Clemastine Treatment in Individuals With Williams Syndrome

NCT ID: NCT06087757

Last Updated: 2024-05-07

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-04-01
• Study Completion Date: 2025-12-31

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.

Conditions

Williams Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Open Label

Group Type: EXPERIMENTAL

Open Label Clemastine with a blinded randomize withdrawal

Intervention Type: DRUG

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.

Blinded randomize withdrawal

Group Type: EXPERIMENTAL

Open Label Clemastine with a blinded randomize withdrawal

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tel Aviv University

OTHER

Sponsor Role: collaborator

Sheba Medical Center

OTHER_GOV

Sponsor Role: lead

Responsible Party

Prof. Doron Gothelf MD

Head of The Child and Adolescent Psychiatry Unit

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Child and Adolescent Psychiatry Unit, Sheba Medical Center

Ramat Gan, , Israel

Site Status: RECRUITING

Tel Aviv University

Tel Aviv, , Israel

Site Status: RECRUITING

Countries

Israel

Facility Contacts

Prof. Doron Gothelf, MD

Role: primary

gothelf@post.tau.ac.il

972-52-6669360

Doron Gothelf

Role: primary

gothelf@tauex.tau.ac.il

972-54-666-9360

Other Identifiers

9011-21-SMC

Identifier Type: -

Identifier Source: org_study_id