A Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Investigate the Efficacy, Safety, and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 168
- 试验地点
- 58
- 主要终点
- Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score
研究概览
简要总结
ML-004-002 is a multi-center, randomized, double-blind, parallel-group, placebo-controlled study that will enroll approximately 150 adolescent and adult subjects with ASD. The primary objective is to evaluate the efficacy of ML-004 compared with placebo in the improvement of social communication deficits in subjects with ASD.
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
Double-blind
入排标准
- 年龄范围
- 12 Years 至 45 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 12 to 45 at screening
- •Has a designated care/study partner who can reliably report on symptoms
- •Has a diagnosis of Autism Spectrum Disorder (ASD)
- •Has a body mass index (BMI) 18 through 34 kg/m², inclusive
- •Full scale IQ (or equivalent) ≥55 score, with adequate verbal fluency, as determined by the Principal Investigator.
- •Psychoactive medications and adjunctive therapies are stable for 4 weeks prior to screening
- •Must be able to swallow study medication
排除标准
- •Has Rett syndrome or Child Disintegrative Disorder
- •Has participated in any other study and received any other investigational medication (other than COVID-19 vaccination) or device within 60 days prior to screening
- •History of epilepsy without current adequate control, or any seizure in the 6 months preceding screening
- •History of suicidal ideation or behavior in the past 12 months, or a positive response to C-SSRS questions 4 and/or 5
- •Systolic blood pressure ≥140 mmHg (if adult) or >135 mmHg (if adolescent), or diastolic blood pressure ≥90 (if adult) or >85 mmHg (if adolescent), or a clinical history of uncontrolled or severe hypertension
- •If female, is pregnant or lactating
研究组 & 干预措施
ML-004 (IR)/(ER) tablet
ML-004 is a bilayer immediate-release (IR)/extended-release (ER, gastroretentive) oral tablet formulation. ML-004 is taken orally once daily and provided as a tablet in two dose strengths of 12 mg (3 mg IR/9 mg ER) and 24 mg tablet (6 mg IR/18 mg ER). Dose levels for this study are 12 mg (provided as one 12 mg tablet), 24 mg (one 24 mg tablet), 48 mg (two 24 mg tablets), and 72 mg (three 24 mg tablets).
干预措施: ML-004 (IR)/(ER) tablet (Drug)
ML-004 Placebo
Matched placebo oral tablets will consist of same excipients as the investigational drug but without any ML-004. Placebo taken orally once daily to match tablet number of ML-004 dose levels for 12 mg (1 tablet), 24 mg (1 tablet), 48 mg (2 tablets), and 72 mg (3 tablets).
干预措施: ML-004 Placebo (Drug)
结局指标
主要结局
Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score
时间窗: Baseline up to Day 110
Change from baseline in the ABI-Social Communication Domain Score will be reported. The ABI is a 62-item questionnaire for reporting the behaviors of subjects (ages: 3 years-adulthood) diagnosed with ASD. The tool is suitable for completion by parents or care/study partners of people with ASD. Each item assesses either quality (from not at all to without help) or frequency (never to very often) of a particular behavior. The Social Communication domain score is the sum of the scores in the social communication domain divided by the number of items in the domain.
次要结局
- Change from Baseline in Clinician Global Impression of Improvement (CGI-I)(Day 110)
- Change from Baseline in Autism Behavior Inventory-Clinician (ABI-C) Score(Baseline up to Day 110)
- Change from Baseline in Aberrant Behavior Checklist 2-Irritability (ABC-I) Subscale Score(Baseline up to Day 110)
- Change from baseline in the Clinician Global Impression of Severity (CGI-S) Score(Baseline up to Day 110)
- Change from baseline in the ABI Self-regulation Domain Score(Baseline up to Day 110)
- Change from baseline in the ABI-Short Form (ABI-S) Score(Baseline up to Day 110)
- Change from baseline in the ABC-Social Withdrawal (ABC-SW) Subscale Score(Baseline up to Day 110)
- Change from baseline in the Social Responsiveness Scale 2 (SRS-2) Score(Baseline up to Day 110)
- Change from baseline in the Vineland-3 (Domain Level Version) Score: total of Communication, Socialization, and Maladaptive behavior domains(Baseline up to Day 110)
- Change from baseline in the ABI Repetitive/Restrictive Behavior Domain Score(Baseline up to Day 110)
- Change from baseline in the ABI Mood and Anxiety Domain Score(Baseline up to Day 110)
- Change from baseline in the ABI Challenging Behavior Domain Score(Baseline up to Day 110)