Clinical trial to investigate the effects of clonazepam on patients with ARID1B-related intellectual disability.

Phase 1

Active, not recruiting

• Conditions: Therapeutic area: Diseases [C] - Nervous System Diseases [C10]; ARID1B-related intellectual disability

• Registration Number: EUCTR2019-003558-98-NL
• Lead Sponsor: Centre for Human Drug Research

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2020-01-08
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

Part A, saliva PK.
• Healthy male or female volunteers
• 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

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Part A
• 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
• 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified