[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07549698":3,"trial-entities:NCT07549698":183,"trial-summary:NCT07549698":188},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":23,"study_type":33,"primary_purpose":34,"phases":35,"enrollment_info":38,"interventions":41,"primary_outcomes":48,"secondary_outcomes":53,"sex":65,"minimum_age":66,"maximum_age":67,"healthy_volunteers":15,"eligibility_criteria":68,"std_ages":72,"locations":75,"central_contacts":174,"overall_officials":180,"references":181,"see_also_links":182},"NCT07549698","CRSP-AID-501","Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed\u002FRefractory Hematologic Autoimmune Disease","A Phase 1\u002F2 Dose Evaluation Trial of the Safety and Preliminary Efficacy of Anti CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Participants With Relapsed\u002FRefractory Hematologic Autoimmune Disease","RECRUITING","2033-12","2026-07","2026-07-22","2026-06","CRISPR Therapeutics AG","INDUSTRY",false,"This is a single-arm, open-label, multicenter, ascending dose Phase 1\u002F2 trial evaluating the safety and preliminary efficacy of CTX112 or Zugocabtagene geleucel (zugo-cel) in adult participants with relapsed\u002Frefractory primary Immune Thrombocytopenia (ITP) and relapsed\u002Frefractory primary Warm Autoimmune Hemolytic Anemia (wAIHA).","This trial will assess the safety and preliminary efficacy of CTX112 or Zugocabtagene geleucel (zugo-cel) in adults with relapsed or refractory hematologic autoimmune diseases (AID), including primary ITP and primary wAIHA. In these B-cell-mediated conditions, autoantibodies target platelets (ITP) or red blood cells (wAIHA), causing severe thrombocytopenia or anemia. Although several treatments exist, some patients relapse or remain refractory, resulting in significant morbidity, mortality, and reduced quality of life. This underscores the need for new therapeutic options.\n\nB-cell-directed therapies are central to current management, and emerging data show promising activity of anti-CD19 CAR T cell therapies in AID. CTX112 (zugo-cel) is an allogeneic, CD19-targeted CAR T cell product derived from healthy donors and genetically modified ex vivo using CRISPR-Cas9. Similar to autologous CD19 CAR T therapies, CTX112 (zugo-cel) may induce clinical responses after a single treatment and offers the advantages of off-the-shelf availability.\n\nUp to 60 participants may be enrolled. Study duration will be up to 5 years.",[19,20,21,22],"Warm Autoimmune Hemolytic Anemia (WAIHA)","ITP - Immune Thrombocytopenia","Warm Autoimmune Hemolytic Anemia","Immune Thrombocytopenic Purpura",[24,25,26,27,28,29,30,31,22,32],"CD19","CTX112","Zugocabtagene geleucel","zugo-cel","CAR-T","ITP","warm autoimmune hemolytic anemia","wAIHA","Immune Thrombocytopenia","INTERVENTIONAL","TREATMENT",[36,37],"PHASE1","PHASE2",{"count":39,"type":40},60,"ESTIMATED",[42],{"type":43,"name":25,"description":44,"armGroupLabels":45,"otherNames":47},"BIOLOGICAL","CTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components",[46],"CTX112 (zugo-cel)",[26,27],[49],{"measure":50,"description":51,"timeFrame":52},"To evaluate the safety of CTX112 in adult participants with refractory hematologic autoimmune diseases, including ITP or wAIHA.","Incidence of dose-limiting toxicities.","From CTX112 infusion up to 28 days post infusion.",[54,58,62],{"measure":55,"description":56,"timeFrame":57},"To assess the pharmacodynamics response to CTX112 in adult participants with ITP or wAIHA.","Change from baseline in B cell levels","From CTX112 infusion up to 60 months post-infusion",{"measure":59,"description":60,"timeFrame":61},"To assess the pharmacokinetics (PK) of CTX112 in adult participants with ITP or wAIHA.","Levels of CTX112 in blood over time.","From CTX112 infusion up to 60 months post-infusion.",{"measure":63,"description":64,"timeFrame":57},"To assess the preliminary efficacy of CTX112 in adult participants with ITP or wAIHA.","For participants with ITP, platelet response. For participants with wAIHA, hemoglobin response.","ALL","18 Years",null,{"inclusion":69,"exclusion":70,"raw_text":71},[],[],"Inclusion Criteria:\n\n1. Age ≥18 years.\n2. Participants must voluntarily sign a written informed consent and be willing and able to comply with all trial requirements.\n3. Adequate hematologic, renal, liver, cardiac and pulmonary function.\n4. Participants must agree to use acceptable methods of contraception.\n5. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other trial procedures.\n6. Diagnosis of relapsed\u002Frefractory primary Immune Thrombocytopenic Purpura (ITP) or Warm Autoimmune Hemolytic Anemia (WAIHA)\n\nExclusion Criteria:\n\n1. Prior treatment with anti-CD19 therapy or any gene therapy or genetically modified cell therapy.\n2. Prior solid organ (e.g., heart, liver, kidney, lung) transplant or hematopoietic cell transplant.\n3. Severe active or history of central nervous (CNS) involvement.\n4. Presence of other active autoimmune disease or other conditions that are likely to pose increased safety risks and\u002For confound disease assessments, or pose significant risk to those receiving CAR T cell therapy.\n5. History of primary or secondary immunodeficiency.\n6. Presence or history of certain bacterial, viral or fungal infection\n7. Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence).\n8. Diagnosis of a genetic disorder associated with bone marrow failure or myelodysplastic syndrome.\n9. History or current diagnosis that requires uninterrupted, ongoing anticoagulation.\n10. Pregnant or lactating.\n11. Presence or history of disease requiring treatment that is not compatible with the study protocol; presence or history of other conditions that are not compatible with the study protocol.",[73,74],"ADULT","OLDER_ADULT",[76,89,100,110,120,129,139,150,159,165],{"facility":77,"status":8,"city":78,"state":79,"zip":80,"country":81,"contacts":82,"geoPoint":86},"Mayo Clinic in Rochester","Rochester","Minnesota","55905","United States",[83],{"name":84,"role":85},"Jithma Abeykoon","PRINCIPAL_INVESTIGATOR",{"lat":87,"lon":88},44.02163,-92.4699,{"facility":90,"status":8,"city":91,"state":92,"zip":93,"country":81,"contacts":94,"geoPoint":97},"University of Nebraska Medical Center","Omaha","Nebraska","68198",[95],{"name":96,"role":85},"Marcel Devetten",{"lat":98,"lon":99},41.25626,-95.94043,{"facility":101,"status":8,"city":102,"country":103,"contacts":104,"geoPoint":107},"Universitaetsklinikum Koeln","Cologne","Germany",[105],{"name":106,"role":85},"Ruth Flümann",{"lat":108,"lon":109},50.93333,6.95,{"facility":111,"status":112,"city":113,"country":103,"contacts":114,"geoPoint":117},"Medizinische Hochschule Hannover","NOT_YET_RECRUITING","Hannover",[115],{"name":116,"role":85},"Christian Schultze-Florey",{"lat":118,"lon":119},53.1742,8.50694,{"facility":121,"status":112,"city":122,"zip":123,"country":103,"contacts":124,"geoPoint":126},"Medizinische Hochschule Hannover (MHH)","Hanover","30625",[125],{"name":116,"role":85},{"lat":127,"lon":128},52.37052,9.73322,{"facility":130,"status":112,"city":131,"zip":132,"country":103,"contacts":133,"geoPoint":136},"UCT University Medical Center Mainz","Mainz","55131",[134],{"name":135,"role":85},"Eva Wagner-Drouet",{"lat":137,"lon":138},49.98185,8.28008,{"facility":140,"status":8,"city":141,"zip":142,"country":143,"contacts":144,"geoPoint":147},"Hospital Universitario Reina Sofia","Córdoba","14004","Spain",[145],{"name":146,"role":85},"Clara Aparicio Perez",{"lat":148,"lon":149},37.89155,-4.77275,{"facility":151,"status":8,"city":152,"country":143,"contacts":153,"geoPoint":156},"Gregorio Marañón General University Hospital","Madrid",[154],{"name":155,"role":85},"Cristina Pascual Izquierdo",{"lat":157,"lon":158},40.4165,-3.70256,{"facility":160,"status":112,"city":152,"country":143,"contacts":161,"geoPoint":164},"Hospital Universitario La Paz",[162],{"name":163,"role":85},"Ana Mendoza Martínez",{"lat":157,"lon":158},{"facility":166,"status":8,"city":167,"country":143,"contacts":168,"geoPoint":171},"Hospital Universitario Virgen del Rocio","Seville",[169],{"name":170,"role":85},"Maria Eva Mingot Castellano",{"lat":172,"lon":173},37.38283,-5.97317,[175],{"name":176,"role":177,"phone":178,"email":179},"Clinical Trials","CONTACT","877-214-4634","medicalaffairs@crisprtx.com",[],[],[],{"nct_id":4,"conditions":184,"biomarkers":187},[185,186],"Thrombocytopenia Due to Immune Destruction","Warm autoimmune hemolytic anemia",[],{"nct_id":4,"found":15,"summary":67,"prompt_version":67}]