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

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ClinicalTrials.gov Identifier: NCT04035434
Recruitment Status : Recruiting
First Posted : July 29, 2019
Last Update Posted : October 6, 2021
Sponsor:
Information provided by (Responsible Party):
CRISPR Therapeutics ( CRISPR Therapeutics AG )

Brief Summary:
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.

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

Detailed Description:
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).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 143 participants
Allocation: N/A
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), for all cohorts: 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), for NHL: 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. Duration of Clinical Benefit (DOCB) [ Time Frame: From date of first objective response of CR/PR until the relapse or death that followed the last response, assessed up to 60 months ]
  3. 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 ]
  4. Overall Survival [ Time Frame: From date of CTX110 infusion until date of death due to any cause, assessed up to 60 months ]
  5. Objective Response Rate (for B cell ALL) [ Time Frame: From CTX110 infusion up to 60 months post-infusion ]
    For B cell ALL, objective response rate (ORR) (complete remission + complete remission with incomplete blood count recovery [CRi]) will be assessed.



Information from the National Library of Medicine

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

Key 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.
  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. For NHL patients: prior allogeneic HSCT. For B cell ALL patients: prior allogeneic HSCT within 6 months, and/or any evidence of GvHD.
  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. 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.
  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 ClinicalTrials.gov identifier (NCT number): NCT04035434


Contacts
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Contact: Clinical Trials +1 (877) 214-4634 MedicalAffairs@crisprtx.com

Locations
Show Show 23 study locations
Sponsors and Collaborators
CRISPR Therapeutics AG
Investigators
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Study Director: Ewelina Morawa, MD CRISPR Therapeutics
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Responsible Party: CRISPR Therapeutics AG
ClinicalTrials.gov Identifier: NCT04035434    
Other Study ID Numbers: CRSP-ONC-001
First Posted: July 29, 2019    Key Record Dates
Last Update Posted: October 6, 2021
Last Verified: October 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 ):
CAR T
Non-Hodgkin Lymphoma
NHL
Lymphoma
Allogeneic
Leukemia
Additional relevant MeSH terms:
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Lymphoma
Neoplasms
Lymphoma, Non-Hodgkin
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases