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

NCT ID: NCT04035434

Last Updated: 2025-10-24

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 93 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-07-22
• Study Completion Date: 2024-10-04

Brief Summary

This is an open-label, multicenter, Phase 1/2 study evaluating the safety and efficacy of CTX110 in subjects with relapsed or refractory B-cell malignancies.

Detailed Description

The study may enroll up to 227 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).

Conditions

B-cell Malignancy; Non-Hodgkin Lymphoma; B-cell Lymphoma; Adult B Cell ALL

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

CTX110

Administered by IV infusion following lymphodepleting chemotherapy.

Group Type: EXPERIMENTAL

CTX110

Intervention Type: BIOLOGICAL

CTX110 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components

Interventions

CTX110

CTX110 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. For NHL patients: Age ≥18 years. For B cell ALL patients: age ≥18 years to ≤70 years

2. 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.

3. Eastern Cooperative Oncology Group performance status 0 or 1.

4. Adequate renal, liver, cardiac and pulmonary organ function

5. 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.

Exclusion Criteria

1. For NHL patients: prior allogeneic HSCT. For B cell ALL patients: prior allogeneic HSCT within 6 months, and/or any evidence of GvHD.

2. History of central nervous system (CNS) involvement by malignancy

3. History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or any autoimmune disease with CNS involvement.

4. Presence of bacterial, viral, or fungal infection that is uncontrolled.

5. Positive for HIV, or active hepatitis B virus or hepatitis C virus infection.

6. 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.

7. For NHL patients: Use of systemic anti-tumor therapy or investigational agent within 14 days or 5 half-lives, whichever is longer, of CTX110 infusion. For B cell ALL patients: Use of systemic antitumor therapy within 7 days of CTX110 infusion.

8. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.

9. Women who are pregnant or breastfeeding.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

CRISPR Therapeutics AG

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Annie Weaver, PhD

Role: STUDY_DIRECTOR

CRISPR Therapeutics

Locations

Cedars Sinai

Los Angeles, California, United States

UCSF Medical Center

San Francisco, California, United States

Mayo Clinic

Jacksonville, Florida, United States

Emory University Winship Cancer Institute

Atlanta, Georgia, United States

University of Chicago

Chicago, Illinois, United States

University of Kansas

Westwood, Kansas, United States

Markey Cancer Center, University of Kentucky

Lexington, Kentucky, United States

University of Maryland

Baltimore, Maryland, United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

University of Minnesota

Minneapolis, Minnesota, United States

Washington University

St Louis, Missouri, United States

Roswell Park Cancer Insitute

Buffalo, New York, United States

Weill Cornell Medical College / New York Presbyterian Hospital

New York, New York, United States

Duke University

Durham, North Carolina, United States

Oregon Health and Science University

Portland, Oregon, United States

Fox Chase Cancer Center

Philadelphia, Pennsylvania, United States

Sarah Cannon Research Institute

Nashville, Tennessee, United States

Harold C. Simmons Comprehensive Cancer Center, UT Southwestern Medical Center

Dallas, Texas, United States

Texas Transplant Institute

San Antonio, Texas, United States

Virginia Commonwealth University Massey Cancer Center

Richmond, Virginia, United States

Swedish Cancer Institute

Seattle, Washington, United States

Royal Prince Alfred Hospital

Sydney, New South Wales, Australia

Peter MacCallum Cancer Centre

Melbourne, Victoria, Australia

Sir Charles Gairdner Hospital

Nedlands, Western Australia, Australia

Princess Margaret Cancer Centre

Toronto, Ontario, Canada

CHU de Lille

Lille, , France

Institut Paoli-Calmettes

Marseille, , France

Hôpital Saint Antoine

Paris, , France

University of Hamburg

Hamburg, , Germany

University Hospital Würzburg

Würzburg, , Germany

Clinica Universidad de Navarra

Pamplona, Navarre, Spain

Hospital Clínic de Barcelona

Barcelona, , Spain

Hospital Universitario de Salamanca

Salamanca, , Spain

Countries

United States; Australia; Canada; France; Germany; Spain

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CRSP-ONC-001

Identifier Type: -

Identifier Source: org_study_id