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).
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
60
CTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
Mayo Clinic in Rochester
Rochester, Minnesota, United States
RECRUITINGUniversity of Nebraska Medical Center
Omaha, Nebraska, United States
RECRUITINGUniversitaetsklinikum Koeln
Cologne, Germany
RECRUITINGMedizinische Hochschule Hannover
Hannover, Germany
RECRUITINGMedizinische Hochschule Hannover (MHH)
Hanover, Germany
RECRUITINGUCT University Medical Center Mainz
Mainz, Germany
RECRUITINGHospital Universitario Reina Sofia
Córdoba, Spain
RECRUITINGGregorio Marañón General University Hospital
Madrid, Spain
RECRUITINGHospital Universitario La Paz
Madrid, Spain
RECRUITINGHospital Universitario Virgen del Rocio
Seville, Spain
RECRUITINGTo evaluate the safety of CTX112 in adult participants with refractory hematologic autoimmune diseases, including ITP or wAIHA.
Incidence of dose-limiting toxicities.
Time frame: From CTX112 infusion up to 28 days post infusion.
To assess the pharmacodynamics response to CTX112 in adult participants with ITP or wAIHA.
Change from baseline in B cell levels
Time frame: From CTX112 infusion up to 60 months post-infusion
To assess the pharmacokinetics (PK) of CTX112 in adult participants with ITP or wAIHA.
Levels of CTX112 in blood over time.
Time frame: From CTX112 infusion up to 60 months post-infusion.
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.
Time frame: From CTX112 infusion up to 60 months post-infusion
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