Nctid:
NCT06614569
Payload:
{"hasResults"=>false, "derivedSection"=>{"miscInfoModule"=>{"versionHolder"=>"2024-10-04"}, "conditionBrowseModule"=>{"meshes"=>[{"id"=>"D000005733", "term"=>"Gangliosidoses"}, {"id"=>"D000013661", "term"=>"Tay-Sachs Disease"}, {"id"=>"D000020143", "term"=>"Gangliosidoses, GM2"}, {"id"=>"D000012497", "term"=>"Sandhoff Disease"}], "ancestors"=>[{"id"=>"D000013106", "term"=>"Sphingolipidoses"}, {"id"=>"D000020140", "term"=>"Lysosomal Storage Diseases, Nervous System"}, {"id"=>"D000020739", "term"=>"Brain Diseases, Metabolic, Inborn"}, {"id"=>"D000001928", "term"=>"Brain Diseases, Metabolic"}, {"id"=>"D000001927", "term"=>"Brain Diseases"}, {"id"=>"D000002493", "term"=>"Central Nervous System Diseases"}, {"id"=>"D000009422", "term"=>"Nervous System Diseases"}, {"id"=>"D000008661", "term"=>"Metabolism, Inborn Errors"}, {"id"=>"D000030342", "term"=>"Genetic Diseases, Inborn"}, {"id"=>"D000008064", "term"=>"Lipidoses"}, {"id"=>"D000008052", "term"=>"Lipid Metabolism, Inborn Errors"}, {"id"=>"D000016464", "term"=>"Lysosomal Storage Diseases"}, {"id"=>"D000008659", "term"=>"Metabolic Diseases"}, {"id"=>"D000052439", "term"=>"Lipid Metabolism Disorders"}], "browseLeaves"=>[{"id"=>"M8850", "name"=>"Gangliosidoses", "asFound"=>"Gangliosidosis", "relevance"=>"HIGH"}, {"id"=>"M16433", "name"=>"Tay-Sachs Disease", "asFound"=>"Tay-Sachs Disease", "relevance"=>"HIGH"}, {"id"=>"M21978", "name"=>"Gangliosidoses, GM2", "asFound"=>"GM2 Gangliosidosis", "relevance"=>"HIGH"}, {"id"=>"M15316", "name"=>"Sandhoff Disease", "asFound"=>"Sandhoff Disease", "relevance"=>"HIGH"}, {"id"=>"M15904", "name"=>"Sphingolipidoses", "relevance"=>"LOW"}, {"id"=>"M18871", "name"=>"Lysosomal Storage Diseases", "relevance"=>"LOW"}, {"id"=>"M5204", "name"=>"Brain Diseases", "relevance"=>"LOW"}, {"id"=>"M5205", "name"=>"Brain Diseases, Metabolic", "relevance"=>"LOW"}, {"id"=>"M22498", "name"=>"Brain Diseases, Metabolic, Inborn", "relevance"=>"LOW"}, {"id"=>"M5742", "name"=>"Central Nervous System Diseases", "relevance"=>"LOW"}, {"id"=>"M11641", "name"=>"Metabolism, Inborn Errors", "relevance"=>"LOW"}, {"id"=>"M23686", "name"=>"Genetic Diseases, Inborn", "relevance"=>"LOW"}, {"id"=>"M11064", "name"=>"Lipidoses", "relevance"=>"LOW"}, {"id"=>"M11054", "name"=>"Lipid Metabolism, Inborn Errors", "relevance"=>"LOW"}, {"id"=>"M11639", "name"=>"Metabolic Diseases", "relevance"=>"LOW"}, {"id"=>"M27029", "name"=>"Lipid Metabolism Disorders", "relevance"=>"LOW"}, {"id"=>"T2433", "name"=>"Gangliosidosis", "asFound"=>"Gangliosidosis", "relevance"=>"HIGH"}, {"id"=>"T5588", "name"=>"Tay-Sachs Disease", "asFound"=>"Tay-Sachs Disease", "relevance"=>"HIGH"}, {"id"=>"T5103", "name"=>"Sandhoff Disease", "asFound"=>"Sandhoff Disease", "relevance"=>"HIGH"}, {"id"=>"T5335", "name"=>"Sphingolipidosis", "relevance"=>"LOW"}], "browseBranches"=>[{"name"=>"Nervous System Diseases", "abbrev"=>"BC10"}, {"name"=>"Diseases and Abnormalities at or Before Birth", "abbrev"=>"BC16"}, {"name"=>"Nutritional and Metabolic Diseases", "abbrev"=>"BC18"}, {"name"=>"All Conditions", "abbrev"=>"All"}, {"name"=>"Rare Diseases", "abbrev"=>"Rare"}]}}, "protocolSection"=>{"designModule"=>{"bioSpec"=>{"retention"=>"SAMPLES_WITH_DNA", "description"=>"May possibly retain frozen blood samples."}, "studyType"=>"OBSERVATIONAL", "designInfo"=>{"timePerspective"=>"PROSPECTIVE", "observationalModel"=>"COHORT"}, "enrollmentInfo"=>{"type"=>"ESTIMATED", "count"=>7}, "patientRegistry"=>false}, "statusModule"=>{"overallStatus"=>"ACTIVE_NOT_RECRUITING", "startDateStruct"=>{"date"=>"2024-09-17", "type"=>"ACTUAL"}, "expandedAccessInfo"=>{"hasExpandedAccess"=>false}, "statusVerifiedDate"=>"2024-09", "completionDateStruct"=>{"date"=>"2028-12", "type"=>"ESTIMATED"}, "lastUpdateSubmitDate"=>"2024-09-25", "studyFirstSubmitDate"=>"2024-09-23", "studyFirstSubmitQcDate"=>"2024-09-25", "lastUpdatePostDateStruct"=>{"date"=>"2024-09-26", "type"=>"ACTUAL"}, "studyFirstPostDateStruct"=>{"date"=>"2024-09-26", "type"=>"ACTUAL"}, "primaryCompletionDateStruct"=>{"date"=>"2028-12", "type"=>"ESTIMATED"}}, "outcomesModule"=>{"primaryOutcomes"=>[{"measure"=>"Long term Safety of AXO-AAV-GM2", "timeFrame"=>"Every 6 months, up to five years", "description"=>"Participants will be monitored for adverse events, including delayed adverse events, as a result of AXO-AAV-GM2 including de novo cancer, neurologic, rheumatologic, and hematologic/immunologic disorders."}], "secondaryOutcomes"=>[{"measure"=>"Long term Impact of AXO-AAV-GM2", "timeFrame"=>"Every 6 months, up to five years", "description"=>"Participants will be monitored for the impact of treatment with AXO-AAV-GM2 on the expected natural history of their condition in the domains of neurocognitive adaptive, developmental, neurological and motor function."}]}, "oversightModule"=>{"isUsExport"=>false, "oversightHasDmc"=>false, "isFdaRegulatedDrug"=>true, "isFdaRegulatedDevice"=>false}, "conditionsModule"=>{"conditions"=>["GM2 Gangliosidosis", "Tay Sachs Disease", "Sandhoff Disease"]}, "descriptionModule"=>{"briefSummary"=>"This study is to continue Long-Term Follow-Up of Patients who were previously treated with AXO-AAV-GM2 Gene Therapy as treatment for Tay-Sachs or Sandhoff Disease to follow the subjects through 5 years after their initial gene therapy treatment.", "detailedDescription"=>"Subjects will be contacted bi-annually for a total of five years following the administration of AXO-AAV-GM2 for the collection of clinical information, especially pertaining to de novo cancer, neurologic, rheumatologic, and hematologic/immunologic disorders. This will be done to comply with FDA Recommendations and NIH Guidelines for long-term follow-up for research involving gene therapy with AAV Vectors."}, "eligibilityModule"=>{"sex"=>"ALL", "stdAges"=>["CHILD", "ADULT", "OLDER_ADULT"], "samplingMethod"=>"NON_PROBABILITY_SAMPLE", "studyPopulation"=>"Subjects with Tay-Sachs or Sandhoff Disease who were previously treated with AXO-AAV-GM2", "healthyVolunteers"=>false, "eligibilityCriteria"=>"Inclusion Criteria:\n\n* Subjects who were previously enrolled in the study \"A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal thalamic and intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease.\"\n\nExclusion Criteria:\n\n* Subjects who were not previously enrolled under the initial protocol."}, "identificationModule"=>{"nctId"=>"NCT06614569", "briefTitle"=>"Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease", "organization"=>{"class"=>"OTHER", "fullName"=>"University of Massachusetts, Worcester"}, "officialTitle"=>"Long-Term Follow-Up of A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease", "orgStudyIdInfo"=>{"id"=>"STUDY00001894"}}, "armsInterventionsModule"=>{"armGroups"=>[{"label"=>"Subjects previously treated with AXO-AAV-GM2 gene therapy for Tay-Sachs or Sandhoff Disease", "description"=>"All subjects have previously received gene therapy and will be followed for the collection of clinical information, especially pertaining to de novo cancer, neurologic, rheumatologic, and hematologic/immunologic disorders following AAV gene therapy.", "interventionNames"=>["Drug: AXO-AAV-GM2"]}], "interventions"=>[{"name"=>"AXO-AAV-GM2", "type"=>"DRUG", "description"=>"Participants who received AXO-AAV-GM2 will be tracked in long term follow up", "armGroupLabels"=>["Subjects previously treated with AXO-AAV-GM2 gene therapy for Tay-Sachs or Sandhoff Disease"]}]}, "contactsLocationsModule"=>{"locations"=>[{"zip"=>"01655", "city"=>"Worcester", "state"=>"Massachusetts", "country"=>"United States", "facility"=>"UMass Chan Medical School", "geoPoint"=>{"lat"=>42.26259, "lon"=>-71.80229}}], "overallOfficials"=>[{"name"=>"Terence Flotte, MD", "role"=>"PRINCIPAL_INVESTIGATOR", "affiliation"=>"UMass Chan Medical School"}]}, "ipdSharingStatementModule"=>{"ipdSharing"=>"NO"}, "sponsorCollaboratorsModule"=>{"leadSponsor"=>{"name"=>"Terence Flotte", "class"=>"OTHER"}, "responsibleParty"=>{"type"=>"SPONSOR_INVESTIGATOR", "investigatorTitle"=>"Professor and Dean", "investigatorFullName"=>"Terence Flotte", "investigatorAffiliation"=>"University of Massachusetts, Worcester"}}}}
