Ignite Creation Date:
2025-12-24 @ 9:40 PM
Ignite Modification Date:
2026-01-02 @ 8:40 PM
Study NCT ID:
NCT06717932
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-12-05
First Post:
2024-12-01
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D017086', 'term': 'beta-Thalassemia'}], 'ancestors': [{'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 8}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2025-05', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-12', 'completionDateStruct': {'date': '2039-11', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-12-01', 'studyFirstSubmitDate': '2024-12-01', 'studyFirstSubmitQcDate': '2024-12-01', 'lastUpdatePostDateStruct': {'date': '2024-12-05', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-12-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2039-11', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0', 'timeFrame': 'Through 15 years post CS-101 infusion', 'description': 'CommonTerminology Criteria for Adverse Events#CTCAE#has 5 levels of AE determination, increasing in severity as the level increases'}, {'measure': 'Occurrence of all-cause death', 'timeFrame': 'Through 15 years post CS-101 infusion'}, {'measure': 'Occurrence of achieving transfusion independence for at least 12 consecutive months', 'timeFrame': 'Through 15 years post CS-101 infusion'}], 'secondaryOutcomes': [{'measure': 'Change in fetal hemoglobin(HbF) concentration over time', 'timeFrame': 'Through 15 years post CS-101 infusion'}, {'measure': 'Change in total hemoglobin(Hb) concentration over time', 'timeFrame': 'Through 15 years post CS-101 infusion'}, {'measure': 'Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in', 'timeFrame': 'Through 15 years post CS-101 infusion'}, {'measure': 'Chimerism level in bone marrow Proportion of alleles with intended genetic modification in', 'timeFrame': 'Through 15 years post CS-101 infusion'}, {'measure': 'Change in F-Cell concentration over time', 'timeFrame': 'Through 15 years post CS-101 infusion'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Beta-Thalassemia']}, 'descriptionModule': {'briefSummary': 'This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-01 (NCT06291961),No investigational drug product will be administered in the study.', 'detailedDescription': 'This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-01 (NCT06291961). Subjects in the CS-101-01 study will be entered into long-term follow-up of this study up to 15 years post-infusion at the completion of the last (12-month) follow up visit after treatment with CS-101 Injection.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '35 Years', 'minimumAge': '12 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subject with β-thalassemia treated with CS-101 infusion in CS-101-01 study', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participants must have received CS-101 infusion in CS-101-01 study\n* Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form\n\nExclusion Criteria:\n\n* There are no exclusion criteria'}, 'identificationModule': {'nctId': 'NCT06717932', 'briefTitle': 'A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection', 'organization': {'class': 'INDUSTRY', 'fullName': 'CorrectSequence Therapeutics Co., Ltd'}, 'officialTitle': 'A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With CS-101 Transplantation', 'orgStudyIdInfo': {'id': 'CS-101-05'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Safety and efficacy assessments', 'description': 'Safety evaluations,disease-specific assessments,and assessments to monitor for long-term complications of autologous transplant'}]}, 'contactsLocationsModule': {'locations': [{'city': 'Nanning', 'country': 'China', 'facility': 'The First Affiliated Hospital of Guangxi Medical University', 'geoPoint': {'lat': 22.81667, 'lon': 108.31667}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'CorrectSequence Therapeutics Co., Ltd', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}