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NCT Number: NCT07655115

Single Dose Double-blind, Placebo-controlled Cross-over (SDDBPCCO) Shiftability Study, Will be Followed by a 10-week Open-label Study With Arbaclofen (4 Weeks of Titration and Then 6 Weeks of Active/Stable Treatment). The Effects of Arbaclofen on Target EEG and ERG Metrics Will be Associated With th

study with arbaclofen (4 weeks of titration and then 6 weeks of active/stable treatment). The effects of arbaclofen on target EEG and ERG metrics will be associated with the clinical response in measures of social and general function, adaptive behaviour, social anxiety, sensory behaviours, global functioning, and quality of life in Children and Adolescents with Autism Spectrum Disorders

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Key information

Age range

0 year–64 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Assistance Publique Hopitaux De Paris, Paris, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Signed Written Informed Consent a.Participants or their legal representative must have signed and dated an IRB/IEC approved written informed consent form
  • Diagnosis of an Autism Spectrum Disorder according to the DSM-5 criteria
  • Participation in the AIMS-2 CT1 (ages at recruitment 5 to 17).
  • Current pharmacological treatment regimen affecting behaviour has been stable for at least 6 weeks prior to screening and is expected to be stable during the duration of the study
  • Current psychotherapeutic/psychosocial interventions affecting behaviour stable for 3 months prior to screening and expected to be stable during the duration of the study
  • Participants with a history of seizure disorder must currently be receiving stable treatment with anticonvulsant medication and must have been seizure free for 6 months prior to screening or must be seizure free for 3 years prior to screening if not currently on a stable (>3 months) dose of antiepileptics
  • Male or female participants 7 to 23 years of age at the time of providing consent, inclusive.
  • Reside or regular contact (at least twice a week) with the parent/carer who is interviewed for the study.
  • Negative pregnancy test for females of childbearing potential (participant has experienced onset of menses)
  • Females of childbearing potential who are sexually active must agree to use a highly effective form of contraception (i.e., existing surgical sterilization, complete or abstinence or a combination of two affective forms of contraception, such as, for example, condoms plus hormonal treatment). Please, refer to Appendix 4 for a complete list of acceptable contraception methods.(protocol)
  • Male participants with female partners of childbearing potential are eligible to participate if they agree to the conditions stated in section 8.2.1.(protocol)

Exclusion criteria

  • Participants with any condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being.
  • Participants who are currently receiving treatment with racemic baclofen, vigabatrin, tiagabine, or riluzole or other GABA-related medications (e.g. gabapentin or pregabalin) other than arbaclofen in the context of AIMS-2 CT1
  • Participants who are currently receiving pharmacologic treatment affecting behaviour (see concomitant medication section) need to have a stable dose during the 6 weeks prior to the screening visit and for the duration of the study.
  • Participating in programs including non-pharmacologic educational, behavioural, and/or dietary interventions affecting behaviour, participation in these programs must have been continuous during the 3 months prior to screening and participants or their parent/caregiver/LAR may not electively initiate new or modify ongoing interventions for the duration of the study. Typical school vacations are not considered modifications of stable programming
  • Participants who have taken another investigational drug within the last 30 days.
  • Participants with evidence of any significant haematological, endocrine, cardiovascular (including uncorrected symptomatic congenital heart disease), respiratory, renal, hepatic, or gastrointestinal disease, not including mild common paediatric diseases in these areas that are stable (e.g. mild asthma, constipation, etc.), as judged by the investigator.
  • Participants who are not able to take oral medications.
  • Participants who have a history of hypersensitivity to racemic baclofen
  • Participants with rare hereditary problems of galactose intolerance, the lactase deficiency or glucose-galactose malabsorption should not take this medicine.
  • Active peptic ulceration as Baclofen stimulates gastric acid secretion.
  • Porphyria.
  • Participants who are currently engaged in illicit drug use or alcohol abuse, according to DSM-5 criteria.
  • Participants who have previously participated in a clinical trial with arbaclofen (other than our AIMS-2-CT1).
  • Women who are breastfeeding

Treatment and study plan

Arbaclofen

Drug

initial single dose arbaclofen

Placebo

Other

initial single dose placebo

Primary outcomes

  1. Change in power in the low frequency bands (theta/alpha) (between visits 1 and 2).

    Time frame: baseline to day 7

    To predict long term response to arbaclofen based on a single dose response during the placebo-controlled randomized single dose double blind stage.

Secondary outcomes

  1. Latency of N170 change (between visits 1 and 2).

    Time frame: baseline to day 7

    To test the effect of arbaclofen on an EEG biomarker for response to faces during the placebo-controlled randomized single dose double blind stage.

  2. Change of Autism Impact Measure (AIM total (Kanne et al., 2014b, Silkey et al., 2023) and subscales. • Change in the Social Responsiveness Scale (SRS total and subscales; Constantino & Gruber, 2012a).

    Time frame: from baseline to end of treatment

    To explore the effect of arbaclofen on other measures of defining features of autism.

  3. Change in power in the higher frequency bands (gamma/beta); connectivity in the theta and alpha bands; (between visits 1 and 2).

    Time frame: baseline to day 7

    To explore EEG marker sensitive to excitatory/inhibitory changes.

Sponsors and collaborators

Lead sponsor

Hospital General Universitario Gregorio Marañon

Other

Collaborators

  • Fundación para la Investigación Biomédica del Hospital Gregorio Maranon

Registry information

Official study title

A Follow-Up Shiftability Study of Arbaclofen With an Open-Label Extension for the Study of Biomarkers in Children and Adolescents With Autism Spectrum Disorders.

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Jun 17, 2026
Registry last updated
Jun 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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