A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma


This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and efficacy of CTX120 in subjects with relapsed or refractory multiple myeloma.

Full Title of Study: “A Phase 1 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Anti-BCMA Allogeneic CRISPR-Cas9-Engineered T Cells (CTX120) in Subjects With Relapsed or Refractory Multiple Myeloma”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: November 2026


  • Biological: CTX120
    • CTX120 B-cell maturation antigen (BCMA)-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components.

Arms, Groups and Cohorts

  • Experimental: CTX120
    • Administered by IV infusion following lymphodepleting chemotherapy.

Clinical Trial Outcome Measures

Primary Measures

  • Part A (dose escalation): Incidence of adverse events
    • Time Frame: From CTX120 infusion up to 28 days post-infusion
    • Adverse events defined as dose-limiting toxicities
  • Part B (cohort expansion): Objective response rate
    • Time Frame: From CTX120 infusion up to 60 months post-infusion
    • Objective response rate per International Myeloma Working Group (IMWG) response criteria.

Secondary Measures

  • Progression Free Survival
    • Time Frame: From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months
  • Overall Survival
    • Time Frame: From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months

Participating in This Clinical Trial

Key Inclusion Criteria:

1. Age ≥18 years. 2. Relapsed or refractory multiple myeloma, as defined by IMWG response criteria and treatment with at least 2 prior lines of therapy. 3. Eastern Cooperative Oncology Group performance status 0 or 1. 4. Adequate renal, liver, cardiac and pulmonary organ function 5. Female subjects of childbearing potential and male subjects must agree to use acceptable method(s) of contraception from enrollment through at least 12 months after CTX120 infusion. Key Exclusion Criteria:

1. Prior allogeneic stem cell transplant (SCT). 2. Less than 60 days from autologous SCT at time of screening and with unresolved serious complications. 3. Prior treatment with any gene therapy or genetically modified cell therapy, including CAR T cells or natural killer cells, or BCMA-directed therapy. 4. Evidence of direct central nervous system (CNS) involvement by multiple myeloma. 5. History or presence of clinically relevant CNS pathology such as a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, any autoimmune disease with CNS involvement. 6. Unstable angina, clinically significant arrhythmia, or myocardial infarction within 6 months of enrollment. 7. Active HIV, hepatitis B virus or hepatitis C virus infection. 8. Previous or concurrent malignancy, except basal cell or squamous cell skin carcinoma, adequately resected and in situ carcinoma of cervix, or a previous malignancy that was completely resected and has been in remission for ≥5 years. 9. Use of systemic anti-tumor therapy or investigational agent within 14 days prior to enrollment. 10. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy. 11. Women who are pregnant or breastfeeding.

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • CRISPR Therapeutics AG
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Sarah Cohen, MD, Study Chair, CRISPR Therapeutics

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