Ignite Creation Date:
2025-12-25 @ 5:19 AM
Ignite Modification Date:
2025-12-26 @ 4:26 AM
Study NCT ID:
NCT03201627
Status:
UNKNOWN
Last Update Posted:
2019-09-24
First Post:
2017-06-26
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D052556', 'term': 'Niemann-Pick Disease, Type C'}], 'ancestors': [{'id': 'D009542', 'term': 'Niemann-Pick Diseases'}, {'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'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': 'D015616', 'term': 'Histiocytosis, Non-Langerhans-Cell'}, {'id': 'D015614', 'term': 'Histiocytosis'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D016651', 'term': 'Lithium Carbonate'}], 'ancestors': [{'id': 'D002254', 'term': 'Carbonates'}, {'id': 'D000468', 'term': 'Alkalies'}, {'id': 'D007287', 'term': 'Inorganic Chemicals'}, {'id': 'D002255', 'term': 'Carbonic Acid'}, {'id': 'D017554', 'term': 'Carbon Compounds, Inorganic'}, {'id': 'D018020', 'term': 'Lithium Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 18}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2017-07-05', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-09', 'completionDateStruct': {'date': '2021-06-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2019-09-21', 'studyFirstSubmitDate': '2017-06-26', 'studyFirstSubmitQcDate': '2017-06-26', 'lastUpdatePostDateStruct': {'date': '2019-09-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-06-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2020-09-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'NPC clinical severity score', 'timeFrame': '52 weeks'}], 'secondaryOutcomes': [{'measure': '7-ketocholesterol', 'timeFrame': '52 weeks'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Niemann-Pick Disease, Type C1']}, 'referencesModule': {'references': [{'pmid': '34177581', 'type': 'DERIVED', 'citation': 'Han S, Zhang H, Yi M, Liu X, Maegawa GHB, Zou Y, Wang Q, Wu D, Ye Z. Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1. Front Pharmacol. 2021 Jun 9;12:667361. doi: 10.3389/fphar.2021.667361. eCollection 2021.'}]}, 'descriptionModule': {'briefSummary': 'This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '40 Years', 'minimumAge': '7 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. aged from 7 years to 40 years\n2. onset of neurological symptoms prior to 15 years of age\n3. confirmed diagnosis of NPC1 determined by either two NPC1 mutations or one NPC1 mutation with elevated plasma 7-ketocholesterol\n4. ability to walk either independently or with assistance\n5. Subject or parent/guardian able to communicate with investigator, understand and abide the rules of this clinical trial.\n6. parent/guardian able to accompany the subjects to participate in the clinical trial\n7. Subject or parent/guardian must provide written informed consent\n\nExclusion Criteria:\n\n1. One NPC clinical severity scale score reached 5\n2. Female in pregnancy or breastfeeding\n3. The predicted life span less than 1 year\n4. Severe liver insufficiency (defined as hepatic laboratory parameters, AST and/or ALT greater than three-times the upper limit of normal\n5. Renal insufficiency, with the serum creatine greater than 1.5 times the upper limit of normal\n6. Free with neurological symptoms\n7. Take antidiuretic hormone'}, 'identificationModule': {'nctId': 'NCT03201627', 'briefTitle': 'Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease', 'organization': {'class': 'OTHER', 'fullName': 'Xinhua Hospital, Shanghai Jiao Tong University School of Medicine'}, 'officialTitle': 'a Single-center, Prospective, Open, and Non-randomized Case-control Study of Lithium Carbonate Effect on Niemann Disease C1 Type', 'orgStudyIdInfo': {'id': 'XH-17-010'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'treatment', 'interventionNames': ['Drug: Lithium Carbonate']}], 'interventions': [{'name': 'Lithium Carbonate', 'type': 'DRUG', 'description': 'To observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1', 'armGroupLabels': ['treatment']}]}, 'contactsLocationsModule': {'locations': [{'zip': '200092', 'city': 'Shanghai', 'state': 'Shanghai Municipality', 'country': 'China', 'facility': 'Xin Hua Hospital', 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Xinhua Hospital, Shanghai Jiao Tong University School of Medicine', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Associate Prof.', 'investigatorFullName': 'Huiwen Zhang, MD', 'investigatorAffiliation': 'Xinhua Hospital, Shanghai Jiao Tong University School of Medicine'}}}}