Dose Finding Study of Pioglitazone in Children With Autism Spectrum Disorders (ASD) (PIO)
Verified June 2014 by Anagnostou, Evdokia, M.D.
Holland Bloorview Kids Rehabilitation Hospital
Information provided by (Responsible Party):
Evdokia Anagnostou, Anagnostou, Evdokia, M.D.
First received: September 16, 2010
Last updated: June 19, 2014
Last verified: June 2014
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.
Autism Spectrum Disorders
||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
||A Pilot Dose Finding Study of Pioglitazone in Children With ASD
Primary Outcome Measures:
- Clinical Global Impression - Improvement (CGI-I) [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||November 2014 (Final data collection date for primary outcome measure)
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). The dose has been based on the per weight maximum adult dose (see Dose Calculation Chart). Specifically, the FDA has approved 45mg as the maximum adult dose. For a 60kg adult, this is 0.75mg/kg.
|Ages Eligible for Study:
||5 Years to 12 Years
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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 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.
- 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01205282
|Holland Bloorview Kids Rehabilitation Hospital
|Toronto, Ontario, Canada, M4G 1R8 |
|Contact: Allyson Graham, MSc 416-425-6220 ext 6515 firstname.lastname@example.org |
|Principal Investigator: Evdokia Anagnostou, MD |
Holland Bloorview Kids Rehabilitation Hospital
||Evdokia Anagnostou, MD
||Holland Bloorview Kids Rehabilitation Hospital
No publications provided
||Evdokia Anagnostou, Clinician Scientist, Anagnostou, Evdokia, M.D.
History of Changes
|Other Study ID Numbers:
|Study First Received:
||September 16, 2010
||June 19, 2014
||Canada: Health Canada
Keywords provided by Anagnostou, Evdokia, M.D.:
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on September 18, 2014
Child Development Disorders, Pervasive
Mental Disorders Diagnosed in Childhood
Physiological Effects of Drugs