Ignite Creation Date:
2025-12-24 @ 6:33 PM
Ignite Modification Date:
2026-01-21 @ 5:13 PM
Study NCT ID:
NCT06087757
Status:
RECRUITING
Last Update Posted:
2024-05-07
First Post:
2023-10-05
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Clemastine Treatment in Individuals With Williams Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D018980', 'term': 'Williams Syndrome'}], 'ancestors': [{'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D021921', 'term': 'Aortic Stenosis, Supravalvular'}, {'id': 'D001024', 'term': 'Aortic Valve Stenosis'}, {'id': 'D000082862', 'term': 'Aortic Valve Disease'}, {'id': 'D006349', 'term': 'Heart Valve Diseases'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR'], 'maskingDescription': 'This study is an open-label study with a blinded randomized withdrawal'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-04-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-05', 'completionDateStruct': {'date': '2025-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-05-06', 'studyFirstSubmitDate': '2023-10-05', 'studyFirstSubmitQcDate': '2023-10-11', 'lastUpdatePostDateStruct': {'date': '2024-05-07', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-10-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Neurocognitive measures', 'timeFrame': 'December 2024', '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.\n\nNIH 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/'}], 'secondaryOutcomes': [{'measure': 'Motor and Neurological assessment. Motor and Neurological assessment. Motor and Neurological assessment. Motor and neurolocgial measures', 'timeFrame': 'December 2024', 'description': 'The Beery-Buktenica Developmental Test of Visual-Motor Integration\n\nGAITRite walkway: GAITRite is a sensitive walkway measuring temporal and spatial parameters.\n\nPsychiatric evaluation: a semi-structured interview for neuropsychiatric diagnoses with the Schedule for Affective Disorders and Schizophrenia for School-Aged Children.\n\nThe Child Behavior Checklist: measures behavioral/emotional problems and competencies.\n\nAttention Deficit Hyperactivity Disorder Rating Scale.\n\nThe Screen for Child Anxiety-Related Emotional Disorders.\n\nThe Social Responsiveness Scale-Second Edition.\n\nAdaptive Behavior Assessment System-2nd Edition.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Clemastine', 'Cognition', 'Myelination abnormalities'], 'conditions': ['Williams Syndrome']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '30 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Individuals with Williams syndrome, which has been confirmed by genetic testing.\n* Ages 6-30.\n* Normal values in safety variables (e.g. Normal ECG 120-129/80-84).\n* No change in psychotropic medications and dosage during the last 4 weeks.\n* During the study, no pharmacological change that may impact the study (e.g. ADHD\n* medications).\n\nExclusion Criteria:\n\n* Individuals with another genetic disorder besides Williams syndrome.\n* Individuals with Williams syndrome, younger than 6 or older than 30 years old.\n* Significant change in normal values in safety variables (e.g. high or low ECG).\n* Change in medications and dosage during the last 4 weeks prior the beginning and\n* during the study.\n* Pregnancy.\n* Using addictive substances such as alcohol.'}, 'identificationModule': {'nctId': 'NCT06087757', 'briefTitle': 'Clemastine Treatment in Individuals With Williams Syndrome', 'organization': {'class': 'OTHER_GOV', 'fullName': 'Sheba Medical Center'}, 'officialTitle': 'Clemastine Treatment in Individuals With Williams Syndrome- a Double-blind Placebo Control to Assess the Safety and Efficacy', 'orgStudyIdInfo': {'id': '9011-21-SMC'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Open Label', 'interventionNames': ['Drug: Open Label Clemastine with a blinded randomize withdrawal']}, {'type': 'EXPERIMENTAL', 'label': 'Blinded randomize withdrawal', 'interventionNames': ['Drug: Open Label Clemastine with a blinded randomize withdrawal']}], 'interventions': [{'name': 'Open Label Clemastine with a blinded randomize withdrawal', 'type': 'DRUG', '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.', 'armGroupLabels': ['Blinded randomize withdrawal', 'Open Label']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Ramat Gan', 'status': 'RECRUITING', 'country': 'Israel', 'contacts': [{'name': 'Prof. Doron Gothelf, MD', 'role': 'CONTACT', 'email': 'gothelf@post.tau.ac.il', 'phone': '972-52-6669360'}], 'facility': 'Child and Adolescent Psychiatry Unit, Sheba Medical Center', 'geoPoint': {'lat': 32.08227, 'lon': 34.81065}}, {'city': 'Tel Aviv', 'status': 'RECRUITING', 'country': 'Israel', 'contacts': [{'name': 'Doron Gothelf', 'role': 'CONTACT', 'email': 'gothelf@tauex.tau.ac.il', 'phone': '972-54-666-9360'}], 'facility': 'Tel Aviv University', 'geoPoint': {'lat': 32.08088, 'lon': 34.78057}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sheba Medical Center', 'class': 'OTHER_GOV'}, 'collaborators': [{'name': 'Tel Aviv University', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Head of The Child and Adolescent Psychiatry Unit', 'investigatorFullName': 'Prof. Doron Gothelf MD', 'investigatorAffiliation': 'Sheba Medical Center'}}}}