RECRUITING Phase 1 / Phase 2 ClinicalTrials.gov

Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease

Sponsor: CRISPR Therapeutics AG • ClinicalTrials.gov ID: NCT07549698

Registry updated: 2026-10-20

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Description

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).

Interventions

CTX112 (Biological)
CTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components

Eligibility criteria

Inclusion Criteria: 1. Age ≥18 years. 2. Participants must voluntarily sign a written informed consent and be willing and able to comply with all trial requirements. 3. Adequate hematologic, renal, liver, cardiac and pulmonary function. 4. Participants must agree to use acceptable methods of contraception. 5. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other trial procedures. 6. Diagnosis of relapsed/refractory primary Immune Thrombocytopenic Purpura (ITP) or Warm Autoimmune Hemolytic Anemia (WAIHA) Exclusion Criteria: 1. Prior treatment with anti-CD19 therapy or any gene therapy or genetically modified cell therapy. 2. Prior solid organ (e.g., heart, liver, kidney, lung) transplant or hematopoietic cell transplant. 3. Severe active or history of central nervous (CNS) involvement. 4. 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. 5. History of primary or secondary immunodeficiency. 6. Presence or history of certain bacterial, viral or fungal infection 7. Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence). 8. Diagnosis of a genetic disorder associated with bone marrow failure or myelodysplastic syndrome. 9. History or current diagnosis that requires uninterrupted, ongoing anticoagulation. 10. Pregnant or lactating. 11. 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.

Conditions

Warm Autoimmune Hemolytic Anemia (WAIHA) ITP - Immune Thrombocytopenia Warm Autoimmune Hemolytic Anemia Immune Thrombocytopenic Purpura

Locations

Boston Medical Center — Boston, Massachusetts, United States
Mayo Clinic in Rochester — Rochester, Minnesota, United States
University of Nebraska Medical Center — Omaha, Nebraska, United States
Universitaetsklinikum Koeln — Cologne, Germany
Universitaetsklinikum Carl Gustav Carus Dresden — Dresden, Germany
Medizinische Hochschule Hannover (MHH) — Hanover, Germany
UCT University Medical Center Mainz — Mainz, Germany
Hospital Clinic Barcelona — Barcelona, Spain
Hospital Universitario Reina Sofia — Córdoba, Spain
Gregorio Marañón General University Hospital — Madrid, Spain
Hospital Universitario La Paz — Madrid, Spain
Hospital Universitario Virgen del Rocio — Seville, Spain

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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