Clemastine Treatment in Individuals With Williams Syndrome

Sheba Medical Center

Status and phase

Enrolling

Phase 3

Conditions

Williams Syndrome

Treatments

Drug: Open Label Clemastine with a blinded randomize withdrawal

Study type

Interventional

Funder types

Other

Identifiers

NCT06087757

9011-21-SMC

Details and patient eligibility

About

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.

Full 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.

Enrollment

30 estimated patients

Sex

All

Ages

6 to 30 years old

Volunteers

No 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.