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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04035434
Recruitment Status : Recruiting
First Posted : July 29, 2019
Last Update Posted : January 19, 2021
Information provided by (Responsible Party):
CRISPR Therapeutics ( CRISPR Therapeutics AG )

Brief Summary:
This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and efficacy of CTX110 in subjects with relapsed or refractory B-cell malignancies.

Condition or disease Intervention/treatment Phase
B-cell Malignancy Non-Hodgkin Lymphoma B-cell Lymphoma Biological: CTX110 Phase 1

Detailed Description:
The study may enroll up to 131 subjects in total.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 131 participants
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Allogeneic CRISPR-Cas9-Engineered T Cells (CTX110) in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON)
Actual Study Start Date : July 22, 2019
Estimated Primary Completion Date : July 2026
Estimated Study Completion Date : August 2026

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: CTX110
Administered by IV infusion following lymphodepleting chemotherapy.
Biological: CTX110
CTX110 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components

Primary Outcome Measures :
  1. Part A (Dose Escalation): Incidence of adverse events, defined as dose-limiting toxicities [ Time Frame: From CTX110 infusion up to 28 days post-infusion ]
  2. Part B (Cohort Expansion): Objective response rate [ Time Frame: From CTX110 infusion up to 60 months post-infusion ]

Secondary Outcome Measures :
  1. Duration of Response [ Time Frame: From date of first objective response of CR/PR until date of disease progression or death due to any cause, assessed up to 60 months ]
    Duration of Response (DOR) will only be reported for subjects who have had CR/PR events

  2. Progression Free Survival [ Time Frame: From date of CTX110 infusion until date of disease progression or death due to any cause, assessed up to 60 months ]
  3. Overall Survival [ Time Frame: From date of CTX110 infusion until date of death due to any cause, assessed up to 60 months ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  1. Age ≥18 years.
  2. Refractory or relapsed non-Hodgkin lymphoma, as evidenced by 2 or more lines of prior therapy.
  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.
  6. Agree to participate in an additional long-term follow-up study after completion of this study.

Key Exclusion Criteria:

  1. Treatment with any gene therapy or genetically modified cell therapy, including CAR T cells.
  2. Prior allogeneic HSCT.
  3. History of central nervous system (CNS) involvement by malignancy
  4. History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or any autoimmune disease with CNS involvement.
  5. Presence of bacterial, viral, or fungal infection that is uncontrolled or requires IV anti-infectives.
  6. Active HIV, hepatitis B virus or hepatitis C virus infection.
  7. 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.
  8. Use of systemic anti-tumor therapy or investigational agent within 14 days or 5 half-lives, whichever is longer, of enrollment.
  9. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
  10. Women who are pregnant or breastfeeding.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04035434

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Contact: Clinical Trials +1 (877) 214-4634

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United States, California
UCSF Medical Center Recruiting
San Francisco, California, United States, 94143
Contact: Cancer Immunotherapy Program   
United States, Florida
Mayo Clinic Recruiting
Jacksonville, Florida, United States, 32224
United States, Georgia
Emory University Winship Cancer Institute Recruiting
Atlanta, Georgia, United States, 30322
United States, Illinois
University of Chicago Recruiting
Chicago, Illinois, United States, 60637
United States, Kansas
University of Kansas Recruiting
Westwood, Kansas, United States, 66205
Contact: Joseph P McGuirk, DO    913-708-4032   
Contact: Jeff Roesgen   
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Ashley Lyle, CRC-RN   
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Richard Maziarz, MD   
Contact: Kevin Christmas, PhD    503-494-6474   
United States, Tennessee
Sarah Cannon Research Institute Recruiting
Nashville, Tennessee, United States, 37203
Contact: Ryan Birchfield    615-982-4833   
United States, Texas
Harold C. Simmons Comprehensive Cancer Center, UT Southwestern Medical Center Recruiting
Dallas, Texas, United States, 75390
Contact: Farrukh Awan, MD   
Texas Transplant Institute Recruiting
San Antonio, Texas, United States, 78229
Contact: Paul Shaughnessy, MD   
Contact: Sherri Shade, RN, CCRC    210-575-4238   
Australia, New South Wales
Royal Prince Alfred Hospital Recruiting
Sydney, New South Wales, Australia, 2050
Contact: Joy Ho, MBBS    +61 2 95158036   
Contact: Christine Contacos, PhD    +61 2 95153370   
Australia, Victoria
Peter MacCallum Cancer Centre Recruiting
Melbourne, Victoria, Australia, 3000
Contact: Constantine Tam, MBBS, MD    +61 3 8559 7858   
Contact: Michael Dickinson, MBBS, MD    +61 3 8559 7858   
University of Hamburg Recruiting
Hamburg, Germany, 20148
Contact: Natascha von Huenerbein    +49 (0) 40 7410-23181   
Contact: Marion Heinzelmann    +49 (0) 40 7410 - 54188   
Sponsors and Collaborators
CRISPR Therapeutics AG
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Study Director: Ewelina Morawa, MD CRISPR Therapeutics
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Responsible Party: CRISPR Therapeutics AG Identifier: NCT04035434    
Other Study ID Numbers: CRSP-ONC-001
First Posted: July 29, 2019    Key Record Dates
Last Update Posted: January 19, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by CRISPR Therapeutics ( CRISPR Therapeutics AG ):
Non-Hodgkin Lymphoma
Additional relevant MeSH terms:
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Lymphoma, Non-Hodgkin
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases