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

NCT03887676 | Completed Phase 2 DMC

• 1 other identifier: ARB-05-2018
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 4

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).

Conditions

Autism Spectrum Disorder

Keywords

ASD; Autism

Outcome Measures

Primary Outcomes (1)

• Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) - Social Domain

  To examine the effect of arbaclofen vs. placebo social function

  16 weeks

Secondary Outcomes (7)

• Clinical Global Impressions - Impression Scale - Improvement (CGI-I)

  16 weeks

• Aberrant Behavior Checklist (ABC) - Social Withdrawal Subscale

  16 weeks

• Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) - Communication Domain

  16 weeks

• Safety Monitoring Uniform Report Form (SMURF)

  16 weeks

• Clinical Global Impressions - Impression Scale - Global (CGI-I-Global)

  16 weeks

Study Arms (2)

Arbaclofen

ACTIVE COMPARATOR

Drug: Arbaclofen

Placebo

PLACEBO COMPARATOR

Other: Placebo

Interventions

Arbaclofen DRUG

Administered orally as disintegrating tabs, round, white and beveled edges, at the following strengths: 5mg, 10mg, 15mg and 20mg

Arbaclofen

Placebo OTHER

Administered orally as disintegrating tabs, round, white and beveled edges

Placebo

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Outpatients 5-17 years of age inclusive.
• 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).
• Complex language to qualify for ADOS-2 modules 3 or 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.
• 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.
• 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 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).

You may not qualify if:

• Pregnant females; sexually active females on inadequate birth control.
• 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.).
• 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).
• Have a history of drug abuse.
• Have hypersensitivity to arbaclofen or any components of its formulation.
• Unable to tolerate venipuncture procedures for blood sampling.
• Actively enrolled in another intervention study.
• Taking racemic bacblofen, vigabatrin, tiagapine, riluzole, clobazam or regular benzodiazepine use (prn and hs use is allowed).
• Unable to take oral medications.
• Known hypersensitivity to racemic baclofen.
• Inability to speak and understand English sufficiently enough to allow for the completion of all study assessments (parent/legal guardian; participant).

Sponsors & Collaborators

• Holland Bloorview Kids Rehabilitation Hospital: lead
• McMaster University: collaborator
• Western University, Canada: collaborator
• Queen's University: collaborator
• Unity Health Toronto: collaborator
• University of Toronto: collaborator

Study Sites (4)

McMaster University, Offord Centre for Child Studies

Hamilton, Ontario, L8S 4K1, Canada

Location

Queen's Universtiy

Kingston, Ontario, K7M 8A6, Canada

Location

University of Western Ontario, Lawson Health Research Institute

London, Ontario, N6A 5W9, Canada

Location

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, M4G 1R8, Canada

Location

Related Publications (1)

• Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2.

  PMID: 37811711 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Interventions

arbaclofen placarbil

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 21, 2019
• First Posted: March 25, 2019
• Study Start: March 18, 2019
• Primary Completion: August 4, 2023
• Study Completion: August 4, 2023
• Last Updated: July 16, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CA (4)