A Phase 1/2 interventional study of CTX112 in Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia and Warm Autoimmune Hemolytic Anemia, sponsored by CRISPR Therapeutics AG. Recruiting at 10 sites in 3 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by CRISPR Therapeutics AG · Phase 1/2, Interventional, and Treatment
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.
Exclusion Criteria:
Administered by IV infusion following lymphodepleting chemotherapy
Biological: CTX112
CTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
Also known as: Zugocabtagene geleucel, zugo-cel
To 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
Plan to share: No
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Purpura, Thrombocytopenic, Idiopathic→
CRISPR Therapeutics AG