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Autism Spectrum DisorderPhase 2ARBANCT03887676

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

A Phase 2 study testing Arbaclofen in people with Autism Spectrum Disorder, run by Holland Bloorview Kids Rehabilitation Hospital at 4 sites in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 5 to 17 years
Conditions the study is for
Autism Spectrum Disorder
What is being tested?
Arbaclofen
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
90 participants
Where
4 sitesCanada
Study dates
18 Mar 2019to 4 Aug 2023
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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).

Who cannot join

  • 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.
Show all 11Show fewer
  • 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).

Trial locations and contacts

4 sites in 1 country.

Canada4 sites

  • McMaster University, Offord Centre for Child Studies

    Hamilton, Ontario, L8S 4K1

  • Queen's Universtiy

    Kingston, Ontario, K7M 8A6

  • University of Western Ontario, Lawson Health Research Institute

    London, Ontario, N6A 5W9

  • Holland Bloorview Kids Rehabilitation Hospital

    Toronto, Ontario, M4G 1R8

Other trials in Canada

About this study

Official title: A Randomized Placebo-controlled Trial of ARBaclofen vs. Placebo in the Treatment of Children and Adolescents With ASD

This study will examine the safety and efficacy of arbaclofen vs. placebo on social function in children and adolescents with Autism Spectrum Disorder (ASD).

Read the 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.

Being tested
Arbaclofen, Placebo
Keywords
ASD, Autism
Medical subject headings
Autism Spectrum Disorder, Autistic Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ArbaclofenDrug

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

  • PlaceboOther

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

Study groups in NCT03887676
Study groupRoleWhat they receive
ArbaclofenActive comparatorNot described
Receives: Drug: Arbaclofen
PlaceboPlacebo comparatorNot described
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    To examine the effect of arbaclofen vs. placebo social function

    Measured: 16 weeks

Secondary outcomes (7)

  1. Clinical Global Impressions - Impression Scale - Improvement (CGI-I)

    To examine the effect of arbaclofen vs. placebo on measures of global function

    Measured: 16 weeks

  2. Aberrant Behavior Checklist (ABC) - Social Withdrawal Subscale

    To examine the effect of arbaclofen vs. placebo on social withdrawal

    Measured: 16 weeks

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

    To examine the effect of arbaclofen vs. placebo on communication

    Measured: 16 weeks

  4. Safety Monitoring Uniform Report Form (SMURF)

    To examine the safety and tolerability of arbaclofen in children and adolescents with ASD

    Measured: 16 weeks

  5. Clinical Global Impressions - Impression Scale - Global (CGI-I-Global)

    To examine the safety and tolerability of arbaclofen in children and adolescents with ASD

    Measured: 16 weeks

  6. Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD)

    To examine the safety and tolerability of arbaclofen in children and adolescents with ASD

    Measured: 16 weeks

  7. Suicidality assessment using the Columbia-Suicide Severity Rating Scale (C-SSRS)

    To examine the safety and tolerability of arbaclofen in children and adolescents with ASD

    Measured: 16 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Mar 2019
  2. Study start18 Mar 2019
  3. Primary completion4 Aug 2023
  4. Study completion4 Aug 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
90 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03887676
Sponsor's protocol ID
ARB-05-2018
Acronym
ARBA
Status last verified
Jul 2025
First submitted
21 Mar 2019
Last updated
16 Jul 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 37811711

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Arbaclofen in people with Autism Spectrum Disorder, run by Holland Bloorview Kids Rehabilitation Hospital at 4 sites in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) - Social Domain (time frame: 16 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03887676, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2025.

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