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Autism Spectrum DisordersPhase 2RILISENCT01661855

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

A Phase 2 study testing Riluzole in people with Autism Spectrum Disorders, run by Holland Bloorview Kids Rehabilitation Hospital at 3 sites 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

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. Read more about this study

Who is it for?
Ages 6 to 17 years
Conditions the study is for
Autism Spectrum Disorders
What is being tested?
Riluzole
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
58 participants
Where
3 sitesCanada
Study dates
Sep 2013to Oct 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 6 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

3 sites in 1 country.

Canada3 sites

  • Offord Centre for Child Studies

    Hamilton, Ontario, L8S 4K1

  • Lawson Health Research Institute

    London, Ontario, N6A 5W9

  • Holland Bloorview Kids Rehabilitation Hospital

    Toronto, Ontario, M4G 1R8

Other trials in Canada

About this study

Official title: A Pilot Study of Riluzole vs. Placebo in the Treatment of Children and Adolescents With Autism Spectrum Disorders

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.

Read the detailed description

There are no pharmacologic treatments available for social function deficits in individuals with Autism Spectrum Disorders (ASD). The data for pharmacologic treatment of repetitive behaviors in this disorder has also become difficult to interpret given that the last two large multisite trials of selective serotonin reuptake inhibitors (SSRIs) in autism are reported to be negative for the treatment of repetitive behaviors. Only the associated symptom of irritability has 2 drugs with FDA indications whereas no systematic data exists on the pharmacologic treatment of anxiety in ASD, and response to rates to stimulants for hyperactivity are lower than what is seen in attention deficit hyperactivity disorder (ADHD). In addition, there are no biological markers of treatment response identified in this population at this point. 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.

Being tested
Riluzole, Placebo
Keywords
Autism Spectrum Disorders
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.

  • RiluzoleDrug

    Also called Rilutek

    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

  • PlaceboDrug

    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

Study groups in NCT01661855
Study groupRoleWhat they receive
RiluzoleActive comparatorNot described
Receives: Drug: Riluzole
PlaceboPlacebo comparatorNot described
Receives: Drug: Placebo

What is being measured

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

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

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

    Measured: 12 weeks

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

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

    Measured: 12 weeks

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

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

    Measured: 12 weeks

  4. Safety and tolerability of riluzole in children and adolescents with ASD

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

    Measured: 12 Weeks

  5. 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)

    Measured: 12 Weeks

Timeline

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

  1. First posted10 Aug 2012 (estimated)
  2. Study startSep 2013
  3. Primary completionSep 2015
  4. Study completionOct 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
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
58 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01661855
Sponsor's protocol ID
RILISE-07-2013
Acronym
RILISE
Status last verified
Mar 2017
First submitted
26 Jul 2012
Last updated
16 Jul 2025
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Riluzole in people with Autism Spectrum Disorders, run by Holland Bloorview Kids Rehabilitation Hospital at 3 sites 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 10 inclusion and 10 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 16 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites 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?

Efficacy of riluzole vs. placebo on measures of social function (time frame: 12 weeks). The study lists 5 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 NCT01661855.

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 16 Jul 2025.

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