A Pilot Dose Finding Study of Pioglitazone in Children With ASD
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 28
- 试验地点
- 1
- 主要终点
- Maximum tolerated dose to be used in the follow-up multisite randomized controlled trial
研究概览
简要总结
The investigators propose a pilot, single blind, placebo run-in, dose finding study of pioglitazone in children with autism with the ultimate goal of identifying appropriate dosing and outcome measures for a larger follow-up randomized placebo controlled clinical trial. The specific aims of this study are: 1) To examine the safety of pioglitazone in children with autism spectrum disorders (ASD) ages 5-12 years; 2) To identify appropriate outcome measures to be used in a follow-up multisite randomized control trial of pioglitazone in children with ASD; 3) To determine the maximum tolerated dose to be used in the follow-up multisite randomized controlled trial; 4) To examine the effect of pioglitazone on markers of inflammation (cytokine levels) and oxidative stress (superoxide dismutase, malonyl aldehydes); 5) To explore the relationship between different doses and response to treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- Single (Participant)
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
- •Male or female outpatients 5-12 years of age inclusive (see Note below).
- •Meet Diagnostic and Statistical Manual of Mental Disorders, 4th Edition, Text Revision (DSM-IV) criteria. DSM-IV criteria for Autistic Disorder or Asperger's Disorder (autism spectrum disorder) will be confirmed by a clinician with expertise with individuals with ASD. Best estimate Diagnosis will be reached using DSM-IV criteria, the Autism Diagnostic Observation Schedule (ADOS-G) and the Autism Diagnostic Interview-Revised (ADI-R).
- •Have a Clinical Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at Baseline.
- •If already receiving stable non-pharmacologic educational, behavioural, and/or dietary interventions, have continuous participation during the preceding 3 months prior to Screening and will not electively initiate new or modify ongoing interventions for the duration of the study.
- •Have normal physical examination and laboratory test results at Screening. If abnormal, the finding(s) must be deemed clinically insignificant by the Investigator.
排除标准
- •Patients born prior to 35 weeks gestational age.
- •Families without sufficient command of the English Language.
- •Patients with any primary psychiatric diagnosis other than autism at Screening.
- •Patients with a current neurological disease, including, but not limited to, movement disorder, tuberous sclerosis, fragile X, and any other known genetic syndromes.
- •Pregnant female patients, female patients who are sexually active, female patients using the birth control pill for whatever reason.
- •Patients with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. Patients with evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder), respiratory, renal, hepatic, or gastrointestinal disease. Patients with stable epilepsy (no seizures for 6 months) and on stable doses of antiepileptic medications (no changes in 3 months) will be allowed in the study.
- •Patients taking psychoactive medication(s).
- •Patients taking insulin.
- •Patients unable to tolerate venipuncture procedures for blood sampling.
- •Patients with parent(s)/caregiver(s) who smoke.
- •Patients who have had previous bladder infection(s).
- •Patients with a family history of bladder cancer.
研究组 & 干预措施
Placebo
干预措施: Placebo (Drug)
Pioglitazone
A modified dose finding method will be used to determine safety and dose response among three dose levels (0.25mg/kg QD, 0.5mg/kg QD, and 0.75mg/kg QD). There will be 14 weeks of active treatment.
干预措施: Pioglitazone (Drug)
结局指标
主要结局
Maximum tolerated dose to be used in the follow-up multisite randomized controlled trial
时间窗: 16 Weeks
Maximum Tolerated Dose (MTD)
Safety of pioglitazone in children with ASD ages 5-12 years
时间窗: 16 Weeks
This will be measured by the Safety Monitoring Uniform Report Form (SMURF)
Efficacy of outcome measure to be used in a follow-up multisite randomized control trial of pioglitazone in children with ASD
时间窗: 16 Weeks
This will be measured by the Child and Adolescent Symptom Inventory (CASI) - Anxiety Subscale
次要结局
- Efficacy of pioglitazone on markers of inflammation (cytokine levels) and oxidative stress (superoxide dismutase, malonyl aldehydes)(16 Weeks)
- Relationship between different doses and response to treatment(16 Weeks)
研究者
Evdokia Anagnostou
Clinician Scientist
Anagnostou, Evdokia, M.D.