Allogeneic TRAC Locus-inserted CD19-targeting STAR T Cell Therapy in r/r B-NHL

NCT ID: NCT06321289

Last Updated: 2024-03-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-20

Study Completion Date

2027-03-20

Brief Summary

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The team has developed the synthetic T cell receptor (TCR) and antigen receptor (STAR) T cells which were demonstrated safety in relapsed or refractory (r/r) B-cell non-Hodgkin' s lymphoma (B-NHL) (NCT05631912). Based on this research, allogeneic STAR-T cell products utilized the CRISPR-Cas9 gene editing tool to knock out endogenous receptor α constant (TRAC), human leukocyte antigen (HLA)-A/B, CIITA, and programmed death 1 (PD-1) genes simultaneously in T cells from healthy donors, and integrated the STAR molecule into the TRAC locus using adenovirus associated virus. This strategy can reduce graft-versus-host-disease (GvHD) toxicity and host-versus-graft response, decrease the sensitivity of STAR T cells to immunosuppressive signals, and improve their anti-tumor activity. In this single center, prospective, open-label, single-arm, phase 1/2 study, the safety and efficacy of allogeneic CD19-targeting STAR T cell therapy will be evaluated in patients with r/r B-NHL.

Detailed Description

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Phase 1 (dose escalation)

In phase 1, 6 to 18 subjects will be enrolled. Subjects will receive 3 doses of allogeneic CD19-STAR T cell therapy (2 × 10\^6 cells/kg, 6 × 10\^6 cells/kg, 1.8 × 10\^7 cells/kg) from low dose to high dose according to the "3 + 3" principle:

1. Three patients were enrolled in the lowest dose group.
2. Subsequent patients were enrolled according to the following rules:

1. If the incidence of dose limiting toxicity (DLT) was 0/3, 3 patients were enrolled in the next high-dose group.
2. If the incidence of DLT was 1/3, 3 patients were enrolled at the same dose; If the incidence of DLT was 1/3 + 0/3, 3 patients were enrolled in the next high-dose group. If the incidence of DLT was 1/3 + 1/3, this dose was defined as maximum tolerated dose (MTD); If the incidence of DLT was 1/3 + 2/3 or 1/3 + 3/3, the previous dose was MTD.
3. If the incidence of DLT was 2/3 or 3/3, the previous dose was MTD.

To ensure the safety of the subjects, the first subject in each dose group was observed for at least 28 days after the cell infusion. If no DLT occurred, the remaining two subjects could be enrolled and treated at the same dose level. The safety data of all subjects in each dose group until day 28 should be reviewed and tolerated before proceeding to the next dose group trial. No dose escalation was allowed for the same subject during the trial. If a subject drop out during the observation period due to non-DLT reasons, new subjects should be enrolled to make up for the number of subjects who drop out.

Phase 2 (expansion cohort)

In phase 2, 10 to 12 subjects will be enrolled and receive allogeneic CD19-STAR T cell infusion at dose of recommended phase 2 dose (RP2D), which will be determined based on the MTD, occurrence of DLT, the obtained efficacy results, pharmacokinetics / pharmacodynamics and other data according to the phase 1.

Objectives

The primary objectives of the phase 1 are to evaluate the tolerability, safety, and determine RP2D. The primary purpose of the phase 2 study is to evaluate the efficacy.

Conditions

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Non-hodgkin Lymphoma,B Cell

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Allogeneic TRAC locus-inserted CD19-targeting STAR T cells

A conditioning chemotherapy regimen of fludarabine and cyclophosphamide (FC regimen) will be administered followed by investigational treatment, allogeneic targeting CD19 synthetic T-cell receptor antigen receptor T cells.

Group Type EXPERIMENTAL

Allogeneic CD19-STAR T cell

Intervention Type BIOLOGICAL

Phase 1 dose escalation (3+3): dose 1 (2×10\^6 cells/kg), dose 2 (6×10\^6 cells/kg), dose 3 (1.8×10\^7 cells/kg); Phase 2: dose of RP2D. No more than 5 × 10\^4 per kilogram of allogenic residual TCR-positive T cells harbouring in grafts can only be released for recipient infusion.

Fludarabine

Intervention Type DRUG

Intravenous fludarabine 30-50 mg/m\^2/day on days -5, -4, and -3.

Cyclophosphamide

Intervention Type DRUG

Intravenous cyclophosphamide 500-1000 mg/m\^2/day on days -5, -4, and -3.

Interventions

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Allogeneic CD19-STAR T cell

Phase 1 dose escalation (3+3): dose 1 (2×10\^6 cells/kg), dose 2 (6×10\^6 cells/kg), dose 3 (1.8×10\^7 cells/kg); Phase 2: dose of RP2D. No more than 5 × 10\^4 per kilogram of allogenic residual TCR-positive T cells harbouring in grafts can only be released for recipient infusion.

Intervention Type BIOLOGICAL

Fludarabine

Intravenous fludarabine 30-50 mg/m\^2/day on days -5, -4, and -3.

Intervention Type DRUG

Cyclophosphamide

Intravenous cyclophosphamide 500-1000 mg/m\^2/day on days -5, -4, and -3.

Intervention Type DRUG

Other Intervention Names

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Allogeneic CD19-targeting synthetic T-cell receptor antigen receptor T cells Fludarabine Phosphate for Injection Cyclophosphamide for Injection

Eligibility Criteria

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Inclusion Criteria

1. Age 18-75 (inclusive).
2. Patients with histologically confirmed CD19-positive B-cell NHL, including the following types defined by the World Health Organization (WHO) 2016:

* Diffuse large B-cell lymphoma not otherwise specified (DLBCL-NOS), including activated B-cell type (ABC) / germinal center B-cell Type (GCB);
* Primary mediastinal (thymic) large B-cell lymphoma (PMBCL);
* Transformed follicular lymphoma (TFL);
* High-grade B-cell lymphoma with MYC and BCL2 and/or BCL6 rearrangements (HGBCL);
* Follicular lymphoma (FL);
* Mantle cell lymphoma (MCL) \[pathologically confirmed, with documentation of monoclonal B cells that have a chromosome translocation t(11;14)(q13;q32) and/or overexpress cyclin D1\];
* Marginal zone lymphoma (MZL), including nodal or splenic marginal zone B-cell lymphoma and mucosa-associated lymphoid tissue (MALT) lymphoma.
3. Relapse after treatment with ≥2 lines systemic therapy for all the above disease types, or refractory disease for aggressive types (DLBCL-NOS, PMBCL, TFL and HGBCL). Relapse disease is defined as disease progression after last regimen. Refractory disease is defined as no CR to first-line therapy:

* Evaluation of PD (never reached response or SD) after standard first-line treatment, or
* SD as best response after at least 4 cycles of first-line therapy (eg, 4 cycles of R-CHOP), or
* PR as best response after at least 6 cycles and biopsy-proven residual disease or disease progression ≤ 6 months of therapy, or
* Refractory post-autologous stem cell transplant (ASCT): i. Disease progression or relapsed less than or equal to 12 months of ASCT (must have biopsy proven recurrence in relapsed individuals); ii. If salvage therapy is given post-ASCT, the individual must have had no response to or relapsed after the last line of therapy.
4. Individuals must have received adequate prior therapy:

For MCL, prior therapy must have included:
* Anthracycline or bendamustine-containing chemotherapy and
* Anti-CD20 monoclonal antibody (unless investigator determines that tumor is CD20-negative) and
* Bruton tyrosine kinase inhibitor (BTKi)

For other types, prior therapy must have included:
* Anti-CD20 monoclonal antibody (unless investigator determines that tumor is CD20-negative) and
* Anthracycline containing chemotherapy regimen.

For individual with TFL must have relapse or refractory disease after transformation to DLBCL.
5. The estimated survival time is over 3 months.
6. The Eastern Cooperative Oncology Group (ECOG) score is 0-2.
7. According to Lugano response criteria 2014, there should be at least one evaluable tumor focus. Evaluable tumor focus was defined as that with the longest diameter of intranodal focus \> 1.5cm, the longest diameter of extranodal focus \> 1.0cm assessed by computed tomography (CT) or magnetic resonance imaging (MRI).
8. Subjects must be willing to undergo either excised or large-needle lymph node or tissue biopsy, or provide formalin-fixed paraffin-embedded (FFPE) tumor tissue block or freshly cut unstained slides.
9. Functions of important organs meet the following requirements:

* Echocardiography showed left ventricular ejection fraction ≥50%;
* Serum creatinine ≤1.5 × upper limit of normal range (ULN) or endogenous creatinine clearance ≥45mL/min (cockcroft-gault formula);
* Total bilirubin ≤1.5× ULN;
* Pulmonary function: ≤CTCAE grade 1 dyspnea and oxygen saturation of blood (SaO2) ≥91% in indoor air environment.
10. Blood routine: hemoglobin (Hgb) ≥ 80g/L, neutrophil count (ANC) ≥ 1 × 10 \^ 9/L, platelet count (PLT) ≥ 75 × 10 \^ 9/L. Excluding when there is bone marrow infiltration. It is not allowed to obtain normal values through growth factor intervention.
11. Pregnancy tests for women of childbearing age shall be negative; Both men and women agreed to use effective contraception during treatment and during the subsequent 1 year.

Exclusion Criteria

13. No obvious hereditary diseases.
14. Able to understand the requirements and matters of the trial, and willing to participate in clinical research as required.
15. Informed consent must be signed.


1. During the screening period, there was central nervous system (CNS) invasion or a history of clinically significant CNS diseases, such as epilepsy and cerebrovascular diseases.
2. Women who are pregnant or breastfeeding, or who do not agree to use effective contraception during treatment and during the subsequent 1 year.
3. History of allogeneic hematopoietic stem cell transplantation, or organ transplantation.
4. History of other malignancies that have not been in remission.
5. Patients with primary immunodeficiency or autoimmune diseases requiring immunosuppressive therapy.
6. Received radiotherapy within 3 months before enrollment.
7. Received immunotherapy drugs within 4 weeks before enrollment, such as anti-PD-1 antibody, anti-programmed death ligand 1 (PD-L1) antibody, CD19/CD3-bispecific antibody, and so on.
8. Confirmed evidence showing positiveness of anti-CD19 scFv reaction in patient serum.
9. Prior CD19 targeted therapy.
10. Prior CAR-T therapy or other genetically modified T cell therapy.
11. Presence of DSA directed against STAR T cells.
12. Patients who participated in other clinical trials within 4 weeks prior to enrollment.
13. Uncontrolled infectious diseases or other serious illnesses, including but not limited to infections \[e.g., human immunodeficiency virus (HIV) infection or acute or chronic active hepatitis B (HBV) or C (HCV) infection\], congestive heart failure, unstable angina, arrhythmias, or that pose an unpredictable risk in the opinion of the attending physician.
14. The presence of uncontrollable serous membrane fluid, such as massive pleural effusion or ascites.
15. A history of stroke or intracranial hemorrhage within 3 months prior to enrollment.
16. Major surgery or trauma occurred within 28 days prior to enrollment, or major side effects have not been recovered.
17. History of allergies to any of the ingredients in cell products.
18. Conditions in which a known mental or physical illness interferes with cooperation with the requirements of the study or disrupts the results or interpretation of the results and, in the opinion of the therapeutic investigator, makes the patient unfit for study participation.
19. There is the situation that the researcher's judgment will interfere with the whole study participation; Situations where there is significant risk to the subject; Or interferes with the interpretation of research data.
20. Inability to understand or unwillingness to sign informed consent.
21. Researchers believe that other reasons are not suitable for clinical trials.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Chinese PLA General Hospital

OTHER

Sponsor Role lead

Responsible Party

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Han weidong

Director of Biotherapeutic Department

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Biotherapeutic Department of Chinese PLA General Hospital

Beijing, Beijing Municipality, China

Site Status RECRUITING

School of medicine, Tsinghua University & Changping Laboratory

Beijing, , China

Site Status RECRUITING

Countries

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China

Central Contacts

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Weidong Han, Ph.D

Role: CONTACT

+86-010-55499341

Facility Contacts

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Weidong Han, M.D

Role: primary

+86-010-66937463

Xin Lin, Ph.D

Role: primary

+86-010-62796319

Other Identifiers

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CHN-PLAGH-BT-084

Identifier Type: -

Identifier Source: org_study_id

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