A Safety and Efficacy Study Evaluating CTX110 in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON)

This is an open-label, multicenter, Phase 1 study evaluating the safety and efficacy of CTX110 in subjects with relapsed or refractory B-cell malignancies.

Inclusion Criteria

• For NHL patients: Age ≥18 years. For B cell ALL patients: age ≥18 years to ≤70 years
• Refractory or relapsed non-Hodgkin lymphoma, as evidenced by 2 or more lines of prior therapy, or histologically confirmed B cell ALL, refractory or relapsed.
• 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 CTX110 infusion.
• Agree to participate in an additional long-term follow-up study after completion of this study.

Exclusion Criteria

• Treatment with any gene therapy or genetically modified cell therapy, including CAR T cells.
• For NHL patients: prior allogeneic HSCT. For B cell ALL patients: prior allogeneic HSCT within 6 months, and/or any evidence of GvHD.
• History of central nervous system (CNS) involvement by malignancy
• History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or any autoimmune disease with CNS involvement.
• Presence of bacterial, viral, or fungal infection that is uncontrolled or requires IV anti-infectives.
• 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.
• For NHL patients: Use of systemic anti-tumor therapy or investigational agent within 14 days or 5 half-lives, whichever is longer, of enrollment. For B cell ALL patients: Use of systemic antitumor therapy within 7 days of enrollment.
• Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
• Women who are pregnant or breastfeeding.

The study may enroll up to 143 subjects in total. CTX110 is a CD19-directed chimeric antigen

receptor (CAR) T cell immunotherapy comprised of allogeneic T cells prepared for the

treatment of B cell malignancies. The cells are from healthy adult volunteer donors that are

genetically modified ex vivo using CRISPR-Cas9 (clustered regularly interspaced short

palindromic repeats/ CRISPR-associated protein 9) gene editing components (single guide RNA

and Cas9 nuclease).

Trial Phase Phase I

Trial Type Treatment

Lead Organization
CRISPR Therapeutics AG

• Primary ID CRSP-ONC-001
• Secondary IDs NCI-2019-07066
• Clinicaltrials.gov ID NCT04035434