Ignite Creation Date:
2025-12-25 @ 3:54 AM
Ignite Modification Date:
2025-12-26 @ 2:43 AM
Study NCT ID:
NCT00724802
Status:
UNKNOWN
Last Update Posted:
2011-06-21
First Post:
2008-06-18
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D017825', 'term': 'Canavan Disease'}], 'ancestors': [{'id': 'D020279', 'term': 'Hereditary Central Nervous System Demyelinating Diseases'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D056784', 'term': 'Leukoencephalopathies'}, {'id': 'D003711', 'term': 'Demyelinating Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D014215', 'term': 'Triacetin'}], 'ancestors': [{'id': 'D014280', 'term': 'Triglycerides'}, {'id': 'D005989', 'term': 'Glycerides'}, {'id': 'D008055', 'term': 'Lipids'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 1}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2008-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2011-06', 'lastUpdateSubmitDate': '2011-06-20', 'studyFirstSubmitDate': '2008-06-18', 'studyFirstSubmitQcDate': '2008-07-29', 'lastUpdatePostDateStruct': {'date': '2011-06-21', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2008-07-30', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2008-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'brain MRI at the end of the study', 'timeFrame': '6 months'}]}, 'conditionsModule': {'keywords': ['babies with canavan disease'], 'conditions': ['Canavan Disease']}, 'referencesModule': {'references': [{'pmid': '21474353', 'type': 'DERIVED', 'citation': 'Segel R, Anikster Y, Zevin S, Steinberg A, Gahl WA, Fisher D, Staretz-Chacham O, Zimran A, Altarescu G. A safety trial of high dose glyceryl triacetate for Canavan disease. Mol Genet Metab. 2011 Jul;103(3):203-6. doi: 10.1016/j.ymgme.2011.03.012. Epub 2011 Mar 15.'}]}, 'descriptionModule': {'briefSummary': 'Canavan disease is caused by Aspartoacylase deficiency. There is no treatment for the disease, but there is a food additive that includes acetate . We suggest an early treatment with acetate and a neurologic evaluation, including MRI, after 4 months of treatment. In any case the treatment will be stopped at the age of 22 months, when myelinization is ended.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '18 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* clinical diagnosis of Canavan disease\n\nExclusion Criteria:\n\n* Age above 18 months'}, 'identificationModule': {'nctId': 'NCT00724802', 'briefTitle': 'Oral Glyceryl Triacetate (GTA) in Newborns With Canavan', 'organization': {'class': 'OTHER', 'fullName': 'Shaare Zedek Medical Center'}, 'officialTitle': 'Oral Glyceryl Triacetate (GTA) in Newborns With Canavan', 'orgStudyIdInfo': {'id': 'canavangta'}}, 'armsInterventionsModule': {'interventions': [{'name': 'GTA (Glyceryl triacetate)', 'type': 'DIETARY_SUPPLEMENT', 'otherNames': ['No other names are available for this drug'], 'description': '0.5 gr/kg x2/day syrup two times a day with increasing dose each 3 days till max 5gr /kg each day for 6 months'}, {'name': 'GTA glyceryl triacetate', 'type': 'DRUG', 'description': '0.5 gr/kg twice a day with increase of 0.5 gr/kg every 3 days up to 5 gr/kg'}]}, 'contactsLocationsModule': {'locations': [{'zip': '91031', 'city': 'Jerusalem', 'status': 'RECRUITING', 'country': 'Israel', 'contacts': [{'name': 'Gheona Alterescu, MD', 'role': 'CONTACT', 'email': 'gheona@szmc.org.il', 'phone': '972-26666435'}, {'name': 'Gheona Alterescu, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Shaare Zedek Medical Center', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}], 'centralContacts': [{'name': 'Gheona Alterescu, MD', 'role': 'CONTACT', 'email': 'gheona@szmc.org.il', 'phone': '972-2-6666435'}], 'overallOfficials': [{'name': 'Gheona Alterescu, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Shaare Zedek Medical Center'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Shaare Zedek Medical Center', 'class': 'OTHER'}, 'responsibleParty': {'oldNameTitle': 'Dr. Gheona Alteresco', 'oldOrganization': 'Shaare Zedek Medical Center'}}}}