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BCMA/GPRC5D Dual-Target CAR-T Cell Immunotherapy for Relapsed/Refractory Multiple Myeloma

4 de novembro de 2026 atualizado por: Liping Dou

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

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

Intervencional

Inscrição (Estimado)

20

Estágio

  • Fase 1

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Locais de estudo

    • Beijing Municipality
      • Beijing, Beijing Municipality, China, 100853
        • Chinese PLA General Hospital
        • Contato:

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

18 anos a 75 anos (Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

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

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

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:
  • SL4903; BCMA-GPRC5D CAR-T injection

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

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Estimado)

30 de novembro de 2026

Conclusão Primária (Estimado)

30 de novembro de 2028

Conclusão do estudo (Estimado)

30 de novembro de 2029

Datas de inscrição no estudo

Enviado pela primeira vez

28 de outubro de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

4 de novembro de 2026

Primeira postagem (Real)

10 de novembro de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

10 de novembro de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

4 de novembro de 2026

Última verificação

1 de novembro de 2026

Mais Informações

Termos relacionados a este estudo

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

NÃO

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

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