Multiple Myeloma
Conditions
Keywords
CAR T, Allogeneic, Multiple myeloma, CRISPR-Cas9
Brief summary
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.
Interventions
BIOLOGICALCTX120
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.
Sponsors
CRISPR Therapeutics AG
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part A (dose escalation): Incidence of adverse events | From CTX120 infusion up to 28 days post-infusion | Adverse events defined as dose-limiting toxicities |
| Part B (cohort expansion): Objective response rate | From CTX120 infusion up to 60 months post-infusion | Objective response rate per International Myeloma Working Group (IMWG) response criteria. |
Secondary
| Measure | Time frame |
|---|---|
| Progression Free Survival | From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months |
| Overall Survival | From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months |
Countries
Australia, Canada, Spain, United States
Outcome results
None listed