Ignite Creation Date:
2025-12-24 @ 5:08 PM
Ignite Modification Date:
2025-12-27 @ 5:37 AM
Study NCT ID:
NCT01942850
Status:
COMPLETED
Last Update Posted:
2014-12-31
First Post:
2013-09-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
International Ataxia Rating Scale in Younger Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D001260', 'term': 'Ataxia Telangiectasia'}], 'ancestors': [{'id': 'D020754', 'term': 'Spinocerebellar Ataxias'}, {'id': 'D002524', 'term': 'Cerebellar Ataxia'}, {'id': 'D002526', 'term': 'Cerebellar Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020752', 'term': 'Neurocutaneous Syndromes'}, {'id': 'D001259', 'term': 'Ataxia'}, {'id': 'D020820', 'term': 'Dyskinesias'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D013684', 'term': 'Telangiectasis'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D049914', 'term': 'DNA Repair-Deficiency Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 63}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-08'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-12', 'completionDateStruct': {'date': '2014-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-12-30', 'studyFirstSubmitDate': '2013-09-06', 'studyFirstSubmitQcDate': '2013-09-11', 'lastUpdatePostDateStruct': {'date': '2014-12-31', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2013-09-16', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-05', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Correlation Analysis', 'timeFrame': 'single visit', 'description': 'The primary outcome is validity of ICARS in children younger than 10 years of age (by comparing the mean scores and subscores between children aged 6-10 years of age to those aged 10-18 years of age).'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Ataxia Telangiectasia']}, 'descriptionModule': {'briefSummary': 'The project will collect information on the mapping of clinical ratings on a number of scales that are used in the assessment of patients with ataxias.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '6 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Known patients with ataxia/ataxia telangiectasia', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion criteria\n\n1. Patients aged 6-18 years with diagnosed with ataxia, preferably AT will be included. The diagnosis of AT will be made based on the criteria: typical clinical picture plus one of the following: 1. a proven mutation in the ATM gene 2. Deficient ATM protein proven by Western blotting 3. Elevated α-fetoprotein, cerebellar atrophy on MRI and immune deficiency/ chromosomal breakage/ T- cell lymphoreticular malignancy.\n2. Children aged 6-18 years with suspected AT (fulfilling partially the above criteria will be included)\n3. Children aged 6-18 years with AT like disease, with or without proven mutation in the MRE11 gene will be included\n4. Children aged 6-18 years of age with ataxia of other known or unknown etiologies might be included, but their total number should not exceed 20% of the cohort in each site'}, 'identificationModule': {'nctId': 'NCT01942850', 'briefTitle': 'International Ataxia Rating Scale in Younger Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Quince Therapeutics S.p.A.'}, 'officialTitle': 'APPLICABILITY OF THE INTERNATIONAL ATAXIA RATING SCALE (ICARS) IN YOUNGER PATIENTS AND DEVELOPMENT OF GLOBAL RATING INSTRUMENTS FOR PATIENTS WITH ATAXIA TELANGIECTASIA (AT)', 'orgStudyIdInfo': {'id': 'ICARS-CGI-Ery01-2013'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'No Treatment', 'description': 'Collect pilot data on the performance of the ICARS in patients younger than 10 years of age, as well as to introduce definitions for the various clinically defined stages of AT, and attempt to develop descriptors of change that could help in the assessment of patients longitudinally.'}]}, 'contactsLocationsModule': {'locations': [{'city': 'Tampa', 'state': 'Florida', 'country': 'United States', 'facility': 'Carol Morsani USF Health Center', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'city': 'Mumbai', 'country': 'India', 'facility': 'Jaslok Hospital', 'geoPoint': {'lat': 19.07283, 'lon': 72.88261}}, {'city': 'Tel Litwinsky', 'country': 'Israel', 'facility': 'Edmond and Lilly Safra Childrens Hospital Pediatric Unit', 'geoPoint': {'lat': 32.05096, 'lon': 34.84588}}, {'zip': '00100', 'city': 'Roma', 'state': 'Italy', 'country': 'Italy', 'facility': 'Università La Sapienza', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'city': 'Brescia', 'country': 'Italy', 'facility': 'A.O Spedalli Civilli Brescia', 'geoPoint': {'lat': 45.53558, 'lon': 10.21472}}], 'overallOfficials': [{'name': 'Luca Benatti, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Quince Therapeutics S.p.A.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Quince Therapeutics S.p.A.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}