ARBACLOFEN for Autism spectrum disorder

Inclusion criteria

• {"criterion_text":"-1)\tSigned Written Informed Consent a.\tParticipants 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.\tParticipants must be willing and able to comply with scheduled visits, treatment schedule, and laboratory testing. c.\tThe 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.\tPatient 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."}
• {"criterion_text":"-2)\tType of Participant and Target Disease Characteristics a.\tDiagnosis of an Autism Spectrum Disorder according to the DSM-5 criteria b.\tParticiparts are within the age-range: 5 to 23yo. c.\tCurrent 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.\tCurrent 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.\tParticipants 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."}
• {"criterion_text":"-3)\tAge, Residential and Reproductive Status a.\tMale or female participants 7 to 23 years of age at the time of providing consent, inclusive. b.\tReside or regular contact (at least twice a week) with the parent/carer who is interviewed for the study. c.\tNegative pregnancy test for females of childbearing potential (participant has experienced onset of menses) d.\tFemales 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.\tMale 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

• {"criterion_text":"-1)\tMedical Conditions a.\tParticipants 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.\tParticipants previously excluded from AIMS-2 CT1 due to adverse events."}
• {"criterion_text":"-2)\tPrior/Concomitant Therapy a.\tParticipants 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.\tParticipants 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.\tParticipating 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.\tParticipants who have taken another investigational drug within the last 30 days."}
• {"criterion_text":"-3)\tPhysical and Laboratory Test Findings a.\tParticipants 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."}
• {"criterion_text":"-4)\tStudy-medication related a.\tParticipants who are not able to take oral medications. b.\tParticipants who have a history of hypersensitivity to racemic baclofen. c.\tParticipants with rare hereditary problems of galactose intolerance, the lactase deficiency or glucose-galactose malabsorption should not take this medicine. d.\tActive peptic ulceration as Baclofen stimulates gastric acid secretion. e.\tPorphyria."}
• {"criterion_text":"-5)\tOther exclusion criteria a.\tParticipants who are currently engaged in illicit drug use or alcohol abuse, according to DSM-5 criteria. b.\tParticipants who have previously participated in a clinical trial with arbaclofen (other than our AIMS-2-CT1). c.\tWomen who are breastfeeding."}

Primary endpoints

• {"endpoint_text":"-Change in power in the low frequency bands (theta/alpha) (between visits 1 and 2).","definition_or_measurement_approach":"Change in theta/alpha power between visits 1 and 2, measured using EEG-derived power in low frequency bands."}

Secondary endpoints

• {"endpoint_text":"-Latency of N170 change (between visits 1 and 2)","definition_or_measurement_approach":"Change in latency of the N170 event-related potential between visits 1 and 2, measured using EEG/ERP recordings."}

France

Earliest CTIS Part Ii Submission Date

15-07-2024

Latest Decision Or Authorization Date

23-01-2026

Processing Time Days

557

Number Of Sites

1

Number Of Participants

20

Spain

Earliest CTIS Part Ii Submission Date

11-09-2024

Latest Decision Or Authorization Date

23-01-2026

Processing Time Days

499

Number Of Sites

4

Number Of Participants

85

Primary sponsor

Full Name

Fundacion Para La Investigacion Biomedica Del Hospital Gregorio Maranon

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Spain