Tideglusib vs. Placebo in the Treatment of Adolescents With Autism Spectrum Disorders

NCT02586935 | Completed Phase 2 DMC

• 1 other identifier: TIDE-06-2015
• Study Type: interventional
• Target: 83
• Locations: 1 country
• Sites: 3

Brief Summary

This study will examine the safety and efficacy of tideglusib vs. placebo for the treatment of core symptom domains in adolescents with Autism Spectrum Disorders

Conditions

Autism Spectrum Disorders

Keywords

ASD; Autism

Outcome Measures

Primary Outcomes (1)

• Effect of tideglusib vs. placebo on measures of social engagement/withdrawal

  This will be measured by the Aberrant Behavior Checklist (ABC) - Lethargy / Social Withdrawal Subscale

  12 weeks

Secondary Outcomes (8)

• Efficacy of tideglusib vs. placebo on measures of repetitive behaviours

  12 weeks

• Efficacy of tideglusib vs. placebo on measures of repetitive behaviours

  12 weeks

• Efficacy of tideglusib vs. placebo on measures of social function

  12 weeks

• Safety and tolerability of tideglusib in adolescents with ASD

  12 weeks

• Safety and tolerability of tideglusib in adolescents with ASD

  12 weeks

Study Arms (2)

Tideglusib

ACTIVE COMPARATOR

Drug: Tideglusib

Placebo

PLACEBO COMPARATOR

Other: Placebo

Interventions

Tideglusib DRUG

Administered orally after dispersion in approximately 100 ml of water at dose levels of 400 to 1000 mg

Tideglusib

Placebo OTHER

Administered orally after dispersion in approximately 100 ml of water

Placebo

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Outpatients 12-17 years of age inclusive with a mental age equivalent ≥ 18 months at Screening.
• Weigh a minimum of 30 kg (the 3rd percentile for 12 years of age)
• Meet Diagnostic and Statistical Manual of Mental Disorders. Diagnostic and Statistical Manual (DSM-5) criteria will be established by a clinician with expertise with individuals with ASD.
• Have a Clinician's Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at Screening.
• If already receiving stable concomitant medications affecting behaviour, have stable regimens with no changes during the preceding 1 month prior to Screening (with the exception of fluoxetine, where a period of 6 weeks is needed), and will not electively initiate new or modify ongoing medications for the duration of the study
• If already receiving stable non-pharmacological educational and behavioural interventions, have continuous participation during the preceding 3 months prior to Screening, and 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.
• Ability to obtain written informed consent from the participant, if developmentally appropriate. If a participant does not have the capacity to consent, ability to obtain assent (if developmentally appropriate), as well as written informed consent from their parent(s)/legal guardian.

You may not qualify if:

• Patients with a primary psychiatric diagnosis other than ASD
• Pregnant female patients; sexually active female patients on inadequate birth control.
• Patients with known phosphatase and tensin homolog (PTEN) mutations as they are unlikely to respond to this medication
• Patients with a serious medical condition that, based on Investigator judgment, might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. Patients with evidence of any significant hematological, endocrine, cardiovascular (including uncorrected symptomatic congenital heart disease), respiratory, renal, hepatic, or gastrointestinal disease, not including mild common pediatric diseases in these areas that are stable (e.g. mild asthma, constipation, etc.).
• Patients with unstable epilepsy (i.e. seizures occurring within the last 6 months), or patients with epilepsy who are not on stable doses of antiepileptic medications (i.e. dose changes within the last 3 months).
• Patients with hypersensitivity to tideglusib or any components of its formulation.
• Patients unable to tolerate venipuncture procedures for blood sampling.
• Patients actively enrolled in another intervention study.
• Patients who have elevated liver enzymes ≥ 3 times the normal amount before the study begins.
• Patients who have serum creatinine of \>150 μmol/L and creatinine clearance ≤60ml/m (according to Cockcroft-Gault formula) at Screening.
• Patients taking strong CYP3A4 inhibitors (e.g. clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, indinavir, ritonavir)
• Inability to speak and understand English sufficiently enough to allow for the completion of all study assessments (parent; patient, if verbal).

Sponsors & Collaborators

• Holland Bloorview Kids Rehabilitation Hospital: lead
• McMaster University: collaborator
• Western University, Canada: collaborator
• Unity Health Toronto: collaborator
• University of Toronto: collaborator

Study Sites (3)

McMaster University, Offord Centre for Child Studies

Hamilton, Ontario, L8S 4K1, Canada

Location

University of Western Ontario, Lawson Health Research Institute

London, Ontario, N6A 5W9, Canada

Location

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, M4G 1R8, Canada

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Interventions

tideglusib

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Evdokia Anagnostou, M.D.

  Holland Bloorview Kids Rehabilitation Hospital

  PRINCIPAL INVESTIGATOR

• Terry Bennett, M.D.

  MacMaster University, Offord Centre for Child Studies

  PRINCIPAL INVESTIGATOR

• Robert Nicolson, M.D.

  University of Western Ontario, Lawson Health Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 16, 2015
• First Posted: October 27, 2015
• Study Start: February 10, 2016
• Primary Completion: February 25, 2018
• Study Completion: February 25, 2018
• Last Updated: July 16, 2025

Record last verified: 2018-05

Data Sharing

• IPD Sharing: Will not share

Locations

CA (3)