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Arbaclofen vs. Placebo in the Treatment of Children and Adolescents With ASD (ARBA) (ARBA)

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ClinicalTrials.gov Identifier: NCT03887676
Recruitment Status : Recruiting
First Posted : March 25, 2019
Last Update Posted : July 3, 2019
Sponsor:
Collaborators:
Holland Bloorview Kids Rehabilitation Hospital
McMaster University
University of Western Ontario, Canada
Queen's University
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 safety and efficacy of arbaclofen vs. placebo on social function in children and adolescents with Autism Spectrum Disorder (ASD).

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: Arbaclofen Other: Placebo Phase 2

Detailed Description:
There are no pharmacologic treatments available for social function deficits in individuals with ASD. The data for pharmacologic treatment of repetitive behaviours 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 behaviours. Only the associated symptom of irritability has 2 drugs with Food and Drug Administration (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 arbaclofen for social function, and will explore biological markers of safety and treatment response.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized Placebo-controlled Trial of ARBaclofen vs. Placebo in the Treatment of Children and Adolescents With ASD
Actual Study Start Date : March 18, 2019
Estimated Primary Completion Date : May 15, 2020
Estimated Study Completion Date : May 15, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Active Comparator: Arbaclofen Drug: Arbaclofen
Administered orally as disintegrating tabs, round, white and beveled edges, at the following strengths: 5mg, 10mg, 15mg and 20mg

Placebo Comparator: Placebo Other: Placebo
Administered orally as disintegrating tabs, round, white and beveled edges




Primary Outcome Measures :
  1. Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) - Social Domain [ Time Frame: 16 weeks ]
    To examine the effect of arbaclofen vs. placebo social function


Secondary Outcome Measures :
  1. Clinical Global Impressions - Impression Scale - Improvement (CGI-I) [ Time Frame: 16 weeks ]
    To examine the effect of arbaclofen vs. placebo on measures of global function

  2. Aberrant Behavior Checklist (ABC) - Social Withdrawal Subscale [ Time Frame: 16 weeks ]
    To examine the effect of arbaclofen vs. placebo on social withdrawal

  3. Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) - Communication Domain [ Time Frame: 16 weeks ]
    To examine the effect of arbaclofen vs. placebo on communication

  4. Safety Monitoring Uniform Report Form (SMURF) [ Time Frame: 16 weeks ]
    To examine the safety and tolerability of arbaclofen in children and adolescents with ASD

  5. Clinical Global Impressions - Impression Scale - Global (CGI-I-Global) [ Time Frame: 16 weeks ]
    To examine the safety and tolerability of arbaclofen in children and adolescents with ASD

  6. Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) [ Time Frame: 16 weeks ]
    To examine the safety and tolerability of arbaclofen in children and adolescents with ASD

  7. Suicidality assessment using the Columbia-Suicide Severity Rating Scale (C-SSRS) [ Time Frame: 16 weeks ]
    To examine the safety and tolerability of arbaclofen in children and adolescents with ASD



Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Outpatients 5-17 years of age inclusive.
  2. Meet Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5). DSM-5 criteria will be established by a clinician with expertise with individuals with ASD. Diagnosis will be supported by the Autism Diagnostic Observation Schedule, Second Edition (ADOS-2).
  3. Complex language to qualify for ADOS-2 modules 3 or 4.
  4. If already receiving stable concomitant medications affecting behaviour, have stable regimens with no changes during the preceding 6 weeks prior to Screening, and will not electively initiate new or modify ongoing medications for the duration of the study.
  5. If already receiving stable non-pharmacological educational and behavioural interventions, have continuous participation during the preceding 3 months prior to Screening, and will not electively initiate new or modify ongoing interventions for the duration of the study.
  6. Have normal physical examination and laboratory test results at Screening. If abnormal, the finding(s) must be deemed clinically insignificant by the Investigator.
  7. 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(s).

Exclusion Criteria:

  1. Pregnant females; sexually active females on inadequate birth control.
  2. Have 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. Have evidence of any significant hematological, endocrine, cardiovascular (including uncorrected symptomatic congenital heart disease), 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. Have unstable epilepsy (i.e. seizures occurring within the last 6 months), or have epilepsy and not on stable doses of antiepileptic medications (i.e. dose changes within the last 3 months).
  4. Have a history of drug abuse.
  5. Have hypersensitivity to arbaclofen or any components of its formulation.
  6. Unable to tolerate venipuncture procedures for blood sampling.
  7. Actively enrolled in another intervention study.
  8. Taking racemic bacblofen, vigabatrin, tiagapine, riluzole, clobazam or regular benzodiazepine use (prn and hs use is allowed).
  9. Unable to take oral medications.
  10. Known hypersensitivity to racemic baclofen.
  11. Inability to speak and understand English sufficiently enough to allow for the completion of all study assessments (parent/legal guardian; participant).

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): NCT03887676


Contacts
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Contact: Karima Noordin 416-864-6060 ext 3932 noordink@smh.ca
Contact: Vanessa Ballin 416-425-6220 ext 6602 vballin@hollandbloorview.ca

Locations
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Canada, Ontario
McMaster University, Offord Centre for Child Studies Not yet recruiting
Hamilton, Ontario, Canada, L8S 4K1
Contact: Julia Frei, M.D         
Queen's Universtiy Not yet recruiting
Kingston, Ontario, Canada, K7M 8A6
Contact: Muhammad Ayub, M.D         
University of Western Ontario, Lawson Health Research Institute Recruiting
London, Ontario, Canada, N6A 5W9
Contact: Robert Nicolson, M.D         
Holland Bloorview Kids Rehabilitation Hospital Recruiting
Toronto, Ontario, Canada, M4G 1R8
Contact: Evokdia Anagnostou, M.D         
Sponsors and Collaborators
Evdokia Anagnostou
Holland Bloorview Kids Rehabilitation Hospital
McMaster University
University of Western Ontario, Canada
Queen's University
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: Julia Frei, M.D McMaster University, Offord Centre for Child Studies
Principal Investigator: Muhammad Ayub, M.D Queen's University

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Responsible Party: Evdokia Anagnostou, Principal Investigator, Anagnostou, Evdokia, M.D.
ClinicalTrials.gov Identifier: NCT03887676     History of Changes
Other Study ID Numbers: ARB-05-2018
First Posted: March 25, 2019    Key Record Dates
Last Update Posted: July 3, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Evdokia Anagnostou, Anagnostou, Evdokia, M.D.:
ASD
Autism
Additional relevant MeSH terms:
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Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders