Ignite Creation Date:
2025-12-24 @ 11:53 PM
Ignite Modification Date:
2026-01-01 @ 3:16 AM
Study NCT ID:
NCT04146051
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-05-28
First Post:
2019-10-29
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009157', 'term': 'Myasthenia Gravis'}], 'ancestors': [{'id': 'D020361', 'term': 'Paraneoplastic Syndromes, Nervous System'}, {'id': 'D009423', 'term': 'Nervous System Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D010257', 'term': 'Paraneoplastic Syndromes'}, {'id': 'D020274', 'term': 'Autoimmune Diseases of the Nervous System'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D020511', 'term': 'Neuromuscular Junction Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2019-12-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2026-03-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-05-22', 'studyFirstSubmitDate': '2019-10-29', 'studyFirstSubmitQcDate': '2019-10-29', 'lastUpdatePostDateStruct': {'date': '2025-05-28', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-10-31', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-07-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of patients with MG Composite improvement of ≥5 points.', 'timeFrame': 'Day 0 to Day 85', 'description': 'In Part 3, the primary objective is to compare the effect of Descartes-08 versus placebo, as measured by the change in MGC score from baseline to Week 12. In Part 1, up to three ascending doses of Descartes-08 will be administered to each of 3 to 6 patients; the patients will be staggered at least 21 days apart and will complete a safety review between each dose. In Part 2, patients will receive up to 6 doses in three different schedules depending on the arm they were enrolled into and will be monitored for up to 1 year to assess the safety and tolerability of a repeated dosing schedule.'}], 'secondaryOutcomes': [{'measure': 'Comparison on Descartes-08 versus placebo on QMG and MG ADL', 'timeFrame': 'Day 0 to Day 85', 'description': 'The proportion of patients achieving a ≥6-point change from Baseline at each posttreatment follow-up in the MG ADL score and QMG score (all parts), and the difference between this proportion in the treatment arm compared to placebo at Day 85.'}, {'measure': 'Comparison on Descartes-08 versus placebo on MG assessments in crossover patients', 'timeFrame': 'Day 0 to Day 85', 'description': 'The proportion of patients initially randomized to placebo who then cross over to Descartes-08 and achieve a ≥5-point reduction in MGC score from Crossover Day\n\n1 to Crossover Day 85.'}, {'measure': 'Change in titer of myasthenia specific autoantibody titers', 'timeFrame': 'Day 0 to Day 168', 'description': 'Determine the change from baseline over a period of 24 weeks in the titer of myasthenia specific autoantibody titers, e.g. nicotinic acetylcholine receptor autoantibody (anti-nAChR) or muscle-specific tyrosine kinase autoantibody (anti- MusK or anti-Low-Density-Lipoprotein-Related-Protein4 (anti-LRP4) or anti-AChR cluster antibody and the 4 Immunoglobulin types (IgG, IgM, IgA, IgE) following single or multiple infusions of Descartes-08 in MG auto-antibody seropositive patients (Parts 1 and 2 only)'}, {'measure': 'Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales', 'timeFrame': 'Day 0 to Day 168', 'description': 'Quantify the clinical effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales (MG ADL, QMG, MG QoL 15R, MG PIS, MG Composite) over a period of 24 weeks in MG auto-antibody seropositive and seronegative patients together and separately (all parts).'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Descartes-08', 'CART', 'CAR-T', 'Autoimmune', 'Auto-antibody', 'Auto antibody', 'chimeric antigen receptor', 'T cells', 'MG', 'gMG', 'Myasthenia Gravis', 'Generalized Myasthenia gravis'], 'conditions': ['Myasthenia Gravis, Generalized']}, 'referencesModule': {'references': [{'pmid': '37353278', 'type': 'DERIVED', 'citation': 'Granit V, Benatar M, Kurtoglu M, Miljkovic MD, Chahin N, Sahagian G, Feinberg MH, Slansky A, Vu T, Jewell CM, Singer MS, Kalayoglu MV, Howard JF Jr, Mozaffar T; MG-001 Study Team. Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study. Lancet Neurol. 2023 Jul;22(7):578-590. doi: 10.1016/S1474-4422(23)00194-1.'}, {'pmid': '37353270', 'type': 'DERIVED', 'citation': 'Meisel A. Are CAR T cells the answer to myasthenia gravis therapy? Lancet Neurol. 2023 Jul;22(7):545-546. doi: 10.1016/S1474-4422(23)00211-9. No abstract available.'}]}, 'descriptionModule': {'briefSummary': 'This is a Phase IIb study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patient must be at least 18 years of age.\n* Patient must have Generalized Myasthenia Gravis at the time of screening.\n* Concomitant immunosuppressive drugs must be deemed necessary by the investigator.\n* Seronegative Patients are included\n\nExclusion Criteria:\n\n* Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator may increase the risk to the patient.\n* Patient is pregnant or lactating.'}, 'identificationModule': {'nctId': 'NCT04146051', 'briefTitle': 'Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Cartesian Therapeutics'}, 'officialTitle': 'Autologous T-Cells Expressing A Chimeric Antigen Receptor Directed To B-Cell Maturation Antigen (BCMA) In Patients With Generalized Myasthenia Gravis (MG)', 'orgStudyIdInfo': {'id': 'MG-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Phase 1b Dose-Escalation', 'description': 'Generalized Myasthenia Gravis', 'interventionNames': ['Drug: Descartes-08']}, {'type': 'EXPERIMENTAL', 'label': 'Phase IIa Expansion', 'description': 'Generalized Myasthenia Gravis', 'interventionNames': ['Drug: Descartes-08']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Phase IIb Randomized Control Trial', 'description': 'Generalized Myasthenia Gravis', 'interventionNames': ['Drug: Descartes-08']}], 'interventions': [{'name': 'Descartes-08', 'type': 'DRUG', 'description': 'Autogolous T-cells expressing a chimeric antigen receptor directed to BCMA', 'armGroupLabels': ['Phase 1b Dose-Escalation', 'Phase IIa Expansion', 'Phase IIb Randomized Control Trial']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92011', 'city': 'Carlsbad', 'state': 'California', 'country': 'United States', 'facility': 'Profound Research', 'geoPoint': {'lat': 33.15809, 'lon': -117.35059}}, {'zip': '92697', 'city': 'Irvine', 'state': 'California', 'country': 'United States', 'facility': 'University of California Irvine', 'geoPoint': {'lat': 33.66946, 'lon': -117.82311}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'University of Colorado Anschutz Medical Campus', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '33487', 'city': 'Boca Raton', 'state': 'Florida', 'country': 'United States', 'facility': 'SFM Clinical Research, LLC', 'geoPoint': {'lat': 26.35869, 'lon': -80.0831}}, {'zip': '32751', 'city': 'Orlando', 'state': 'Florida', 'country': 'United States', 'facility': 'Neurology Associates, P.A.', 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}, {'zip': '33612', 'city': 'Tampa', 'state': 'Florida', 'country': 'United States', 'facility': 'University of South Florida', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'zip': '30912', 'city': 'Augusta', 'state': 'Georgia', 'country': 'United States', 'facility': 'University of Augusta', 'geoPoint': {'lat': 33.47097, 'lon': -81.97484}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center Research Institute', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '14226', 'city': 'Amherst', 'state': 'New York', 'country': 'United States', 'facility': 'Dent Neurologic Institute', 'geoPoint': {'lat': 42.97839, 'lon': -78.79976}}, {'zip': '27599', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'University of North Carolina', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Oregon Health & Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15212', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Allegheny Health Network', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '23219', 'city': 'Richmond', 'state': 'Virginia', 'country': 'United States', 'facility': 'Virginia Commonwealth University', 'geoPoint': {'lat': 37.55376, 'lon': -77.46026}}, {'zip': 'T6G2C8', 'city': 'Edmonton', 'state': 'Alberta', 'country': 'Canada', 'facility': 'University of Alberta', 'geoPoint': {'lat': 53.55014, 'lon': -113.46871}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Cartesian Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}