A Phase 1/2 Dose Evaluation Trial of the Safety and Preliminary Efficacy of Anti CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease
CRISPR Therapeutics AG
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).
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 the…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
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.…