Randomized, double-blind, placebo-controlled, two way crossover, single centre study evaluating the acute and chronic effect of clonazepam on cognitive tests and patient-reported outcome measures in patients with ARID1B-related intellectual disability

Phase 4

Completed

• Conditions: ARID1B syndrome; ARID1B-related intellectual disability; 10057167

• Registration Number: NL-OMON52899
• Lead Sponsor: Centre for Human Drug Research

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 11 June 2024

Recruitment & Eligibility

• Status: Completed
• 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

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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified