A Pilot Study of Riluzole Versus Placebo in the Treatment of Children and Adolescents With ASD
Information source: Anagnostou, Evdokia, M.D.
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Autism Spectrum Disorders
Intervention: Riluzole (Drug)
Phase: Phase 2
Status: Recruiting
Sponsored by: Evdokia Anagnostou Official(s) and/or principal investigator(s): Evdokia Anagnostou, MD, Principal Investigator, Affiliation: Holland Bloorview Kids Rehabilitation Hospital Peter Szatmari, MD, Principal Investigator, Affiliation: McMaster University Robert Nicolson, MD, Principal Investigator, Affiliation: University of Western Ontario, Canada Paul Arnold, MD, Principal Investigator, Affiliation: The Hospital for Sick Children Kevin Thorpe, MMath, Principal Investigator, Affiliation: Applied Health Research Centre, St Michael's Hospital ; Dalla Lana School of Public Health, University of Toronto Sharon Smile, MD, Principal Investigator, Affiliation: Holland Bloorview Kids Rehabilitation Hospital Jessica Brian, PhD, Principal Investigator, Affiliation: Holland Bloorview Kids Rehabilitation Hospital Olabode Akintan, MD, Principal Investigator, Affiliation: McMaster University; Offord Centre for Child Studies Terry Bennett, MD, Principal Investigator, Affiliation: McMaster University; Offord Centre for Child Studies Olaf Kraus de Camargo, MD, Principal Investigator, Affiliation: McMaster University: Offord Centre for Child Studies
Overall contact: Evdokia Anagnostou, MD, Email: eanagnostou@hollandbloorview.ca
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.
Clinical Details
Official title: A Pilot Study of Riluzole vs. Placebo in the Treatment of Children and Adolescents With Autism Spectrum ASD (Rilise)
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: To examine the effect of riluzole vs. placebo on measures of social functionTo examine the effect of riluzole vs. placebo on measures of repetitive behaviors To examine the safety and tolerability of riluzole in children and adolescents with ASD
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.
Eligibility
Minimum age: 6 Years.
Maximum age: 17 Years.
Gender(s): Both.
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 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 POND 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, HBV, HCV, 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.
Locations and Contacts
Evdokia Anagnostou, MD, Email: eanagnostou@hollandbloorview.ca
Offord Centre for Child Studies, Hamilton, Ontario L8S 4K1, Canada; Recruiting Alessia Greco, MA, Phone: 905-521-2100, Ext: 74906, Email: algreco@mcmaster.ca Terry Bennett, MD, Principal Investigator
Lawson Health Research Institute, London, Ontario N6A 5W9, Canada; Recruiting Ahsan Ahmad, MD, Phone: (519) 685-8500, Ext: 74906, Email: ahsan.ahmad@lhsc.on.ca Robert Nicolson, MD, Principal Investigator
Holland Bloorview Kids Rehabilitation Hospital, Toronto, Ontario M4G 1R8, Canada; Recruiting Rianne Hastie Adams, MSW, RSW, Phone: 416-425-6220, Ext: 6515, Email: rhastieadams@hollandbloorview.ca Evdokia Anagnostou, MD, Principal Investigator
Additional Information
Starting date: September 2013
Last updated: April 15, 2015
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