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- Ensaio Clínico NCT07927842
BCMA/GPRC5D Dual-Target CAR-T Cell Immunotherapy for Relapsed/Refractory Multiple Myeloma
A Clinical Study on the Safety of BCMA/GPRC5D Dual-Target Autologous CAR-T Cell Immunotherapy for Relapsed/Refractory Multiple Myeloma
This is a single-center, open-label, single-arm, investigator-initiated clinical study to evaluate the safety, tolerability, and preliminary efficacy of SL4903, a BCMA/GPRC5D dual-target autologous CAR-T cell injection, in patients with relapsed or refractory multiple myeloma (R/R MM) whose clonal plasma cells are positive for BCMA and/or GPRC5D. After leukapheresis, participants receive lymphodepleting conditioning (fludarabine 30 mg/m^2/day plus cyclophosphamide 500 mg/m^2/day on Days -5 to -3), followed by a single intravenous infusion of SL4903 at a dose of 1×10^6 CAR-positive cells/kg; bridging therapy (e.g., immunomodulatory drugs or anti-CD38 monoclonal antibody) is permitted between leukapheresis and conditioning at the investigator's discretion. Treatment-emergent adverse events, including cytokine release syndrome (CRS), neurotoxicity, hematologic toxicity, and organ toxicity, will be assessed within 3 months after infusion. Preliminary antitumor activity will be evaluated according to the 2016 International Myeloma Working Group (IMWG) criteria, including objective response rate (ORR), complete response rate (CRR), time to response (TTR), duration of response (DOR), progression-free survival (PFS), overall survival (OS), and measurable residual disease (MRD) negativity rate. Pharmacokinetics (Cmax, Tmax, persistence, and AUCd0-28 of CAR-T cells in peripheral blood by flow cytometry and qPCR) and pharmacodynamics (peripheral blood plasma cell proportion and cytokine levels) will also be evaluated. Participants will be followed for safety and efficacy for up to 2 years after cell therapy.
Visão geral do estudo
Status
Intervenção / Tratamento
Descrição detalhada
This is a single-center, single-arm, open-label, investigator-initiated clinical study of SL4903 autologous T-cell injection in adults with R/R MM. SL4903 is an autologous CAR-T cell product in which the CAR comprises, in order, a GPRC5D-targeting nanobody (VHH1), a BCMA-targeting nanobody (VHH2), a CD8 hinge region, a CD8 transmembrane domain, a 4-1BB costimulatory domain, and a CD3-zeta signaling domain; autologous peripheral blood mononuclear cells are collected by leukapheresis (approximately 80-120 mL), transduced ex vivo with a lentiviral vector, and expanded over a manufacturing period of approximately 7-8 days. Participants receive lymphodepleting conditioning with fludarabine (30 mg/m^2/day) and cyclophosphamide (500 mg/m^2/day) on Days -5 to -3, which may be adjusted at the investigator's discretion, followed by a single intravenous infusion of SL4903 at 1×10^6 CAR-positive cells/kg on Day 0. Planned enrollment is 20 participants; enrollment accounts for potential CAR-T manufacturing failure or rapid disease progression that may preclude infusion. Safety assessments include adverse events and serious adverse events graded per NCI CTCAE v5.0, with CRS and immune effector cell-associated neurotoxicity syndrome (ICANS) graded per the ASTCT consensus criteria; infection, hematologic toxicity, organ toxicity, hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), and tumor lysis syndrome (TLS) are also monitored. Disease response is assessed per the 2016 IMWG criteria at baseline, Day 28, and Months 2, 3, 6, 12, 18, and 24, including serum/urine M-protein studies, serum free light chains, bone marrow examination with flow cytometry/MRD, and imaging (preferably PET-CT) for extramedullary disease. Pharmacokinetic assessments include CAR-T cell expansion in peripheral blood by flow cytometry and qPCR (Cmax, Tmax, in vivo persistence, and AUCd0-28); pharmacodynamic assessments include peripheral blood plasma cell proportion/count and cytokine quantification (at least IL-6, IL-2, IL-10, IFN-gamma, and TNF-alpha).
Tipo de estudo
Inscrição (Estimado)
Estágio
- Fase 1
Contactos e Locais
Contato de estudo
- Nome: LiPing Dou, Doctor
- Número de telefone: +86-13881207138
- E-mail: lipingruirui@163.com
Locais de estudo
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Beijing Municipality
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Beijing, Beijing Municipality, China, 100853
- Chinese PLA General Hospital
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Contato:
- Li-Ping Dou, Doctor
- Número de telefone: +8613881207138
- E-mail: lipingruirui@163.com
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-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
Inclusion Criteria:
- Voluntarily agrees to participate in the study (signed informed consent form by the participant or a legally authorized representative), and is willing and able to comply with scheduled visits, study treatment, laboratory tests, and other study procedures.
- Clinically diagnosed relapsed/refractory multiple myeloma, meeting both of the following:
Clonal plasma cells positive for BCMA and/or GPRC5D by flow cytometry or immunohistochemistry.
Prior treatment with at least 3 drug classes (proteasome inhibitors [PIs], immunomodulatory drugs [IMiDs], and an anti-CD38 monoclonal antibody), with best response less than partial response (PR) or disease progression after achieving PR or better.
- Male or female participants aged 18 to 75 years, inclusive.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
- Estimated life expectancy of more than 3 months from the date of signing the informed consent form.
- Adequate renal, hepatic, cardiac, and pulmonary function, defined as follows:
Creatinine clearance (estimated by the Cockcroft-Gault formula) ≥60 mL/min or serum creatinine ≤1.5 × the upper limit of normal (ULN); Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST)≤2.5×upper limit of normal (ULN), total bilirubin≤1.5×ULN; Cardiac ejection fraction≥50%, no pericardial effusion on echocardiography, and no significant abnormalities on electrocardiogram (ECG); No clinically significant pleural effusion, and oxygen saturation >92% on room air at baseline.
- Participants of reproductive potential must agree to use highly effective contraception before enrollment and for at least 12 months after CAR-T cell infusion. A negative serum pregnancy test is required for women of childbearing potential at screening. Participants must immediately notify the investigator if pregnancy occurs or is suspected.
Exclusion Criteria:
- The patient has severe cardiac dysfunction, or left ventricular ejection fraction (LVEF) <50%, or a history within the 12 months prior to enrollment of myocardial infarction, coronary angioplasty or coronary stent implantation, unstable angina, clinically significant arrhythmia, or other severe cardiovascular diseases.
- A history of severe pulmonary disease with impaired lung function.
- Concurrent progressive malignancy other than multiple myeloma.
- Severe active infection that cannot be effectively controlled.
- Severe autoimmune disease or congenital immunodeficiency.
- Active hepatitis, defined as hepatitis B virus DNA (HBV-DNA) or hepatitis C virus RNA (HCV-RNA) levels above the lower limit of detection, or human immunodeficiency virus (HIV) infection, known acquired immunodeficiency syndrome (AIDS), or syphilis infection.
- A history of severe allergic reactions to biological products, including antibiotics.
- The patient has severe autoimmune disease, congenital immunodeficiency or active autoimmune disease requiring ongoing systemic immunosuppressive treatment, or is currently within the washout period after having received medications that may affect CAR-T cell immunotherapy, or is anticipated to require medications during the study period that may affect CAR-T cell immunotherapy treatment.
- The investigator judges that the patient is unlikely to complete all visits and procedures required by the study protocol (including medium- and long-term follow-up visits), such as insufficient willingness of the patient and their family members to participate in the study, refusal to participate, inability of the patient to fully cooperate with the study arrangements, or inadequate compliance on the part of the patient and their family members.
- Any other severe physical or psychiatric disorder or clinically significant laboratory abnormality that may increase the risk associated with study participation, interfere with the interpretation of study results, or, in the investigator's judgment, make the participant unsuitable for participation in the study.
- Female participants of childbearing potential who are pregnant or breastfeeding.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
Experimental: The experimental group
SL4903 Treatment Group - after leukapheresis and lymphodepleting conditioning (fludarabine 30 mg/m^2/day plus cyclophosphamide 500 mg/m^2/day on Days -5 to -3), participants receive a single intravenous infusion of SL4903 autologous T-cell injection at a dose of 1 x 10^6 CAR-positive cells/kg. Bridging therapy (e.g., immunomodulatory drugs or anti-CD38 monoclonal antibody) may be given between leukapheresis and conditioning at the investigator's discretion. |
SL4903 is an investigational autologous dual-target CAR T-cell therapy (lentiviral vector-based) directed against BCMA and GPRC5D, for patients with relapsed or refractory multiple myeloma whose clonal plasma cells are positive for BCMA and/or GPRC5D. The CAR comprises a GPRC5D-targeting nanobody (VHH1), a BCMA-targeting nanobody (VHH2), a CD8 hinge region, a CD8 transmembrane domain, a 4-1BB costimulatory domain, and a CD3-zeta signaling domain. After leukapheresis and lymphodepleting conditioning (fludarabine plus cyclophosphamide), participants receive a single intravenous infusion of SL4903 at 1×10^6 CAR-positive cells/kg.
Outros nomes:
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Adverse Events
Prazo: From SL4903 infusion through 3 months after infusion; long-term safety follow-up continues through 12 months
|
AEs are graded per NCI CTCAE v5.0 |
From SL4903 infusion through 3 months after infusion; long-term safety follow-up continues through 12 months
|
|
Cytokine Release Syndrome
Prazo: From SL4903 infusion through 3 months after infusion; long-term safety follow-up continues through 12 months
|
Cytokine release syndrome (CRS) is graded according to the ASTCT consensus criteria. |
From SL4903 infusion through 3 months after infusion; long-term safety follow-up continues through 12 months
|
|
Immune Effector Cell-associated Neurotoxicity Syndrome
Prazo: From SL4903 infusion through 3 months after infusion; long-term safety follow-up continues through 12 months
|
Immune effector cell-associated neurotoxicity syndrome (ICANS) is graded according to the ASTCT consensus criteria. |
From SL4903 infusion through 3 months after infusion; long-term safety follow-up continues through 12 months
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Objective Response Rate
Prazo: At Day 28 and Months 2, 3, 6, 12, 18, and 24 after SL4903 infusion
|
The proportion of participants achieving a partial response or better (ORR), assessed by the investigator according to the 2016 International Myeloma Working Group (IMWG) criteria. |
At Day 28 and Months 2, 3, 6, 12, 18, and 24 after SL4903 infusion
|
|
Complete Response Rate
Prazo: At Day 28 and Months 2, 3, 6, 12, 18, and 24 after SL4903 infusion
|
The proportion of participants achieving a complete response (CR) or stringent complete response (sCR), per the 2016 IMWG criteria. |
At Day 28 and Months 2, 3, 6, 12, 18, and 24 after SL4903 infusion
|
|
Time to Response and Duration of Response
Prazo: From SL4903 infusion through 24 months after treatment
|
Time to response (TTR), defined as the time from SL4903 infusion to first documented response, and duration of response (DOR), defined as the time from first documented response to disease progression or death, per the 2016 IMWG criteria. |
From SL4903 infusion through 24 months after treatment
|
|
Progression-Free Survival
Prazo: From SL4903 infusion through 24 months after treatment
|
Progression-free survival (PFS), defined as the time from SL4903 infusion to disease progression or death from any cause, whichever occurs first, per the 2016 IMWG criteria; PFS rates at 6, 9, 12, 18, and 24 months will also be estimated. |
From SL4903 infusion through 24 months after treatment
|
|
Overall Survival
Prazo: From SL4903 infusion through 24 months after treatment
|
Overall survival (OS), defined as the time from SL4903 infusion to death from any cause; OS rates at 6, 9, 12, 18, and 24 months will also be estimated. Participants who are alive at the last follow-up will be censored on the date of their last known survival status. |
From SL4903 infusion through 24 months after treatment
|
|
Measurable Residual Disease (MRD) Negativity Rate
Prazo: At Day 28 and Months 2, 3, 6, 12, 18, and 24 after SL4903 infusion
|
The proportion of participants achieving MRD negativity in bone marrow, assessed by flow cytometry, among responding participants. |
At Day 28 and Months 2, 3, 6, 12, 18, and 24 after SL4903 infusion
|
|
Peak Expansion of SL4903 CAR-T Cells in Peripheral Blood
Prazo: Through Day 28 for AUCd0-28, Cmax, and Tmax; CAR-T persistence through 24 months after infusion
|
Peak expansion of SL4903 CAR-T cells in peripheral blood (Cmax, measured by both flow cytometry and qPCR), |
Through Day 28 for AUCd0-28, Cmax, and Tmax; CAR-T persistence through 24 months after infusion
|
|
Time to Peak Expansion
Prazo: Through Day 28 for AUCd0-28, Cmax, and Tmax; CAR-T persistence through 24 months after infusion
|
Time to Peak Expansion |
Through Day 28 for AUCd0-28, Cmax, and Tmax; CAR-T persistence through 24 months after infusion
|
|
Area Under The Gene-copy Expansion Curve from Day 0 to Day 28
Prazo: Through Day 28 for AUCd0-28, Cmax, and Tmax; CAR-T persistence through 24 months after infusion
|
in vivo persistence of CAR-T cells, and the area under the gene-copy expansion curve from Day 0 to Day 28 (AUCd0-28). |
Through Day 28 for AUCd0-28, Cmax, and Tmax; CAR-T persistence through 24 months after infusion
|
|
Pharmacodynamic Markers (Exploratory)
Prazo: Through 24 months after SL4903 infusion
|
Proportion and absolute count of plasma cells in peripheral blood by flow cytometry, and peripheral blood cytokine levels (at least IL-6, IL-2, IL-10, IFN-gamma, and TNF-alpha) at prespecified time points. |
Through 24 months after SL4903 infusion
|
Colaboradores e Investigadores
Patrocinador
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
- Doenças Vasculares
- Doenças cardiovasculares
- Processos Patológicos
- Neoplasias
- Atributos da doença
- Doenças do sistema imunológico
- Neoplasias por Tipo Histológico
- Doenças Hematológicas
- Distúrbios Linfoproliferativos
- Distúrbios imunoproliferativos
- Neoplasias de Células Plasmáticas
- Distúrbios hemostáticos
- Paraproteinemias
- Distúrbios das Proteínas Sanguíneas
- Distúrbios hemorrágicos
- Condições Patológicas, Sinais e Sintomas
- Doenças hemic e linfáticas
- Recorrência
- Mieloma múltiplo
Outros números de identificação do estudo
- 2026-750
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
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