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

NCT07655115 | Recruiting Phase 3 DMC

• 2 other identifiers: AIMS-2-CT22023-508407-20-00
• Study Type: interventional
• Target: 103
• Locations: 2 countries
• Sites: 5

Brief Summary

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

Conditions

Autism Spectrum Disorders

Outcome Measures

Primary Outcomes (1)

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

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

  baseline to day 7

Secondary Outcomes (3)

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

  baseline to day 7

• 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).

  from baseline to end of treatment

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

  baseline to day 7

Study Arms (2)

arbaclofen

EXPERIMENTAL

Drug: Arbaclofen

placebo

PLACEBO COMPARATOR

Other: Placebo

Interventions

Arbaclofen DRUG

initial single dose arbaclofen

arbaclofen

Placebo OTHER

initial single dose placebo

placebo

Eligibility Criteria

• Age: 0 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Hospital General Universitario Gregorio Marañon: lead
• Fundación para la Investigación Biomédica del Hospital Gregorio Maranon: collaborator

Study Sites (5)

Assistance Publique Hopitaux De Paris

Paris, 75019, France

RECRUITING

Hospital Clinic De Barcelona

Barcelona, 08036, Spain

RECRUITING

Hospital General Universitario Gregorio Marañón

Madrid, 28007, Spain

RECRUITING

Hospital Universitario De Salamanca

Salamanca, Spain

RECRUITING

Complejo Asistencial De Zamora Hospital Provincial De Zamora

Zamora, 49020, Spain

RECRUITING

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

arbaclofen placarbil

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: An initial single dose double-blind, placebo-controlled cross-over (SDDBPCCO) shiftability study, will be followed on autistic population by a 10-week open-label study with arbaclofen (4 weeks of titration and then 6 weeks of active/stable treatment).

Study Record Dates

• First Submitted: March 17, 2025
• First Posted: June 17, 2026
• Study Start: November 20, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: June 17, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: CSR
• Time Frame: 365 days after study finalizes

Locations

FR (1); ES (4)