Clonazepam in ARID1B Evaluation (CARE study)

Recruitment & Eligibility

Status: Pending

Sex: Not specified

Target Recruitment: 40

Inclusion Criteria

Part A, correlation blood-saliva PK.
• Healthy male or female volunteers aged 18-30 years
• Informed consent provided by volunteer

Part B: ARID1B patients.
• Informed consent provided by both parents, or the legal guardian prior to any study mandated procedure.
• Known mutation in ARID1B
• Assent provided by the participant.
• Aged 6 years or older
• Able to perform at least 5 of the 6 NeuroCart® activities.

Exclusion Criteria

Part A, healthy volunteers
• Disorder that could interfere with saliva production.
• Known hypersensitivity to clonazepam, other benzodiazepines or other excipients of the study medication.
• Treatment with another investigational drug within 3 months prior to screening or more than 4 times a year.
• History or clinical evidence of any disease and/or existence of a surgical or medical condition which might interfere with the absorption, distribution, metabolism or excretion of the study drug.
• History of severe respiratory problems or severe liver- or renal insufficiency.
• Other medical or psychosocial history making the participant unsuitable for participation as determined by the treating paediatrician.
• History or clinical evidence of alcoholism within the 3-year period prior to screening (i.e. regular use of more than 21 units of alcohol/week).
• Clinically significant findings on physical examination.
• Medications with a strong influence on CYP3A4 metabolism
• Clinically meaningful blood loss (including blood donation), or a transfusion of any blood product within 12 weeks before screening.
• Subjects with a BMI > 30 and/or cardiovascular, respiratory or immune system disorders

Part B: ARID1B patients.
• Clear indication of not wanting to participate during the study
• Use of benzodiazepines or any other medication or drug with the potential to influence study related endpoints in the investigator’s opinion (including e.g. CYP3A4-related drugs).
• Known hypersensitivity to clonazepam, other benzodiazepines or other excipients of the study medication.
• History of severe respiratory problems or severe liver- or renal insufficiency.
• Other medical or psychosocial history making the participant unsuitable for participation as determined by the treating paediatrician.