A Randomized, Placebo-controlled Phase 2 Study With Open-Label and Double-Blind Portions to Evaluate the Safety of L1-79 in Adolescent and Young Adult Males With Autism
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 42
- 试验地点
- 2
- 主要终点
- Adverse Events
研究概览
简要总结
This is a five-arm designed to assess the safety of L1-79 that incorporates 15 prospectively randomized, placebo controlled patients and 5 open label patients at either 100 tid (three times daily) or 200 tid dosing for 28 days. The open label patients will be assessed for the purpose of understanding PK/PD and to determine if there are any EKG changes associated with the administration of L1-79. Additional safety information will be provided by the 30 patients randomized 2:1 active:placebo.
详细描述
Protocol Number: HT 02-121
Protocol Title: Phase 2 Safety Study of L1-79 for the Treatment of Autism Study Phase: 2
The first cohort of 20 patients to be enrolled will all receive L1-79 100 mg t.i.d., and will be comprised of 3 groups of patients. The first group of patients to receive 100 mg will differ from the others in that they will get blood samples drawn for PK analysis and EKGs will be taken. The safety and PK data from this group will be submitted for FDA review and acceptance before the 200 mg t.i.d. cohort will be enrolled. The remaining 15 patients in this cohort will be randomized to receive either L1-79 100 mg t.i.d. or placebo on a 2:1 basis (2 L1-79 patients for each placebo patient). While the FDA is reviewing the data from the first 5 patients all 100 mg t.i.d. patients will continue to be treated.
The second cohort is identical to the first. The initial 5 patients to be enrolled will differ from the others in that they will get blood samples drawn for PK analysis and EKGs will be taken. The remaining 15 patients in this cohort will be randomized to receive either L1-79 200 mg t.i.d. or placebo on a 2:1 active:placebo.
Sample Size: N=40
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
- 12 Years 至 21 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males who are not sexually active
- •12 and 21 years of age
- •Signed informed consent
- •Normal clinical laboratory values
- •DSM-5 compliant diagnosis of autism spectrum disorder, confirmed by the Autistic Diagnosis Interview Review (ADIR), and by the Autism Diagnosis Observation Schedule (ADOS) score consistent with a diagnosis of autism
- •No more than one concomitant medication for the treatment of autism, on a stable for at least 2 weeks prior to enrollment and no planned changes in psychosocial interventions during the trial
- •No medications for any other pathology
排除标准
- •Any co-morbidities, including Fragile-X syndrome, epilepsy, Retts syndrome, ADHD, or other disease or syndrome aside from autism that requires treatment
- •Any other psychiatric disorder, or out of range lab values
- •DSM-5 diagnosis of schizophrenia, schizoaffective disorder, alcohol use disorder
- •Active medical problems: unstable seizures (>2 in past month)
- •Concomitant physical illness
研究组 & 干预措施
100 mg open
open-label lead-in 100 mg L1-79 t.i.d.
干预措施: L1-79 (Drug)
100 mg blinded
blinded and randomized 100 mg L1-79 t.i.d.
干预措施: L1-79 (Drug)
200 mg open
open-label lead-in 200 mg L1-79 t.i.d.
干预措施: L1-79 (Drug)
200 mg blinded
blinded and randomized 200 mg L1-79 t.i.d.
干预措施: L1-79 (Drug)
Placebo
placebo t.i.d.
干预措施: Placebo (Drug)
结局指标
主要结局
Adverse Events
时间窗: 4 weeks
Number of Participants with On-treatment Adverse Events. To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
次要结局
- Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score(Week 0 and Week 4)
- Change From Baseline in Clinical Global Impression Scale (CGI)(Week 0 and Week 4)
- Change From Baseline in Vineland Adaptive Behavior Scale - 2nd Edition Communication Standard Score(Week 0 and Week 4)
- Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C)(Week 0 to Week 4)
- Change From Baseline in Vineland Adaptive Behavior Scale - 2nd Edition Socialization Standard Score(Day 0 and Week 4)
- Change From Baseline in the Autism Diagnostic Observation Schedule 2nd Edition (ADOS-2) Overall Total Score(Day 0 and Week 4)
- Change From Baseline in the Repetitive Behavior Scale - Revised (RBS-R) Total Score(Week 0 and Week 4)