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

NCT ID: NCT03887676

Last Updated: 2025-07-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 90 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-03-18
• Study Completion Date: 2023-08-04

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).

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.

Conditions

Autism Spectrum Disorder

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Arbaclofen

Group Type: ACTIVE_COMPARATOR

Arbaclofen

Intervention Type: DRUG

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

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

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

Interventions

Arbaclofen

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

Intervention Type: DRUG

Placebo

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

Intervention Type: OTHER

Eligibility 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).

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

McMaster University

OTHER

Sponsor Role: collaborator

Western University, Canada

OTHER

Sponsor Role: collaborator

Queen's University

OTHER

Sponsor Role: collaborator

Unity Health Toronto

OTHER

Sponsor Role: collaborator

University of Toronto

OTHER

Sponsor Role: collaborator

Holland Bloorview Kids Rehabilitation Hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Evdokia Anagnostou, M.D

Role: PRINCIPAL_INVESTIGATOR

Holland Bloorview Kids Rehabilitation Hospital

Robert Nicolson, M.D

Role: PRINCIPAL_INVESTIGATOR

University of Western Ontario, Lawson Health Research Institute

Julia Frei, M.D

Role: PRINCIPAL_INVESTIGATOR

McMaster University, Offord Centre for Child Studies

Muhammad Ayub, M.D

Role: PRINCIPAL_INVESTIGATOR

Queen's University

Locations

McMaster University, Offord Centre for Child Studies

Hamilton, Ontario, Canada

Queen's Universtiy

Kingston, Ontario, Canada

University of Western Ontario, Lawson Health Research Institute

London, Ontario, Canada

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, Canada

Countries

Canada

References

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.

Reference Type: DERIVED

PMID: 37811711 (View on PubMed)

Other Identifiers

ARB-05-2018

Identifier Type: -

Identifier Source: org_study_id