A Pilot Study of Riluzole Versus Placebo in the Treatment of Children and Adolescents With ASD

NCT01661855 | Completed Phase 2 DMC

• 1 other identifier: RILISE-07-2013
• Study Type: interventional
• Target: 58
• Locations: 1 country
• Sites: 3

Brief Summary

This study will examine the potential efficacy and safety of riluzole for core and associated symptom domains of autism and will explore biological markers of safety and treatment response.

Conditions

Autism Spectrum Disorders

Keywords

Autism Spectrum Disorders

Outcome Measures

Primary Outcomes (5)

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

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

  12 weeks

• Efficacy of riluzole vs. placebo on measures of repetitive behaviors

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

  12 weeks

• Efficacy of riluzole vs. placebo on measures of repetitive behaviors

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

  12 weeks

• Safety and tolerability of riluzole in children and adolescents with ASD

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

  12 Weeks

• Safety and tolerability of riluzole in children and adolescents with ASD

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

  12 Weeks

Study Arms (2)

Riluzole

ACTIVE COMPARATOR

Drug: Riluzole

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Riluzole DRUG

50mg once daily (QD) for 12 weeks for participants 6-11 years old; 50mg twice daily (BID) for 12 weeks for participants 12-17 years old

Also known as: Rilutek

Riluzole

Placebo DRUG

Placebo comparator once daily (QD) for 12 weeks for participants 6-11 years old; Placebo comparator twice daily (BID) for 12 weeks for participants 12-17 years old

Placebo

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female outpatients 6-17 years of age inclusive, with a mental age equivalent ≥ 18 months at Screening visit.
• Meet Diagnostic and Statistical Manual (DSM-IV) criteria for an ASD.
• Have a Clinician's Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at Screening.
• If already receiving stable interventions must meet the following criteria:
• If already receiving stable concomitant medications affecting behavior, must be on a stable dose 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 study duration.
• If already receiving stable non-pharmacological educational, behavioral, and/or dietary 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 complete assessments- fluency in English (parent; patient, if verbal).
• Consent to participate in the Province of Ontario Neurodevelopmental (POND) study and commitment to completing as many stages as possible of the phenotyping measures (Stages 1, 2 and 3), genomics component, and interest in being imaged through POND.
• 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:

• Pregnant female patients; sexually active female patients on inadequate birth control.
• 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 or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder), 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 riluzole or any components of its formulation.
• Patients with one or more of the following: HIV, Hepatitis B virus, Hepatitis C virus, hemophilia (bleeding problems, recent nose and brain injuries), abnormal blood pressure (hypotension or hypertension), drug abuse, immunity disorder, major depressive episode or psychosis.
• Patients unable to tolerate venipuncture procedures for blood sampling.
• Patients receiving concomitant medications that specifically target the glutamate system (e.g. memantine, d-cycloserine), or decrease the elimination of riluzole (e.g. theophylline, quinolones), less than 30 days prior to the screening visit.
• Patients actively enrolled in another intervention study.
• Patients who are unable to swallow pills.
• Patients who have elevated liver enzymes ≥ 3 times the normal amount before the study begins.

Sponsors & Collaborators

• Holland Bloorview Kids Rehabilitation Hospital: lead
• McMaster University: collaborator
• The Hospital for Sick Children: collaborator
• The University of Western Ontario: collaborator
• Unity Health Toronto: collaborator
• University of Toronto: collaborator

Study Sites (3)

Offord Centre for Child Studies

Hamilton, Ontario, L8S 4K1, Canada

Location

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

Interventions

Riluzole

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Evdokia Anagnostou, M.D.

  Holland Bloorview Kids Rehabilitation Hospital

  PRINCIPAL INVESTIGATOR

• Robert Nicolson, M.D.

  University of Western Ontario, Lawson Health Research Institute

  PRINCIPAL INVESTIGATOR

• Terry Bennett, M.D.

  McMaster University; Offord Centre for Child Studies

  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: July 26, 2012
• First Posted: August 10, 2012
• Study Start: September 1, 2013
• Primary Completion: September 1, 2015
• Study Completion: October 1, 2015
• Last Updated: July 16, 2025

Record last verified: 2017-03

Data Sharing

• IPD Sharing: Will not share

Locations

CA (3)