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

CRISPR Therapeutics AG10 sites in 4 countries26 target enrollmentJanuary 22, 2020

ConditionsMultiple Myeloma

InterventionsCTX120

DrugsCTX120

Overview

Phase: Phase 1
Intervention: CTX120
Conditions: Multiple Myeloma
Sponsor: CRISPR Therapeutics AG
Enrollment: 26
Locations: 10
Primary Endpoint: Part A (dose escalation): Incidence of adverse events
Status: Terminated
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

January 22, 2020

End Date

January 4, 2024

Last Updated

last year

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

CRISPR Therapeutics AG

Responsible Party

Sponsor

Eligibility Criteria

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
•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.

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.

Arms & Interventions

CTX120

Administered by IV infusion following lymphodepleting chemotherapy.

Intervention: CTX120

Outcomes

Primary Outcomes

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 Outcomes

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