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| Name | Class |
|---|---|
| McMaster University | OTHER |
| The Hospital for Sick Children | OTHER |
| The University of Western Ontario | UNKNOWN |
| Unity Health Toronto |
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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.
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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Riluzole | Active Comparator |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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 |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
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:
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.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| 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 |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Offord Centre for Child Studies | Hamilton | Ontario | L8S 4K1 | Canada | ||
| Lawson Health Research Institute |
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| ID | Term |
|---|---|
| D000067877 | Autism Spectrum Disorder |
| ID | Term |
|---|---|
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| ID | Term |
|---|---|
| D019782 | Riluzole |
| ID | Term |
|---|---|
| D013844 | Thiazoles |
| D013457 | Sulfur Compounds |
| D009930 | Organic Chemicals |
| D052160 | Benzothiazoles |
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| OTHER |
| University of Toronto | OTHER |
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| 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 |
|
| London |
| Ontario |
| N6A 5W9 |
| Canada |
| Holland Bloorview Kids Rehabilitation Hospital | Toronto | Ontario | M4G 1R8 | Canada |
| D001393 |
| Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |