Clemastine Treatment in Individuals With Williams Syndrome

Williams Syndrome Clinical Trial

Official title:

Clemastine Treatment in Individuals With Williams Syndrome- a Double-blind Placebo Control to Assess the Safety and Efficacy

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06087757
• Other study ID #: 9011-21-SMC
• Status: Recruiting
• Phase: Phase 3
• Start date: April 1, 2024
• Est. completion date: December 2025

Study information

• Verified date: May 2024
• Source: Sheba Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: December 2025
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 30 Years

Eligibility

Inclusion Criteria:

• 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).

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Syndrome
• Williams Syndrome

Intervention

Drug:
• Open Label Clemastine with a blinded randomize withdrawal
Clemastine will be given in doses as the maximum dose recommended for allergy condition. Age 6-12 years 4.02 mg/day, age 12-30 years 8.04 mg/day.

Locations

Country	Name	City	State
Israel	Child and Adolescent Psychiatry Unit, Sheba Medical Center	Ramat-Gan
Israel	Tel Aviv University	Tel Aviv

Sponsors (2)

Lead Sponsor	Collaborator
Sheba Medical Center	Tel Aviv University

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Neurocognitive measures	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/	December 2024
Secondary	Motor and Neurological assessment. Motor and Neurological assessment. Motor and Neurological assessment. Motor and neurolocgial measures	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.	December 2024