Ignite Creation Date:
2025-12-24 @ 11:24 PM
Ignite Modification Date:
2025-12-27 @ 1:24 AM
Study NCT ID:
NCT06665256
Status:
RECRUITING
Last Update Posted:
2025-10-28
First Post:
2024-10-29
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase 2 Study of Rapcabtagene Autoleucel in Myositis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009220', 'term': 'Myositis'}, {'id': 'D003882', 'term': 'Dermatomyositis'}, {'id': 'D017563', 'term': 'Lung Diseases, Interstitial'}], 'ancestors': [{'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D017285', 'term': 'Polymyositis'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['OUTCOMES_ASSESSOR'], 'maskingDescription': 'The study investigator and the participant will be unblinded to the study treatment. A blinded assessor will perform the efficacy assessments to minimize bias in data collection.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 123}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-12-17', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2030-07-17', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-10-24', 'studyFirstSubmitDate': '2024-10-29', 'studyFirstSubmitQcDate': '2024-10-29', 'lastUpdatePostDateStruct': {'date': '2025-10-28', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-10-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-07-17', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of participants achieving at least moderate improvement in Total Improvement Score (TIS) at Week 52', 'timeFrame': 'Week 52', 'description': 'The percentage of participants with a TIS of at least 40 at the 52nd week after the start of the study, corresponding to at least moderate improvement.'}], 'secondaryOutcomes': [{'measure': 'Adjusted annual cumulative glucocorticoid dose up to Week 52', 'timeFrame': 'Week 52', 'description': 'The total amount of glucocorticoids administered over the course of a year measured up to the 52nd week of treatment.'}, {'measure': 'Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52', 'timeFrame': 'Baseline, Week 52', 'description': 'The difference in the percentage of the predicted Forced Vital Capacity (FVC) from the start of the study to the 52nd week.'}, {'measure': 'Proportion of participants achieving major improvement in TIS at Week 52', 'timeFrame': 'Week 52', 'description': 'The percentage of participants with a TIS of at least 60 at the 52nd week after the start of the study, corresponding to at least major improvement.'}, {'measure': 'Change from baseline in Patient-Reported-Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52', 'timeFrame': 'Baseline, Week 52', 'description': 'The difference in the fatigue levels reported by participants from the start of the study to the 52nd week.'}, {'measure': 'Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Week 104', 'description': 'The distribution of adverse events will be done via the analysis of frequencies for Adverse Event (AEs) and Serious Adverse Event (SAEs) through the monitoring of relevant clinical and laboratory safety parameters.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Chimeric Antigen Receptor T cells (CAR-T)', 'rapcabtagene autoleucel', 'idiopathic inflammatory myopathies (IIM)', 'dermatomyositis', 'Anti-Synthetase Syndrome', 'Immune-Mediated Necrotizing Myopathy', 'Interstitial Lung Disease'], 'conditions': ['Idiopathic Inflammatory Myopathies']}, 'descriptionModule': {'briefSummary': 'A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies (IIM)', 'detailedDescription': 'This is a Phase 2, two-year, randomized, assessor- blinded, active-controlled study. This study comprises two cohorts:\n\n* A lead-in cohort enrolling participants to receive rapcabtagene autoleucel\n* A randomized cohort with participants receiving either rapcabtagene autoleucel or a comparator option.\n\nAfter end of study (EOS), participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period lasting up to 15 years after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n1. Men and women, aged \\>18 and ≤65 years, with a diagnosis of probable or definite myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria\n2. Participants who had inadequate response to prior therapy\n3. Diagnosed with active disease\n4. Participant must meet criteria for severe myositis\n\nKey Exclusion Criteria:\n\n1. Any condition during Screening that could prevent a complete washout of medications or could otherwise make the participant ineligible for anti-CD19 CAR-T therapy and further participation in the study\n2. BMI at Screening of ≤18.5 or ≥35 kg/m2\n3. Severe muscle damage at Screening\n4. Inadequate organ function\n5. Hypersensitivity and/or contraindications to any product (including its ingredients) to be given to the participant as per the study protocol\n6. Other inflammatory and non-inflammatory myopathies\n7. Any medical conditions that are not related to IIM that would jeopardize the ability of the participant to tolerate CD19 CAR-T cell therapy\n\nOther protocol-defined inclusion/exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT06665256', 'briefTitle': 'Phase 2 Study of Rapcabtagene Autoleucel in Myositis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novartis'}, 'officialTitle': 'A Phase 2, Randomized, Open-label, Controlled Study to Evaluate the Efficacy and Safety of Rapcabtagene Autoleucel Versus Comparator in Participants With Severe Refractory Idiopathic Inflammatory Myopathies (IIM)', 'orgStudyIdInfo': {'id': 'CYTB323L12201'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Rapcabtagene autoleucel', 'description': 'Single infusion of rapcabtagene autoleucel (YTB323)', 'interventionNames': ['Biological: Rapcabtagene autoleucel']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Comparator', 'description': 'Investigator choice of treatment as per protocol', 'interventionNames': ['Other: Active Comparator Option']}], 'interventions': [{'name': 'Rapcabtagene autoleucel', 'type': 'BIOLOGICAL', 'otherNames': ['YTB323'], 'description': 'Single infusion of rapcabtagene autoleucel', 'armGroupLabels': ['Rapcabtagene autoleucel']}, {'name': 'Active Comparator Option', 'type': 'OTHER', 'description': 'Investigator choice of treatment as per protocol', 'armGroupLabels': ['Comparator']}]}, 'contactsLocationsModule': {'locations': [{'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Matthew Selle', 'role': 'CONTACT', 'email': 'autoimmunesct@nm.org'}, {'name': 'George Georges', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Northwestern University', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Ashley Pieper', 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to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novartis Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}