A Follow-Up Shiftability Study of Arbaclofen with an Open-Label Extension for the study of Biomarkers for Treatment for Social Function in Children and Adolescents with Autism Spectrum Disorders

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Psychiatry and Psychology [F] - Behavior and Behavior Mechanisms [F01]

Trial duration

20 Nov 2025 → ongoing

Decision date (initial)

2024-09-30

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

EU funded (IMI programme)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy

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

Secondary objectives 1

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

Conditions and MedDRA coding

autism spectrum disorder

Study design 1 period

Regulatory references

Plan to share IPD

Yes

IPD plan description

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.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1) Signed Written Informed Consent a. Participants or their legal representative must have signed and dated an IRB/IEC approved written informed consent form in accordance with regulatory and institutional guidelines. This must be obtained before the performance of any protocol related procedures that are not part of normal participant care. Participants who do not have the capacity to consent will give developmentally appropriate assent. Participants who become adults (18 years of age) during the trial will sign a specific consent during the first visit when they are 18. b. Participants must be willing and able to comply with scheduled visits, treatment schedule, and laboratory testing. c. The participant’s parent/caregiver/LAR must be able to speak and understand the local language where the study is conducted sufficiently to understand the nature of the study and to allow for the completion of all study assessments. The same parent/caregiver/LAR must be capable of providing reliable information about the participant’s condition, agree to oversee the administration of study drug, and accompany the participant to all clinic visits. d. Patient must be able to speak and understand the local language where the study is conducted sufficiently to understand the nature of the study and to allow for the completion of all study assessments.
2. 2) Type of Participant and Target Disease Characteristics a. Diagnosis of an Autism Spectrum Disorder according to the DSM-5 criteria b. Participarts are within the age-range: 5 to 23yo. c. 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. d. Current psychotherapeutic/psychosocial interventions affecting behaviour stable for 3 months prior to screening and expected to be stable during the duration of the study (standard regular school breaks and/or annual teacher/classroom change do not qualify for intervention change). e. 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.
3. 3) Age, Residential and Reproductive Status a. Male or female participants 7 to 23 years of age at the time of providing consent, inclusive. b. Reside or regular contact (at least twice a week) with the parent/carer who is interviewed for the study. c. Negative pregnancy test for females of childbearing potential (participant has experienced onset of menses) d. 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. e. Male participants with female partners of childbearing potential are eligible to participate if they agree to the conditions stated in section 8.2.1.

Exclusion criteria 5

1. 1) Medical Conditions a. 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. This includes, but is not limited to impairment of renal function, evidence or history of malignancy or any significant haematological, endocrine, respiratory, hepatic, cardiovascular or gastrointestinal disease, including any clinically significant abnormalities on ECG. In general, any co-morbid conditions that may interact with study procedures. b. Participants previously excluded from AIMS-2 CT1 due to adverse events.
2. 2) Prior/Concomitant Therapy a. 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. Only occasional benzodiazepine (or derivative drugs) use (PM, i.e. at night) will be allowed. However, participants will be asked to abstain from it, if possible, the night before the recording of the EEG (i.e. visits 1, 2 and 7). b. 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. c. 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. d. Participants who have taken another investigational drug within the last 30 days.
3. 3) Physical and Laboratory Test Findings a. 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.
4. 4) Study-medication related a. Participants who are not able to take oral medications. b. Participants who have a history of hypersensitivity to racemic baclofen. c. Participants with rare hereditary problems of galactose intolerance, the lactase deficiency or glucose-galactose malabsorption should not take this medicine. d. Active peptic ulceration as Baclofen stimulates gastric acid secretion. e. Porphyria.
5. 5) Other exclusion criteria a. Participants who are currently engaged in illicit drug use or alcohol abuse, according to DSM-5 criteria. b. Participants who have previously participated in a clinical trial with arbaclofen (other than our AIMS-2-CT1). c. Women who are breastfeeding.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

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

Secondary endpoints 1

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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Sponsor organisation

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Address

Calle Del Dr. Esquerdo 46

City

Madrid

Postcode

28007

Country

Spain

Scientific contact point

Organisation

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Contact name

María de la Cruz

Public contact point

Organisation

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Contact name

María de la Cruz

Locations

2 EU/EEA countries · 5 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 50 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications