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Clemastine Treatment in Individuals With Williams Syndrome

Primary Purpose

Williams Syndrome

Status
Recruiting
Phase
Phase 3
Locations
Israel
Study Type
Interventional
Intervention
Open Label Clemastine with a blinded randomize withdrawal
Sponsored by
Sheba Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Williams Syndrome focused on measuring Clemastine, Cognition, Myelination abnormalities

Eligibility Criteria

6 Years - 30 Years (Child, Adult)All SexesDoes not accept 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.

Sites / Locations

  • Child and Adolescent Psychiatry Unit, Sheba Medical CenterRecruiting
  • Tel Aviv UniversityRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Open Label

Blinded randomize withdrawal

Arm Description

Outcomes

Primary Outcome Measures

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/

Secondary Outcome Measures

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.

Full Information

First Posted
October 5, 2023
Last Updated
October 11, 2023
Sponsor
Sheba Medical Center
Collaborators
Tel Aviv University
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1. Study Identification

Unique Protocol Identification Number
NCT06087757
Brief Title
Clemastine Treatment in Individuals With Williams Syndrome
Official Title
Clemastine Treatment in Individuals With Williams Syndrome- a Double-blind Placebo Control to Assess the Safety and Efficacy
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 2023 (Anticipated)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Sheba Medical Center
Collaborators
Tel Aviv University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Williams Syndrome
Keywords
Clemastine, Cognition, Myelination abnormalities

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Masking
ParticipantInvestigator
Masking Description
This study is an open-label study with a blinded randomized withdrawal
Allocation
Randomized
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Open Label
Arm Type
Experimental
Arm Title
Blinded randomize withdrawal
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Open Label Clemastine with a blinded randomize withdrawal
Intervention Description
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.
Primary Outcome Measure Information:
Title
Neurocognitive measures
Description
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/
Time Frame
December 2024
Secondary Outcome Measure Information:
Title
Motor and Neurological assessment. Motor and Neurological assessment. Motor and Neurological assessment. Motor and neurolocgial measures
Description
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.
Time Frame
December 2024

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
Child and Adolescent Psychiatry Unit, Sheba Medical Center
City
Ramat-Gan
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Prof. Doron Gothelf, MD
Phone
972-52-6669360
Email
gothelf@post.tau.ac.il
Facility Name
Tel Aviv University
City
Tel Aviv
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Doron Gothelf
Phone
972-54-666-9360
Email
gothelf@tauex.tau.ac.il

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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Clemastine Treatment in Individuals With Williams Syndrome

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