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

NCT04035434 | Terminated Phase 1 Results DMC FDA Drug

• 1 other identifier: CRSP-ONC-001
• Study Type: interventional
• Target: 93
• Locations: 6 countries
• Sites: 33

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.

Conditions

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

Keywords

CAR T; Non-Hodgkin Lymphoma; NHL; Lymphoma; Allogeneic; Leukemia

Outcome Measures

Primary Outcomes (2)

• Dose Escalation Phase 1: Number of Participants With Dose-limiting Toxicities (DLT) in NHL and B Cell ALL Population

  A DLT is defined as any of the following events occurring during the DLT evaluation period that persisted beyond the specified duration from onset: Grade ≥2 graft-versus-host disease (GvHD) that was steroid-refractory (e.g., progressive disease after 3 days of steroid treatment \[e.g., 1 mg/kg/day\], stable disease after 7 days, or partial response after 14 days of treatment); death during the DLT period, unless due to disease progression; Grade 4 neurotoxicity of any duration that was related or possibly related to CTX110; or any CTX110-related grade 3 or 4 toxicity deemed clinically significant by the investigator that did not improve within 72 hours.

  Up to 28 days

• Dose Expansion Phase 1 and Phase 2: Percentage of Participants With Objective Response Rate in NHL Population

  The objective response rate (complete response + partial response) was analyzed as per Lugano Response Criteria for Malignant Lymphoma, as determined by independent central radiology review. For participants who received the second course of treatment, the response assessments before and after the second course are combined for the derivation of objective response. Percentages are calculated with the number of participants in the specific analysis set in each column as the denominator. Confidence intervals (CI) of percentage are calculated with Clopper-Pearson exact method.

  Up to 5 years

Secondary Outcomes (9)

• Dose Escalation Phase 1: Percentage of Participants With Objective Response Rate in B Cell ALL Population

  Up to 5 years

• Dose Escalation and Expansion Phase 1: Duration of Response in NHL Population

  Up to 5 years

• Dose Escalation and Expansion Phase 1: Progression Free Survival (PFS) in NHL Population

  Up to 5 years

• Dose Escalation and Expansion Phase 1: Median Overall Survival (OS) in NHL Population

  Up to 5 years

• Dose Escalation and Expansion: Number of Participants With Treatment Emergent Adverse Events (TEAEs) in NHL and B Cell ALL Population

  Up to 5 years

Study Arms (1)

CTX110

EXPERIMENTAL

Administered by IV infusion following lymphodepleting chemotherapy.

Biological: CTX110

Interventions

CTX110 BIOLOGICAL

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

CTX110

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• Positive for HIV, or active 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 CTX110 infusion. For B cell ALL patients: Use of systemic antitumor therapy within 7 days of CTX110 infusion.
• Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
• Women who are pregnant or breastfeeding.

Sponsors & Collaborators

• CRISPR Therapeutics AG: lead

Study Sites (33)

Cedars Sinai

Los Angeles, California, 90048, United States

Location

UCSF Medical Center

San Francisco, California, 94143, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Emory University Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

University of Kansas

Westwood, Kansas, 66205, United States

Location

Markey Cancer Center, University of Kentucky

Lexington, Kentucky, 40536, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Washington University

St Louis, Missouri, 63130, United States

Location

Roswell Park Cancer Insitute

Buffalo, New York, 14203, United States

Location

Weill Cornell Medical College / New York Presbyterian Hospital

New York, New York, 10021, United States

Location

Duke University

Durham, North Carolina, 27710, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States

Location

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

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

Dallas, Texas, 75390, United States

Location

Texas Transplant Institute

San Antonio, Texas, 78229, United States

Location

Virginia Commonwealth University Massey Cancer Center

Richmond, Virginia, 23298, United States

Location

Swedish Cancer Institute

Seattle, Washington, 98104, United States

Location

Royal Prince Alfred Hospital

Sydney, New South Wales, 2050, Australia

Location

Peter MacCallum Cancer Centre

Melbourne, Victoria, 3000, Australia

Location

Sir Charles Gairdner Hospital

Nedlands, Western Australia, 6009, Australia

Location

Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

Location

CHU de Lille

Lille, 59000, France

Location

Institut Paoli-Calmettes

Marseille, 13009, France

Location

Hôpital Saint Antoine

Paris, 75012, France

Location

University of Hamburg

Hamburg, 20148, Germany

Location

University Hospital Würzburg

Würzburg, 97080, Germany

Location

Clinica Universidad de Navarra

Pamplona, Navarre, 31008, Spain

Location

Hospital Clínic de Barcelona

Barcelona, 08036, Spain

Location

Hospital Universitario de Salamanca

Salamanca, 37007, Spain

Location

MeSH Terms

Conditions

Lymphoma, Non-Hodgkin; Lymphoma, B-Cell; Burkitt Lymphoma; Lymphoma; Leukemia

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Epstein-Barr Virus Infections; Herpesviridae Infections; DNA Virus Infections; Virus Diseases; Infections; Tumor Virus Infections; Hematologic Diseases

Limitations and Caveats

Limitations of the trial such as small numbers of participants analysed or technical problems leading to unreliable data.

Results Point of Contact

• Title: Annie Weaver, PhD: Study Director
• Organization: CRISPR Therapeutics AG

info@crisprtx.com

+1 617-315-4600

Study Officials

• Annie Weaver, PhD

  CRISPR Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 22, 2019
• First Posted: July 29, 2019
• Study Start: July 22, 2019
• Primary Completion: October 4, 2024
• Study Completion: October 4, 2024
• Last Updated: October 24, 2025
• Results First Posted: October 24, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (21); AU (3); FR (3); CA (1); DE (2); ES (3)