Clemastine Treatment in Individuals With Williams Syndrome

Sponsor

Sheba Medical Center (Other)

Overall Status

Recruiting

CT.gov ID

NCT06087757

Collaborator

Tel Aviv University (Other)

Enrollment

Locations

Arms

Anticipated Duration (Months)

Patients Per Site

0.6

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Williams Syndrome	Drug: Open Label Clemastine with a blinded randomize withdrawal	Phase 3

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

30 participants

Allocation:

Randomized

Intervention Model:

Crossover Assignment

Masking:

Double (Participant, Investigator)

Masking Description:

This study is an open-label study with a blinded randomized withdrawal

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Oct 1, 2023

Anticipated Primary Completion Date :

Dec 1, 2024

Anticipated Study Completion Date :

Dec 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Open Label	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.
Experimental: Blinded randomize withdrawal	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.

Arm

Intervention/Treatment

Experimental: Open Label

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.

Experimental: Blinded randomize withdrawal

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.

Outcome Measures

Primary Outcome Measures

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

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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 30 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Child and Adolescent Psychiatry Unit, Sheba Medical Center	Ramat-Gan		Israel
2	Tel Aviv University	Tel Aviv		Israel

Sponsors and Collaborators

Sheba Medical Center
Tel Aviv University

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Prof. Doron Gothelf MD, Head of The Child and Adolescent Psychiatry Unit, Sheba Medical Center

ClinicalTrials.gov Identifier:

NCT06087757

Other Study ID Numbers:

9011-21-SMC

First Posted:

Oct 18, 2023

Last Update Posted:

Oct 18, 2023

Last Verified:

Oct 1, 2023

Individual Participant Data (IPD) Sharing Statement:

Undecided

Plan to Share IPD:

Undecided

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Product Manufactured in and Exported from the U.S.:

Keywords provided by Prof. Doron Gothelf MD, Head of The Child and Adolescent Psychiatry Unit, Sheba Medical Center

Clemastine

Cognition

Myelination abnormalities

Additional relevant MeSH terms:

Williams Syndrome

Syndrome

Clemastine

Study Results

No Results Posted as of Oct 18, 2023