Base Edited CAR7 T Cells to Treat T Cell Malignancies (TvT CAR7) (TvT CAR7)
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ClinicalTrials.gov Identifier: NCT05397184 |
Recruitment Status :
Recruiting
First Posted : May 31, 2022
Last Update Posted : July 5, 2023
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Tracking Information | |||||||||
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First Submitted Date ICMJE | May 13, 2022 | ||||||||
First Posted Date ICMJE | May 31, 2022 | ||||||||
Last Update Posted Date | July 5, 2023 | ||||||||
Actual Study Start Date ICMJE | April 19, 2022 | ||||||||
Estimated Primary Completion Date | April 1, 2024 (Final data collection date for primary outcome measure) | ||||||||
Current Primary Outcome Measures ICMJE |
Frequency and description of adverse events after BE-CAR7 infusion [ Time Frame: 1 year ] Incidence of Grade 3-5 toxicities occurring from infusion up to one year follow-up.
Severe Adverse Reactions of special interest will be CRS, ICANS and GvHD. American Society of Bone Marrow Transplantation grading scales for CRS/ICANS and National Institute of Health criteria for GVHD will be applied. Commo Terminology Criteria nomenclature will be used to grade other adverse events.
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Original Primary Outcome Measures ICMJE | Same as current | ||||||||
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Current Secondary Outcome Measures ICMJE |
Number of patients achieving disease remission ahead of allo-SCT [ Time Frame: 28 days ] Remission rate will be assessed by bone marrow and CNS evaluation after 28 days. Disease remission is defined as morphological complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) with MRD <10e-3 by flow and/or PCR.
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Original Secondary Outcome Measures ICMJE | Same as current | ||||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
Descriptive Information | |||||||||
Brief Title ICMJE | Base Edited CAR7 T Cells to Treat T Cell Malignancies (TvT CAR7) | ||||||||
Official Title ICMJE | Phase 1 Study of Base Edited CAR7 T Cells to Treat T Cell Malignancies (TvT CAR7) | ||||||||
Brief Summary | T-cell leukaemia is an uncommon type of blood cell cancer that affects white blood cells (T cells). This phase I clinical trial will treat children aged 6 months up to 16 years with T cell leukaemia which has come back (relapsed) after chemotherapy or is not responding to chemotherapy (refractory). The cell therapy is made from white blood cells (T cells) collected from a healthy donor and changed so they can kill other T cells, including leukaemia cells. These 'ready-made' CAR T cells have been made using a new technique called CRISPR base editing to modify them DNA code and have been given the name BE CAR-7. This technique allows them to work after chemotherapy and also disarms them to prevent effects against normal cells. The main aim of this study is to assess the safety of the BE CAR-7 treatment and to see if ready-made CAR T cells can eradicate T cell leukaemia ahead of a planned bone marrow transplant. | ||||||||
Detailed Description | Who can participate? Patients aged 6 months to 16 years with relapsed/refractory T cell leukaemia ahead of a planned bone marrow transplant What does the study involve? Patients will undergo careful screening to confirm that this treatment is adequate for them. Chemotherapy will be given prior to BE CAR-7 infusion to improve the ability of T-cells to establish and grow. Patients will then receive a single infusion of the BE CAR-7 cells and will be closely monitored in hospital. Patients are expected to be in hospital for 4-6 weeks for the BE CAR-7 treatment and the transplant will be scheduled 2-4 weeks after the end of BE CAR7 if leukaemia cells are no longer detectable. Patients will be monitored on the study for 1 year after transplant and then long term in routine clinics. What are the possible benefits and risks of participating? Taking part in the study of testing 'ready-made' CAR T cells could help reduce the amount of disease and get the patient into remission before a bone marrow transplant. Leukaemia is less likely to come back after a bone marrow transplant if levels in the bone marrow are undetectable. The ready-made CAR T cells are being used to try and improve the chances of successful transplantation. Side effects may include low blood cell counts, infections, cytokine storm (severe immune reaction), graft versus host disease (where the donated cells attack the body) and other complications. |
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Study Type ICMJE | Interventional | ||||||||
Study Phase ICMJE | Phase 1 | ||||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Intervention Model Description: Phase 1, open label, non randomised Masking: None (Open Label)Masking Description: Not applicable (Open Label) Primary Purpose: Other
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Condition ICMJE | Relapsed/Refractory T-cell Acute Lymphoid Leukaemia | ||||||||
Intervention ICMJE | Biological: Cryopreserved BE CAR7 T cells (BE752TBCCLCAR7PBL)
Single-dose intravenous infusion (weight-based dosing) of a banded dose of CAR7+ T cells/kg BECAR7 Total duration of treatment: 28 days Follow-up: 12 months
Other Name: BECAR7
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Study Arms ICMJE | Experimental: Single-dose intravenous infusion of a banded dose of CAR7+ T cells/kg BECAR7
Single-dose intravenous infusion (weight-based dosing) of CAR7+ T cells/kg BECAR7 Total duration of treatment: 28 days Follow-up: 12 months Patients will undergo careful screening to confirm that this treatment is adequate for them. Chemotherapy will be given prior to BE CAR-7 infusion. Patients will then receive a single infusion of the BE CAR-7 cells and will be closely monitored in hospital via blood and bone marrow tests for safety and to check the levels of BE CAR-7 and leukaemia cells. Patients are expected to be in hospital for 4-6 weeks for the BE CAR-7 therapy and the transplant will be scheduled 2-4 weeks after the end of BE CAR7 if leukaemia cells are no longer detectable. Patients will be monitored on the study for 1 year every month for the first 3 months and then every 6 months and then long term in routine clinics.
Intervention: Biological: Cryopreserved BE CAR7 T cells (BE752TBCCLCAR7PBL)
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Publications * | Not Provided | ||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||
Recruitment Status ICMJE | Recruiting | ||||||||
Estimated Enrollment ICMJE |
10 | ||||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||||
Estimated Study Completion Date ICMJE | February 28, 2025 | ||||||||
Estimated Primary Completion Date | April 1, 2024 (Final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria ICMJE | Demographic characteristics:
Medical and therapeutic criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 6 Months to 16 Years (Child) | ||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||
Contacts ICMJE |
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Listed Location Countries ICMJE | United Kingdom | ||||||||
Removed Location Countries | |||||||||
Administrative Information | |||||||||
NCT Number ICMJE | NCT05397184 | ||||||||
Other Study ID Numbers ICMJE | 19IC17 | ||||||||
Has Data Monitoring Committee | Yes | ||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Great Ormond Street Hospital for Children NHS Foundation Trust | ||||||||
Original Responsible Party | Same as current | ||||||||
Current Study Sponsor ICMJE | Great Ormond Street Hospital for Children NHS Foundation Trust | ||||||||
Original Study Sponsor ICMJE | Same as current | ||||||||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | Great Ormond Street Hospital for Children NHS Foundation Trust | ||||||||
Verification Date | June 2023 | ||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |