Ignite Creation Date:
2025-12-25 @ 3:30 AM
Ignite Modification Date:
2025-12-26 @ 2:11 AM
Study NCT ID:
NCT05951205
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-07-02
First Post:
2023-06-23
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}], 'ancestors': [{'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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000729927', 'term': 'exagamglogene autotemcel'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 12}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2027-07-31', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-06', 'completionDateStruct': {'date': '2033-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-06-27', 'studyFirstSubmitDate': '2023-06-23', 'studyFirstSubmitQcDate': '2023-07-14', 'lastUpdatePostDateStruct': {'date': '2025-07-02', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-07-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2033-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of Participants with an Average Fetal Hemoglobin (HbF) Greater Than or Equal To (>=) 20 percent (%) on or After 6 Months', 'timeFrame': 'From 60 Days after Last Red Blood Cell (RBC) transfusion up to 24 Months after exa-cel infusion'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'From Signing of Informed Consent up to 24 Months After exa-cel Infusion'}, {'measure': 'Proportion of Participants With Neutrophil Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count (ANC) >=500 per Microliter [mcgL] on 3 Different Days)', 'timeFrame': 'Within 42 Days After exa-cel Infusion'}, {'measure': 'Time to Neutrophil Engraftment', 'timeFrame': 'Up to 24 months After exa-cel Infusion'}, {'measure': 'Time to Platelet Engraftment', 'timeFrame': 'Up to 24 months After exa-cel Infusion'}, {'measure': 'Incidence of Transplant-Related Mortality (TRM)', 'timeFrame': 'Up to 100 Days After exa-cel Infusion'}, {'measure': 'Incidence of Transplant-Related Mortality (TRM)', 'timeFrame': 'Within 12 Months After exa-cel Infusion'}, {'measure': 'Incidence of All-cause Mortality', 'timeFrame': 'From Signing of Informed Consent up to 24 Months After exa-cel Infusion'}, {'measure': 'Proportion of Participants With No Severe Vaso-Occlusive Crises (VOCs) for At least 12 Months (VF12)', 'timeFrame': 'From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion'}, {'measure': 'Proportion of Participants Free from Inpatient Hospitalization For Severe VOCs Sustained for At least 12 Months (HF12)', 'timeFrame': 'From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion'}, {'measure': 'Relative Reduction in Annualized Rate of Severe VOCs', 'timeFrame': 'From Baseline up to 24 Months After exa-cel Infusion'}, {'measure': 'Duration of Severe VOC Free in Participants who Have Achieved VF12', 'timeFrame': 'From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion'}, {'measure': 'Relative Reduction in Rate of Inpatient Hospitalizations for Severe VOCs', 'timeFrame': 'From Baseline up to 24 Months After exa-cel Infusion'}, {'measure': 'Relative Reduction in Annualized Duration of Hospitalization for Severe VOCs', 'timeFrame': 'From Baseline up to 24 Months After exa-cel Infusion'}, {'measure': 'Proportion of Participants With Sustained HbF >= 20 % for At least 3, 6, or 12 months', 'timeFrame': 'From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion'}, {'measure': 'Relative Reduction in Annualized Volume of RBC Transfusions', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'HbF Concentration Over Time', 'timeFrame': 'Up To 24 Months After exa-cel Infusion'}, {'measure': 'Total Hemoglobin (Hb) Concentration Over Time', 'timeFrame': 'Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change In Reticulocyte Count Over Time', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change in Indirect Bilirubin Over Time', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change in Haptoglobin Over Time', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change in Lactate dehydrogenase (LDH) Over Time', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'Time to First Detectable Haptoglobin', 'timeFrame': 'Up to 24 Months After exa-cel Infusion'}, {'measure': 'Time to First Normalized LDH', 'timeFrame': 'Up to 24 Months After exa-cel Infusion'}, {'measure': 'Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time', 'timeFrame': 'Up To 24 Months After exa-cel Infusion'}, {'measure': 'Proportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over Time', 'timeFrame': 'Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change in Pain Scale (11-point numerical rating scale (NRS)) Assessment Over Time In Adults (>=18 Years)', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Over Time In Adults (>=18 Years)', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change in Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me)', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change in Pain Scale (11-point NRS) Assessment Over Time In Adolescents (12 to <18 years of age)', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change in Pediatric Quality of Life Inventory (PedsQL; self-report and parent proxy versions) Generic Core In Adolescents (12 to <18 years of age)', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}, {'measure': 'Change in PedsQL SCD module (self-report and parent proxy versions) In Adolescents (12 to <18 years of age)', 'timeFrame': 'From Baseline Up To 24 Months After exa-cel Infusion'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Sickle Cell Disease']}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '35 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n* Participants with documented βS/βC (HbSC) genotype\n* Participants must be eligible for autologous stem cell transplant as per investigator's judgment\n\nKey Exclusion Criteria:\n\n* A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement\n* Participants with prior hematopoietic stem cell transplant (HSCT)\n* Treatment with regular RBC transfusions that, in the opinion of the investigator, cannot be interrupted after engraftment.\n\nOther protocol defined Inclusion/Exclusion criteria may apply."}, 'identificationModule': {'nctId': 'NCT05951205', 'briefTitle': 'Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype', 'orgStudyIdInfo': {'id': 'VX21-CTX001-171'}, 'secondaryIdInfos': [{'id': '2023-503247-34-00', 'type': 'OTHER', 'domain': 'EU CT number'}, {'id': '2021-006375-41', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Exa-cel', 'description': 'Participants will receive a single infusion of exa-cel (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene) through a central venous catheter.', 'interventionNames': ['Biological: Exa-cel']}], 'interventions': [{'name': 'Exa-cel', 'type': 'BIOLOGICAL', 'otherNames': ['Exagamglogene autotemcel', 'CTX001'], 'description': 'Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan.', 'armGroupLabels': ['Exa-cel']}]}, 'contactsLocationsModule': {'centralContacts': [{'name': 'Medical Information', 'role': 'CONTACT', 'email': 'medicalinfo@vrtx.com', 'phone': '617-341-6777'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vertex Pharmaceuticals Incorporated', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}