Arbaclofen in Children and Adolescents With ASD
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Autism Spectrum Disorder
- Type
- Interventional
- Phase
- Phase 2
- Design
- Allocation: RandomizedIntervention Model: Parallel AssignmentMasking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)Masking Description: AIMS2-CT-01 is a double-blind study where participants and parent(s)/legal guardians as well as research staff will be unaware of the treatment allocation, with the exception of a dedicated unblinded pharmacist at each individual site. Blinding of the remainder of the study team members will be maintained by utilizing identical tablets, blistercards and boxes containing either Arbaclofen or placebo. Unblinded study drug and placebo supplies will be stocked at each study site pharmacy. An online randomization system will be used, by this unblinded pharmacist, to assign the study treatment. Based on the outcome of the randomisation, the pharmacist prepares the boxes containing the applicable treatment and releases these (blinded) boxes to the investigator for dispensing to the patient. In order to maintain the study blind, the dosing regimen will be consistent whether participants are randomized to Arbaclofen or placebo.Primary Purpose: Treatment
Participation Requirements
- Age
- Between 5 years and 17 years
- Gender
- Both males and females
Description
Autism Spectrum Disorder (ASD) is a clinically and etiologically heterogeneous neurodevelopmental condition affecting approximately 1% of the population. The core symptoms of ASD are deficits in social communication and the presence of repetitive and restricted behaviours and interests, including se...
Autism Spectrum Disorder (ASD) is a clinically and etiologically heterogeneous neurodevelopmental condition affecting approximately 1% of the population. The core symptoms of ASD are deficits in social communication and the presence of repetitive and restricted behaviours and interests, including sensory anomalies. Currently, there are no effective medical treatments for the core symptoms of ASD, and families frequently use costly non evidence based interventions. Developing drugs for ASD has been challenging because of a limited understanding of its underlying pathophysiology(ies), and difficulties modelling it in vitro and in vivo. A recent study from EU-AIMS reported, for the first time in ASD, that differences in E-I balance can be 'shifted' using a GABA acting drug (riluzole), and that abnormalities in functional connectivity can be 'normalised' by targeting E-I, even in adults. This offers promise that drugs targeting specific parts of the GABA pathway may improve symptoms. The aim of the investigator's project is to conduct a double-blind Randomized Control Trial (RCT) focused on GABA/glutamate equilibrium, to assess the efficacy of a drug that targets core and/or comorbid symptoms in ASD. Arbaclofen is a selective GABA-B receptor agonist and augments GABA-ergic activity, inhibits presynaptic release of glutamate, inhibits postsynaptic transmission, and modulates intracellular signalling. Through elevation of GABA-ergic inhibitory activity, Arbaclofen may act to alleviate ASD symptoms with social anxiety and emotional hyperarousal. Hypothesis: Arbaclofen will be superior to placebo in improving social function as measured by the Vineland-3 social domain.
Tracking Information
- NCT #
- NCT03682978
- Collaborators
- UMC Utrecht
- Investigators
- Study Chair: Celso Arango, Prof. Hospital General Universitario Gregorio Marañón Principal Investigator: Mara Parellada, Prof. Hospital General Universitario Gregorio Marañón Principal Investigator: Richard Delorme, Prof. Robert Debré Hospital Principal Investigator: Jeremy Parr, Prof. University of Newcastle Upon-Tyne Principal Investigator: Mallika Punukollu, Dr. University of Glasgow Principal Investigator: Andre Strydom, Prof. King's College London Principal Investigator: Rosa Calvo, Dr. Hospital Clinic of Barcelona
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