A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Aberrant Behavior Checklist - Social Withdrawal Subscale
研究概览
简要总结
The proposed project will pilot the use of IGF-1 as a novel treatment for core symptoms of autism. We will use a double-blind, placebo-controlled crossover trial design in five children with autism to evaluate the impact of IGF-1 treatment on autism-specific impairments in socialization, language, and repetitive behaviors. We expect to provide evidence for the safety and feasibility of IGF-1 in ameliorating social withdrawal in children with Autistic Disorder. Further, we expect to demonstrate that IGF-1 is associated with improvement on secondary outcomes of social impairment, language delay, and repetitive behavior, as well as on functional outcomes of global severity.
详细描述
IGF-1 is an FDA approved, commercially available compound that crosses the blood-brain barrier and has beneficial effects on synaptic development by promoting neuronal cell survival, synaptic maturation, and synaptic plasticity. IGF-1 is effective in reversing mouse and neuronal models of Rett syndrome and Phelan McDermid syndrome, both single gene causes of ASD and may therefore be effective in treating autism spectrum disorders (ASD) more broadly.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 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 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meet DSM-5 criteria for Autism Spectrum Disorder confirmed by the Autism Diagnostic Interview-Revised (ADI-R) and the Autism Diagnostic Observation Schedule- Generic (ADOS-G)
- •Children between the ages of 5-12 years of age
- •Language delay (lack of fluent phrase speech) reflected by use of ADOS Module 1 or 2
- •Must be on stable medication regimens for at least three months prior to enrollment, assuming the concomitant medication is safe for use with IGF-1
排除标准
- •Closed epiphyses
- •Active or suspected neoplasia
- •Intracranial hypertension
- •Hepatic insufficiency
- •Renal insufficiency
- •Cardiomegaly/valvulopathy
- •History of allergy to IGF-1
- •Patients with comorbid conditions deemed too medically compromised to tolerate the risk of experimental treatment with IGF-1
研究组 & 干预措施
Placebo
Placebo
干预措施: Placebo/saline (Drug)
IGF-1
Randomized, placebo-controlled, crossover format with 12 weeks in each treatment arm (IGF-1 and placebo), separated by a four-week wash-out phase.
Dose titration will be initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and preprandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well tolerated dose is established.
干预措施: IGF-1 (Drug)
结局指标
主要结局
Aberrant Behavior Checklist - Social Withdrawal Subscale
时间窗: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Aberrant Behavior Checklist - Social Withdrawal Subscale has16 items, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total Subscale scores range from 0 to 48. Higher score indicates poorer health outcomes.
次要结局
- The Clinical Global Impression - Severity Scale (CGI-S)(Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8)
- Caregiver Strain(Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8)
- Vineland Adaptive Behavior Composite Score(Baseline and Phase 1 Week 12)
- Children's Yale-Brown Obsessive Compulsive Disorder Scale for Pervasive Developmental Disorders(Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8)
- Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)(Week 8 of Phase 2)
- Repetitive Behavior(Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8)
研究者
Alexander Kolevzon
Clinical Director, Seaver Autism Center for Research and Treatment
Icahn School of Medicine at Mount Sinai