An 8-Week, Multicenter, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of Cariprazine in the Treatment of Pediatric Subjects (5 to 17 Years of Age) With Autism Spectrum Disorder
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 161
- 试验地点
- 36
- 主要终点
- Change from Baseline in Aberrant Behavior Checklist, 2nd edition - Community Version - Irritability (ABC-I) Subscale Score
研究概览
简要总结
Autism spectrum disorder (ASD) consists of deficits in social, communication, and cognitive development, repetitive and stereotypic behaviors. Many ASD patients show notably high levels of irritability, including verbal and physical aggression, self injury, and/or property destruction. Autistic infants tend to avoid eye contact and show little interest in others. This study will assess how safe and effective cariprazine is in treating pediatric participants (5 to 17 years of age) with ASD. Adverse events and change in disease activity will be assessed.
Cariprazine is an investigational drug being developed for the treatment of irritability due to ASD. This study is double-blinded means that neither the participants nor the study doctors will know who will be given cariprazine and who will be given placebo (does not contain treatment drug). Study doctors put the participants in 1 of the 2 groups, called treatment arms. Each group receives a different treatment. There is a 1 in 2 chance that participants will be assigned to placebo. Approximately 152 participants diagnosed with ASD will be enrolled in approximately 40 sites globally.
Participants will receive oral capsules or oral solution of cariprazine or placebo once daily for 8-weeks and will undergo a 4-week safety follow-up period.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, checking for side effects and completing questionnaires.
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)
入排标准
- 年龄范围
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants at the time of screening must have a Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) diagnosis of ASD, confirmed by Kiddie Schedule for Affective Disorders and Schizophrenia-Present and Lifetime (K-SADS-PL) administered at screening (Visit 1) by a trained clinician.
- •Participants must have an Aberrant Behavior Checklist, 2nd edition - Community Version - Irritability (ABC-I) subscale score ≥ 18 (Visits 1 and 2).
- •Participants must have a Clinical Global Impressions - Severity - Irritability (CGI-S Irritability) score ≥ 4 at screening (Visits 1 and 2).
排除标准
- •Participants with diagnosis of intellectual disability (Intelligence quotient < 25).
- •No history of major depressive disorder, bipolar disorder, schizophrenia, schizoaffective disorder, schizophreniform disorder, brief psychotic disorder, or psychotic disorder due to another medical condition, as excluded by administration of Kiddie Schedule for Affective Disorders and Schizophrenia-Present and Lifetime (K-SADS-PL).
- •History of ASD that is associated with Rett Disorder, Fragile-X Syndrome, or Childhood Disintegrative Disorder.
研究组 & 干预措施
Cariprazine
Participants will receive age-and weight dependent flexible doses of cariprazine once daily for 8-weeks.
干预措施: Cariprazine (Drug)
Placebo
Participants will receive placebo once daily for 8-weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change from Baseline in Aberrant Behavior Checklist, 2nd edition - Community Version - Irritability (ABC-I) Subscale Score
时间窗: Baseline (Week 0) to Week 8
The Aberrant Behavior Checklist (ABC) - Community version is a 58-item, caregiver-rated scale designed to measure inappropriate and maladaptive behavior of people with developmental disabilities (including intellectual disability and ASD). The ABC has 5 subscales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactivity and Inappropriate Speech). The caregiver rates the child's behavior from 0=not at all a problem to 3= the problem is severe in degree. The range for Irritability is 0 to 45. Higher scores indicate greater severity.
次要结局
- Percentage of Participants with responder status of "Very Much Improved" or "Much Improved" on the Clinical Global Impression- Change Irritability (CGI-C Irritability) Scale(Week 8)
- Change from Baseline in Parent-Rated Anxiety Scale for Youth with Autism Spectrum Disorder (PRAS-ASD) Total Score(Baseline (Week 0) to Week 8)
- Change from Baseline in Caregiver Strain Questionnaire Short Form 7-Item (CGSQ SF-7) total score(Baseline (Week 0) to Week 8)