.
干预措施: RT001 (Drug)
Placebo
Placebo Comparator
Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks
时间窗: 24 weeks
Change from baseline in ALSFRS-R will be compared for the RT001 treated group vs placebo
次要结局
- Frequency, severity and relationship to study drug of AEs and SAEs(24 weeks)
- Change from baseline in SVC(24 weeks)
- Composite of Death or a Specified State of Disease Progression(24 weeks)
- Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40)(24 weeks)
研究者
研究点 (4)
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