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Autism Spectrum DisordersPhase 2NCT01205282

Dose Finding Study of Pioglitazone in Children With Autism Spectrum Disorders (ASD) (PIO)

A Phase 2 study testing Pioglitazone in people with Autism Spectrum Disorders, run by Evdokia Anagnostou at 1 site in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 5 to 12 years
Conditions the study is for
Autism Spectrum Disorders
What is being tested?
Pioglitazone
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
28 participants
Where
1 siteCanada
Study dates
Apr 2013to Sep 2015
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 12 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • Holland Bloorview Kids Rehabilitation Hospital

    Toronto, Ontario, M4G 1R8

Other trials in Canada

About this study

Official title: A Pilot Dose Finding Study of Pioglitazone in Children With ASD

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.

Being tested
Pioglitazone, Placebo
Keywords
ASD
Medical subject headings
Autism Spectrum Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PioglitazoneDrug

    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. Specifically, the FDA has approved 45mg as the maximum adult dose. For a 60kg adult, this is 0.75mg/kg. There will be 14 weeks of active treatment.

  • PlaceboDrug

    There will be a 2 week period of placebo run-in.

Study groups in NCT01205282
Study groupRoleWhat they receive
PioglitazoneExperimentalA 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.
Receives: Drug: Pioglitazone
PlaceboPlacebo comparatorNot described
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety of pioglitazone in children with ASD ages 5-12 years

    This will be measured by the Clinical Global Impressions - Improvement Scale - Global (CGI-I-Global)

    Measured: 16 Weeks

  2. Safety of pioglitazone in children with ASD ages 5-12 years

    This will be measured by the Safety Monitoring Uniform Report Form (SMURF)

    Measured: 16 Weeks

  3. Efficacy of outcome measure to be used in a follow-up multisite randomized control trial of pioglitazone in children with ASD

    This will be measured by the Aberrant Behavior Checklist (ABC)

    Measured: 16 Weeks

  4. Efficacy of outcome measure to be used in a follow-up multisite randomized control trial of pioglitazone in children with ASD

    This will be measured the Social Responsiveness Scale (SRS)

    Measured: 16 Weeks

  5. Efficacy of outcome measure to be used in a follow-up multisite randomized control trial of pioglitazone in children with ASD

    This will be measured by the the Child Yale-Brown Obsessive-Compulsive Scale (CY-BOCS)

    Measured: 16 Weeks

  6. Efficacy of outcome measure to be used in a follow-up multisite randomized control trial of pioglitazone in children with ASD

    This will be measured by the Repetitive Behavior Scale - Revised (RBS-R)

    Measured: 16 Weeks

  7. Efficacy of outcome measure to be used in a follow-up multisite randomized control trial of pioglitazone in children with ASD

    This will be measured by the Behavioral Assessment System for Children (BASC-2)

    Measured: 16 Weeks

  8. Efficacy of outcome measure to be used in a follow-up multisite randomized control trial of pioglitazone in children with ASD

    This will be measured by the Child and Adolescent Symptom Inventory (CASI) - Anxiety Subscale

    Measured: 16 Weeks

  9. Maximum tolerated dose to be used in the follow-up multisite randomized controlled trial

    Maximum Tolerated Dose (MTD)

    Measured: 16 Weeks

Secondary outcomes (2)

  1. Efficacy of pioglitazone on markers of inflammation (cytokine levels) and oxidative stress (superoxide dismutase, malonyl aldehydes)

    Cytokine level and oxidative stress marker measurement

    Measured: 16 Weeks

  2. Relationship between different doses and response to treatment

    Pioglitazone dose and treatment response

    Measured: 16 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Sep 2010 (estimated)
  2. Study startApr 2013
  3. Primary completionSep 2015
  4. Study completionSep 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
Single blind
Who is masked
Participant
Enrolment
28 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01205282
Sponsor's protocol ID
10-002
Status last verified
Mar 2017
First submitted
16 Sep 2010
Last updated
20 Mar 2017
Results posted
No
Data monitoring committee
No
Individual data sharing
No

Publications

  • Capano L, Dupuis A, Brian J, Mankad D, Genore L, Hastie Adams R, Smile S, Lui T, Odrobina D, Foster JA, Anagnostou E. A pilot dose finding study of pioglitazone in autistic children. Mol Autism. 2018 Nov 26;9:59. doi: 10.1186/s13229-018-0241-5. eCollection 2018.

    PubMed 30498564

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pioglitazone in people with Autism Spectrum Disorders, run by Evdokia Anagnostou at 1 site in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety of pioglitazone in children with ASD ages 5-12 years (time frame: 16 Weeks). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01205282, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Mar 2017.

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