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.
|Condition or Disease||Intervention/Treatment||Phase|
The study may enroll approximately 80 subjects in total.
Arms and Interventions
Administered by IV infusion following lymphodepleting chemotherapy.
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.
Primary Outcome Measures
- 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 Outcome Measures
- 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]
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.
Contacts and Locations
|1||University of Chicago||Chicago||Illinois||United States||60637|
|2||Oregon Health and Science University||Portland||Oregon||United States||97239|
|3||University of Pennsylvania||Philadelphia||Pennsylvania||United States||19104|
|4||Sarah Cannon Research Institute||Nashville||Tennessee||United States||37203|
|5||Royal Prince Alfred Hospital||Sydney||New South Wales||Australia||2050|
|6||Peter MacCallum Cancer Centre||Melbourne||Victoria||Australia||3000|
|7||University Health Network, Princess Margaret Cancer Centre||Toronto||Ontario||Canada||M5G 1X6|
|8||Institut Catala d'Oncologia Hospital Germans Trias i Pujol||Badalona||Barcelona||Spain||08916|
|9||Universidad de Navarra||Pamplona||Navarra||Spain||31008|
|10||Hospital Universitario de Salamanca||Salamanca||Spain||37007|
Sponsors and Collaborators
- CRISPR Therapeutics AG
- Study Director: Ewelina Morawa, MD, CRISPR Therapeutics
Study Documents (Full-Text)None provided.