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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Multiple Myeloma

Intervention / Treatment
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* Biological: CTX120


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Age ≥18 years. Relapsed or refractory multiple myeloma, as defined by IMWG response criteria and treatment with at least 2 prior lines of therapy. Eastern Cooperative Oncology Group performance status 0 or 1. Adequate renal, liver, cardiac and pulmonary organ function 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: Prior allogeneic stem cell transplant (SCT). Less than 60 days from autologous SCT at time of screening and with unresolved serious complications. Prior treatment with any gene therapy or genetically modified cell therapy, including CAR T cells or natural killer cells, or BCMA-directed therapy. Evidence of direct central nervous system (CNS) involvement by multiple myeloma. 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. Unstable angina, clinically significant arrhythmia, or myocardial infarction within 6 months of enrollment. Active HIV, hepatitis B virus or hepatitis C virus infection. 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. Use of systemic anti-tumor therapy or investigational agent within 14 days prior to enrollment. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy. Women who are pregnant or breastfeeding.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CTX120<br>Administered by IV infusion following lymphodepleting chemotherapy. | Biological: CTX120<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CAR T, Allogeneic, Multiple myeloma, CRISPR-Cas9