Clemastine Treatment in Individuals With Williams Syndrome
- Conditions
- Williams Syndrome
- Interventions
- Registration Number
- NCT06087757
- Lead Sponsor
- Sheba Medical Center
- Brief Summary
This study explores the neurobiological etiology of Williams syndrome and potential therapeutic targets for associated social, motor, and cognitive abnormalities. The main translational objective will be to test the effectiveness of Clemasntine on neurocognitive and other associated abnormalities in individuals with Williams syndrome.
- Detailed Description
The overarching aim of the research proposed is to examine the safty of Clemastine use for individuals with Williams syndrome. Further, we wish to examine the relationship between Clemastine treatment and cognitive, motor and behavioral deficits in individuals with Williams syndrome. This study is an open-label study with a blinded randomize withdrawal. Each participant will go through a baseline evaluation (see study outcomes) and will be treated with the FDA approved drug Clemastine, in an age dependent dose (dosage table below). After a substantial improvement will be noticed, the participants will be divided into 2 groups, in a randomized, double-blind, placebo-control study design.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 30
- Individuals with Williams syndrome, which has been confirmed by genetic testing.
- Ages 6-30.
- Normal values in safety variables (e.g. Normal ECG 120-129/80-84).
- No change in psychotropic medications and dosage during the last 4 weeks.
- During the study, no pharmacological change that may impact the study (e.g. ADHD
- medications).
- Individuals with another genetic disorder besides Williams syndrome.
- Individuals with Williams syndrome, younger than 6 or older than 30 years old.
- Significant change in normal values in safety variables (e.g. high or low ECG).
- Change in medications and dosage during the last 4 weeks prior the beginning and
- during the study.
- Pregnancy.
- Using addictive substances such as alcohol.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description Open Label Open Label Clemastine with a blinded randomize withdrawal - Blinded randomize withdrawal Open Label Clemastine with a blinded randomize withdrawal -
- Primary Outcome Measures
Name Time Method Neurocognitive measures December 2024 Wechsler Intelligence Scale: The standard age-appropriate Wechsler scale. Wechsler Intelligence Scale for Children, 4th edition(Wechsler 1991, Wechsler 2003) for subjects 17 years and younger and the Wechsler Adult Intelligence Scale, 3rd edition (WAIS III)(Wechsler 1997) for subjects older than 17 years.
NIH ToolBox (NIHTB): the NIHTB is a computerized neurocognitive battery developed by NIH that was designed to include measures of cognitive flexibility and inhibitory control, as well as a measure of working memory. http://www.healthmeasures.net/explore-measurement-systems/nih-toolbox/
- Secondary Outcome Measures
Name Time Method Motor and Neurological assessment. Motor and Neurological assessment. Motor and Neurological assessment. Motor and neurolocgial measures December 2024 The Beery-Buktenica Developmental Test of Visual-Motor Integration
GAITRite walkway: GAITRite is a sensitive walkway measuring temporal and spatial parameters.
Psychiatric evaluation: a semi-structured interview for neuropsychiatric diagnoses with the Schedule for Affective Disorders and Schizophrenia for School-Aged Children.
The Child Behavior Checklist: measures behavioral/emotional problems and competencies.
Attention Deficit Hyperactivity Disorder Rating Scale.
The Screen for Child Anxiety-Related Emotional Disorders.
The Social Responsiveness Scale-Second Edition.
Adaptive Behavior Assessment System-2nd Edition.
Trial Locations
- Locations (2)
Child and Adolescent Psychiatry Unit, Sheba Medical Center
🇮🇱Ramat-Gan, Israel
Tel Aviv University
🇮🇱Tel Aviv, Israel