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).
Age ≥18 years.
Participants must voluntarily sign a written informed consent and be willing and able to comply with all trial requirements.
Adequate hematologic, renal, liver, cardiac and pulmonary function.
Participants must agree to use acceptable methods of contraception.
Prior treatment with anti-CD19 therapy or any gene therapy or genetically modified cell therapy.
Prior solid organ (e.g., heart, liver, kidney, lung) transplant or hematopoietic cell transplant.
Severe active or history of central nervous (CNS) involvement.
Presence of other active autoimmune disease or other conditions that are likely to pose increased safety risks and/or confound disease assessments, or pose significant risk to those receiving CAR T cell therapy.
Single group
CTX112
Biological/VaccineCTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
To evaluate the safety of CTX112 in adult participants with refractory hematologic autoimmune diseases, including ITP or wAIHA.
Incidence of dose-limiting toxicities.
To assess the pharmacodynamics response to CTX112 in adult participants with ITP or wAIHA.
Change from baseline in B cell levels
To assess the pharmacokinetics (PK) of CTX112 in adult participants with ITP or wAIHA.
Levels of CTX112 in blood over time.
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.
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