Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease
Start Date
6/1/2026
Completion Date
12/1/2033
Summary
This is a single-arm, open-label, multicenter, ascending dose Phase 1/2 trial evaluating the safety and preliminary efficacy of CTX112 or Zugocabtagene geleucel (zugo-cel) in adult participants with relapsed/refractory primary Immune Thrombocytopenia (ITP) and relapsed/refractory primary Warm Autoimmune Hemolytic Anemia (wAIHA).
Detailed Description
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. B-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. Up to 60 participants may be enrolled. Study duration will be up to 5 years.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
CTX112
Conditions
Locations
Mayo Clinic in Rochester
Rochester, Minnesota 55905
United States
University of Nebraska Medical Center
Omaha, Nebraska 68198
United States
Universitaetsklinikum Koeln
Cologne,
Germany
Medizinische Hochschule Hannover
Hannover,
Germany
Medizinische Hochschule Hannover (MHH)
Hanover, 30625
Germany
UCT University Medical Center Mainz
Mainz, 55131
Germany
Hospital Universitario Reina Sofia
Córdoba, 14004
Spain
Gregorio Marañón General University Hospital
Madrid,
Spain
Hospital Universitario La Paz
Madrid,
Spain
Hospital Universitario Virgen del Rocio
Seville,
Spain