A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma
- Registration Number
- NCT04244656
- Lead Sponsor
- CRISPR Therapeutics AG
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 26
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description CTX120 CTX120 Administered by IV infusion following lymphodepleting chemotherapy.
- Primary Outcome Measures
Name Time Method 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
Name Time Method 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
Trial Locations
- Locations (10)
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Sarah Cannon Research Institute
🇺🇸Nashville, Tennessee, United States
Peter MacCallum Cancer Centre
🇦🇺Melbourne, Victoria, Australia
Institut Catala d'Oncologia Hospital Germans Trias i Pujol
🇪🇸Badalona, Barcelona, Spain
University of Chicago
🇺🇸Chicago, Illinois, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Royal Prince Alfred Hospital
🇦🇺Sydney, New South Wales, Australia
University Health Network, Princess Margaret Cancer Centre
🇨🇦Toronto, Ontario, Canada
Universidad de Navarra
🇪🇸Pamplona, Navarra, Spain
Hospital Universitario de Salamanca
🇪🇸Salamanca, Spain