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

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ClinicalTrials.gov Identifier: NCT01661855
Recruitment Status : Completed
First Posted : August 10, 2012
Last Update Posted : March 20, 2017
Sponsor:
Collaborators:
Holland Bloorview Kids Rehabilitation Hospital
McMaster University
The Hospital for Sick Children
University of Western Ontario, Canada
St. Michael's Hospital, Toronto
University of Toronto
Information provided by (Responsible Party):
Evdokia Anagnostou, Anagnostou, Evdokia, M.D.

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.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorders Drug: Riluzole Drug: Placebo Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 58 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Pilot Study of Riluzole vs. Placebo in the Treatment of Children and Adolescents With Autism Spectrum Disorders
Study Start Date : September 2013
Actual Primary Completion Date : September 2015
Actual Study Completion Date : October 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Riluzole

Arm Intervention/treatment
Active Comparator: Riluzole Drug: Riluzole
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
Other Name: Rilutek

Placebo Comparator: Placebo Drug: Placebo
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




Primary Outcome Measures :
  1. Efficacy of riluzole vs. placebo on measures of social function [ Time Frame: 12 weeks ]
    This will be measured by the Aberrant Behavior Checklist (ABC) - Lethargy / Social Withdrawal Subscale

  2. Efficacy of riluzole vs. placebo on measures of repetitive behaviors [ Time Frame: 12 weeks ]
    This will be measured by the Child Yale-Brown Obsessive Compulsive Scale (CY-BOCS)

  3. Efficacy of riluzole vs. placebo on measures of repetitive behaviors [ Time Frame: 12 weeks ]
    This will be measured by the Repetitive Behavior Scale (RBS-R)

  4. Safety and tolerability of riluzole in children and adolescents with ASD [ Time Frame: 12 Weeks ]
    This will be measured by the Safety Monitoring Uniform Report Form (SMURF)

  5. Safety and tolerability of riluzole in children and adolescents with ASD [ Time Frame: 12 Weeks ]
    This will be measured by the Clinical Global Impressions - Improvement Scale - Global (CGI-I-Global)



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female outpatients 6-17 years of age inclusive, with a mental age equivalent ≥ 18 months at Screening visit.
  2. Meet Diagnostic and Statistical Manual (DSM-IV) criteria for an ASD.
  3. Have a Clinician's Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at Screening.
  4. If already receiving stable interventions must meet the following criteria:

    1. 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.
    2. 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.
  5. Have normal physical examination and laboratory test results at Screening. If abnormal, the finding(s) must be deemed clinically insignificant by the Investigator.
  6. Ability to complete assessments- fluency in English (parent; patient, if verbal).
  7. 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.
  8. 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.

Exclusion Criteria:

  1. Pregnant female patients; sexually active female patients on inadequate birth control.
  2. 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.).
  3. 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).
  4. Patients with hypersensitivity to riluzole or any components of its formulation.
  5. 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.
  6. Patients unable to tolerate venipuncture procedures for blood sampling.
  7. 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.
  8. Patients actively enrolled in another intervention study.
  9. Patients who are unable to swallow pills.
  10. Patients who have elevated liver enzymes ≥ 3 times the normal amount before the study begins.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01661855


Locations
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Canada, Ontario
Offord Centre for Child Studies
Hamilton, Ontario, Canada, L8S 4K1
Lawson Health Research Institute
London, Ontario, Canada, N6A 5W9
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, Canada, M4G 1R8
Sponsors and Collaborators
Evdokia Anagnostou
Holland Bloorview Kids Rehabilitation Hospital
McMaster University
The Hospital for Sick Children
University of Western Ontario, Canada
St. Michael's Hospital, Toronto
University of Toronto
Investigators
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Principal Investigator: 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

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Responsible Party: Evdokia Anagnostou, Principal Investigator, Anagnostou, Evdokia, M.D.
ClinicalTrials.gov Identifier: NCT01661855     History of Changes
Other Study ID Numbers: RILISE-07-2013
First Posted: August 10, 2012    Key Record Dates
Last Update Posted: March 20, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Evdokia Anagnostou, Anagnostou, Evdokia, M.D.:
Autism Spectrum Disorders

Additional relevant MeSH terms:
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Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Riluzole
Anticonvulsants
Excitatory Amino Acid Antagonists
Excitatory Amino Acid Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Neuroprotective Agents
Protective Agents