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

Biojiva LLC4 个研究点分布在 4 个国家目标入组 43 人2021年3月10日

适应症Amyotrophic Lateral Sclerosis

干预措施RT001 Placebo

概览

阶段: 2 期
干预措施: RT001
疾病 / 适应症: Amyotrophic Lateral Sclerosis
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
状态: 已完成
最后更新: 10个月前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验

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.

注册库

clinicaltrials.gov

开始日期

2021年3月10日

结束日期

2022年6月28日

最后更新

10个月前

研究类型

Interventional

研究设计

Parallel

性别

All

研究者

发起方

Biojiva LLC

责任方

Sponsor

入排标准

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

研究组 & 干预措施

RT001

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

Placebo

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

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)