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RecruitingNCT07655115Updated Jun 17, 2026

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

A Phase 3 interventional study of Arbaclofen and Placebo in Autism Spectrum Disorders, sponsored by Hospital General Universitario Gregorio Marañon. Recruiting at 5 sites in 2 countries. Open to participants aged 0 Years to 64 Years. Per ClinicalTrials.gov, last updated 2026-06-17.

Sponsored by Hospital General Universitario Gregorio Marañon · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
103
Allocation
Randomized
Ages
0 Years to 64 Years
Sex
All
01

Study 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

02

Conditions studied

  • Autism Spectrum Disorders
03

Who can participate

Ages eligible
0 Years to 64 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Crossover assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
103 participants (estimated)

Study arms

  • Experimental
    arbaclofen

    Drug: Arbaclofen

  • Placebo comparator
    placebo

    Other: Placebo

Interventions

  • DrugArbaclofen

    initial single dose arbaclofen

  • OtherPlacebo

    initial single dose placebo

05

What researchers measure

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.

    Time frame: baseline to day 7

Secondary outcomes

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

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

    Time frame: baseline to day 7

  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).

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

    Time frame: from baseline to end of treatment

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

    To explore EEG marker sensitive to excitatory/inhibitory changes.

    Time frame: baseline to day 7

06

Study locations

5 of 5 sites recruiting
  • Assistance Publique Hopitaux De Paris
    Paris, 75019, France
    • Richard Delorme, PhD · Contact · richard.delorme@aphp.fr · +31140034130
    • Richard Delorme, PhD · Principal investigator
    Recruiting
  • Hospital Clinic De Barcelona
    Barcelona, 08036, Spain
    • Rosa Calvo, PhD · Contact · rcalvo@clinic.cat · +34932279974
    • Rosa Calvo, PhD · Principal investigator
    Recruiting
  • Hospital General Universitario Gregorio Marañón
    Madrid, 28007, Spain
    • Maria José Parellada, PhD · Contact · parelladahggm@gmail.com · +34 91 4265005
    • María José Parellada, PhD · Principal investigator
    Recruiting
  • Hospital Universitario De Salamanca
    Salamanca, Spain
    • Ricardo Canal-Bedia, PhD · Contact · rcanal@usal.es · +34638766776
    • Ricardo Canal-Bedia, PhD · Principal investigator
    Recruiting
  • Complejo Asistencial De Zamora Hospital Provincial De Zamora
    Zamora, 49020, Spain
    Recruiting
07

References and documents

Individual participant data

Plan to share: Yes — De-identified data will be entered in a GDPR compliant EDC by individual sites, and after thorough checks shared with the sponsor, according to the CTA. Data will possibly be shared after thorough data cleaning and checks of de-identification of individuals, with other parties of the consortium, if participants have agreed to that in the Informed Consent Form. Examples of these data would be EEG data, that would be uploaded in a secured server, and analysed by a consortium partner.

Supporting information: Csr

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07655115
Lead sponsor
Hospital General Universitario Gregorio Marañon
Collaborators
Fundación para la Investigación Biomédica del Hospital Gregorio Maranon
Responsible party
Sponsor
First posted
Jun 17, 2026
Start date
Nov 20, 2025
Primary completion
Dec 31, 2026 (estimated)
Completion
Dec 31, 2026 (estimated)
Last update
Jun 17, 2026

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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