A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 56
- 试验地点
- 12
- 主要终点
- Incidence of adverse events
研究概览
简要总结
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo.
There will be a single-ascending dose part of the trial and a multiple-ascending dose part. In the single-ascending dose, participants will receive one dose of ATX-01 or placebo. In the multiple-ascending dose part, participants will receive three doses of ATX-01 or placebo.
ATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.
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
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 64 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with a documented clinical diagnosis of DM1 (CTG expansion of >150 repeats in DMPK gene measured in peripheral blood mononuclear cells)
- •Ambulatory, defined as able to complete a 10-meter walk/run test at screening without the use of assistive devices such as canes, walkers, or orthoses, except for ankle-foot orthoses
- •Presence for >3 seconds of grip myotonia as confirmed by a central reader
排除标准
- •Participants with congenital DM1
- •Medical Research Council Muscle Scale score of less than 4 on ankle dorsiflexion or significant tibialis anterior atrophy that prevents a muscle biopsy
- •Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening
研究组 & 干预措施
ATX-01
ATX-01 is a formulation of the anti-microRNA 23b (anti-miR-23b), known as X82108, a novel type of antisense oligonucleotide
干预措施: ATX-01 (Drug)
Placebo
Placebo to ATX-01
干预措施: Placebo (Drug)
结局指标
主要结局
Incidence of adverse events
时间窗: Up to 120 days
To evaluate the safety and tolerability of ATX-01 in adult participants with DM1
次要结局
- Incidence of clinically significant changes in laboratory assessments, electrocardiograms (ECGs), vital signs, suicidal ideation and behavior(Up to 120 days)
- Maximum observed plasma concentration (Cmax) of ATX-01(Up to 48 hours post-dose)
- Area under the plasma concentration-time curve (AUC) of ATX-01(Up to 48 hours post-dose)
- Video hand opening time(Change from baseline up to 120 days)
- Change from baseline in ankle dorsiflexion strength by quantitative myometry(Change from baseline up to 120 days)
- Change from baseline in Impact on Activities of Daily Living questionnaire item scores(Change from baseline up to 120 days)
研究者
Judith Walker
Scientific
Arthex Biotech S.L.