Study to Evaluate the Safety and Preliminary Efficacy of CD19 CAR-T Cell Therapy (TranspoCART19) in Patients With Refractory Lupus Nephritis (CART-NEL)
A National Multicenter Phase I/IIa Study to Evaluate the Safety and Preliminary Efficacy of CD19 CAR-T Cell Therapy (TranspoCART19) in Patients With Refractory Lupus Nephritis.
The goal of this clinical trial is to evaluate the safety and preliminary efficacy of CD19 CAR-T cell therapy (TranspoCART19) in adults with refractory lupus nephritis. Lupus nephritis is a serious kidney complication of systemic lupus erythematosus that may not respond adequately to standard treatments.
The main questions this study aims to answer are:
- Is TranspoCART19 safe and tolerable in patients with refractory lupus nephritis?
- Can TranspoCART19 induce complete or partial clinical and immunological remission?
Participants will:
- Undergo leukapheresis to collect immune cells for manufacturing TranspoCART19.
- Receive lymphodepleting chemotherapy before treatment.
- Receive a single fractionated infusion of TranspoCART19.
- Attend regular follow-up visits for safety, disease activity, kidney function, immune response, and quality-of-life assessments for up to 24 months, with long-term safety follow-up after study completion.
Descripción general del estudio
Estado
Estado
Condiciones
Condiciones
Intervención / Tratamiento
Intervención / Tratamiento
Descripción detallada
This is a Phase I/IIa, academic, multicenter, open-label, single-arm clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of TranspoCART19 in adult patients with refractory lupus nephritis.
TranspoCART19 is an autologous CD19-directed chimeric antigen receptor (CAR) T-cell therapy manufactured using Sleeping Beauty transposon technology. The CAR construct incorporates an anti-CD19 FMC63 single-chain variable fragment, a 4-1BB co-stimulatory domain, a CD3ζ signaling domain, and a truncated human epidermal growth factor receptor (hEGFRt) safety switch.
Eligible participants will undergo leukapheresis for collection of peripheral blood mononuclear cells. Following manufacturing of the investigational product, participants will receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide, or bendamustine when clinically indicated, prior to administration of TranspoCART19.
TranspoCART19 will be administered intravenously at a target dose of 1 × 10^6 CAR-T cells/kg body weight using a fractionated infusion strategy consisting of 10%, 30%, and 60% dose fractions. Participants will remain under close monitoring for early and late treatment-related toxicities.
The primary objective is to evaluate the safety and tolerability of TranspoCART19 during the early post-infusion period. Safety assessments include adverse events, serious adverse events, cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), severe infections, prolonged cytopenias, and hypogammaglobulinemia.
Secondary objectives include evaluation of clinical, renal, histological, and immunological responses; hematologic and immune reconstitution; CAR-T cell persistence and kinetics; corticosteroid and immunosuppressive treatment withdrawal; systemic lupus erythematosus disease activity; and health-related quality of life.
Approximately 10 participants will be enrolled. Participants will be followed for 24 months after infusion, and long-term safety monitoring will continue for up to 15 years in accordance with recommendations for genetically modified cellular therapies.
Tipo de estudio
Tipo de estudio
Inscripción (Estimado)
Inscripción
Fase
Fase
- Fase 2
- Fase 1
Contactos y Ubicaciones
Estudio Contacto
Estudio Contacto
- Nombre: Eva Cerezo
- Número de teléfono: 3214 91 5504800
- Correo electrónico: eva.cerezo@quironsalud.es
Copia de seguridad de contactos de estudio
- Nombre: Lucia Llanos
- Número de teléfono: 3214 91 5504800
- Correo electrónico: lucia.llanos@quironsalud.es
Ubicaciones de estudio
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León, España, 24008
- Aún no reclutando
- Hospital Universitario de León
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Madrid, España, 28040
- Aún no reclutando
- Hospital Universitario Fundación Jiménez Diaz
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Murcia, España, 30120
- Aún no reclutando
- Hospital Clínico Universitario Virgen de la Arrixaca
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Salamanca, España, 37007
- Aún no reclutando
- Hospital Universitario de Salamanca
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Seville, España, 41013
- Aún no reclutando
- Hospital Universitario Virgen del Rocio
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Barcelona
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Badalona, Barcelona, España, 08916
- Aún no reclutando
- Hospital Germans Trias i Pujol
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Galicia
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Santiago de Compostela, Galicia, España, 15706
- Aún no reclutando
- Hospital Clínico Universitario de Santiago
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Navarre
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Pamplona, Navarre, España, 31008
- Reclutamiento
- Clinica Universidad de Navarra
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Criterios de participación
Criterio de elegibilidad
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
Inclusion Criteria:
- Ability and willingness to provide written informed consent.
- Adults aged ≥18 and ≤65 years with a diagnosis of systemic lupus erythematosus (SLE) according to the 2019 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria.
- Positive antinuclear antibody (ANA) result at a titer >1:80, or positive anti-double stranded DNA (anti-dsDNA), or positive anti-Smith (anti-Sm) antibodies at screening.
- Diagnosis of class III or IV proliferative lupus nephritis, with or without concomitant class V disease, confirmed by renal biopsy demonstrating active lupus nephritis according to the 2018 ISN/RPS classification.
- Evidence of refractory or treatment-resistant lupus nephritis according to GLOSEN criteria.
- Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m² during the screening period.
- Urine protein-to-creatinine ratio (UPCR) >0.7 g/g, urine albumin-to-creatinine ratio (UACR) >0.5 g/g, proteinuria >0.7 g/24 h, or albuminuria >0.5 g/24 h at screening with evidence of active lupus nephritis.
- Stable treatment with an ACE inhibitor, angiotensin receptor blocker and/or mineralocorticoid receptor antagonist (with or without an SGLT2 inhibitor) for at least 3 months prior to screening, unless contraindicated or not tolerated.
- Adequate venous access and no contraindication to leukapheresis.
- Women of childbearing potential must have a negative pregnancy test at screening, prior to lymphodepletion and prior to infusion, and must agree to use highly effective contraception. Sexually active men must agree to use condoms and comply with protocol-specified reproductive precautions.
- Completion of recommended vaccinations, including SARS-CoV-2 vaccination/immunization, before study treatment.
- Ability and willingness to comply with all study procedures and follow-up requirements.
Exclusion Criteria:
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Planned initiation of renal replacement therapy during the study period or eGFR <30 mL/min/1.73 m².
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Severe organ dysfunction, including:
- Left ventricular ejection fraction (LVEF) <40%.
- Severe cardiac disease, including recent ischemic heart disease, NYHA class III-IV heart failure, uncontrolled arrhythmias, or severe lupus-related cardiac involvement.
- Significant hepatic impairment (ALT or AST >1.5× ULN, total bilirubin >1.5× ULN except specified exceptions, INR >1.5).
- Inadequate hematopoietic reserve (absolute neutrophil count ≤1000/µL, platelets <75,000/µL, leukocytes <3000/µL, lymphocytes ≤300/µL, hemoglobin <8 g/dL).
- Oxygen saturation <92% on room air.
- Severe pulmonary disease with compromised respiratory reserve.
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Active infection requiring treatment during screening or before lymphodepletion.
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Positive screening for HIV, hepatitis C virus, hepatitis B virus, or evidence of active tuberculosis.
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Grade ≥2 thromboembolic event within 4 weeks before screening.
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History of progressive multifocal leukoencephalopathy (PML) or symptoms suggestive of PML.
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Requirement for systemic glucocorticoids at doses ≥30 mg/day prednisone equivalent.
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Previous treatment with anti-CD19 CAR-T therapy.
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Known hypersensitivity or contraindication to TranspoCART19, fludarabine, cyclophosphamide, bendamustine, or required concomitant medications.
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Concurrent systemic autoimmune disease requiring immunosuppressive therapy independent of SLE treatment.
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Current or previous malignancy, except adequately treated non-melanoma skin cancer, carcinoma in situ, or malignancy in complete remission for more than 3 years.
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Previous solid organ transplantation, hematopoietic stem cell transplantation, or bone marrow transplantation.
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Planned major surgery within one year after study treatment.
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Receipt of live vaccines within 30 days before administration of the investigational product.
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Current drug or alcohol abuse that may interfere with study participation.
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Any severe or uncontrolled medical or psychiatric condition that, in the investigator's opinion, would increase risk or interfere with study participation.
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Pregnancy or breastfeeding.
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Women of childbearing potential unwilling to use highly effective contraception throughout the study period.
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Sexually active men unwilling to use condoms and follow reproductive precautions required by the protocol.
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Participation in another interventional clinical trial within 90 days before informed consent or receipt of another investigational product within the protocol-specified washout period.
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Inability or unwillingness to provide informed consent or comply with study requirements.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Número de brazos
Armas e Intervenciones
Grupo de participantes/brazoGrupo de participantes/brazo |
Intervención / TratamientoIntervención / Tratamiento |
|---|---|
|
Experimental: TranspoCART19
Participants with refractory lupus nephritis will undergo leukapheresis for manufacturing of autologous TranspoCART19 cells, followed by lymphodepleting chemotherapy and administration of TranspoCART19 as a fractionated intravenous infusion at a target dose of 1 × 10^6 CAR-T cells per kg. |
Autologous CD19-directed CAR-T cell therapy administered following lymphodepleting chemotherapy. After leukapheresis and manufacturing, participants receive TranspoCART19 as a fractionated intravenous infusion (10%, 30%, and 60% of the target dose) at a total target dose of 1 × 10^6 CAR-T cells/kg body weight. The product is manufactured using Sleeping Beauty transposon technology and consists of genetically modified T lymphocytes expressing an anti-CD19 chimeric antigen receptor. |
¿Qué mide el estudio?
Medidas de resultado primarias
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Number of Participants With Adverse Events and Serious Adverse Events Following TranspoCART19 Infusion.
Periodo de tiempo: Day 0 through Day 28 after infusion (extended through Day 42 for prolonged cytopenias).
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Assessment of safety and tolerability based on the incidence and severity of adverse events, serious adverse events, unacceptable toxicity, cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, severe infections, prolonged cytopenias, and hypogammaglobulinemia. |
Day 0 through Day 28 after infusion (extended through Day 42 for prolonged cytopenias).
|
Medidas de resultado secundarias
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Number of Participants Achieving Complete or Partial Clinical and Immunological Remission.
Periodo de tiempo: Day 56 after TranspoCART19 infusion.
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Proportion of participants achieving complete or partial clinical and immunological remission following TranspoCART19 infusion. |
Day 56 after TranspoCART19 infusion.
|
|
Number of Participants With Adverse Events and Serious Adverse Events During Long-Term Follow-up.
Periodo de tiempo: Baseline through Year 2
|
Incidence of adverse events, serious adverse events, vital sign abnormalities, physical examination findings, and laboratory abnormalities during long-term follow-up. |
Baseline through Year 2
|
|
Change From Baseline in Hematopoietic and Immunological Reconstitution Parameters.
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in hemoglobin, leukocyte count, platelet count, and immune cell subpopulations including CD3+, CD4+, CD8+, CD19+ B cells, CAR-T cells, NK cells, and NKT cells. |
Baseline through 24 months after infusion
|
|
Number and Percentage of Circulating CD19 CAR-T Cells
Periodo de tiempo: Baseline through 24 months after infusion
|
Number and percentage of circulating CD19 CAR-T cells in peripheral blood and their relationship with clinical response and adverse events. |
Baseline through 24 months after infusion
|
|
Change From Baseline in Serum Immunological Activity Markers.
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in ANA, anti-dsDNA, anti-histone, anti-SSA/Ro52, anti-SSB/La, anti-Sm, C3, C4, C1q, CH50, soluble BAFF, and total IgG levels. |
Baseline through 24 months after infusion
|
|
Change From Baseline in Renal Disease Activity Parameters.
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in estimated and measured glomerular filtration rate, microhematuria, proteinuria, albuminuria, and proportions of participants achieving complete or partial renal remission. |
Baseline through 24 months after infusion
|
|
Change From Baseline in Renal Histological Disease Markers
Periodo de tiempo: Baseline, Day 168, and Day 365
|
Changes in lupus nephritis activity index and chronicity indicators assessed by kidney biopsy, including immunohistochemical and immunofluorescence markers. |
Baseline, Day 168, and Day 365
|
|
Time to Complete or Partial Renal Clinical Remission
Periodo de tiempo: Baseline through 24 months after infusion
|
Time from TranspoCART19 infusion to achievement of complete or partial renal clinical remission. |
Baseline through 24 months after infusion
|
|
Reduction or Discontinuation of Corticosteroids and Immunosuppressive Therapy
Periodo de tiempo: Baseline through 24 months after infusion
|
Proportion of participants receiving prednisone doses of 5 mg/day or less or becoming free of immunosuppressive therapy and time required to achieve these outcomes. |
Baseline through 24 months after infusion
|
|
Change From Baseline in Systemic Lupus Erythematosus Disease Activity
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in SLEDAI-2K |
Baseline through 24 months after infusion
|
|
Change From Baseline in Systemic Lupus Erythematosus Disease Activity
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in BILAG-2004 |
Baseline through 24 months after infusion
|
|
Change From Baseline in Systemic Lupus Erythematosus Disease Activity
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in Physician Global Assessment |
Baseline through 24 months after infusion
|
|
Change From Baseline in Systemic Lupus Erythematosus Disease Activity
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in SLE Flare Index |
Baseline through 24 months after infusion
|
|
Change From Baseline in Systemic Lupus Erythematosus Disease Activity
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in DORIS remission criteria. |
Baseline through 24 months after infusion
|
|
Number of Participants Achieving Overall Treatment Response
Periodo de tiempo: Day 365 after TranspoCART19 infusion.
|
Proportion of participants achieving overall treatment response defined as clinical remission, histological remission, and immunological remission. |
Day 365 after TranspoCART19 infusion.
|
|
Change From Baseline in Health-Related Quality of Life.
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in SF-36v2 |
Baseline through 24 months after infusion
|
|
Change From Baseline in Health-Related Quality of Life.
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in EQ-5D-5L |
Baseline through 24 months after infusion
|
|
Change From Baseline in Health-Related Quality of Life.
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in FACIT-F |
Baseline through 24 months after infusion
|
|
Change From Baseline in Health-Related Quality of Life.
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in Patient Global Assessment |
Baseline through 24 months after infusion
|
|
Change From Baseline in Health-Related Quality of Life.
Periodo de tiempo: Baseline through 24 months after infusion
|
Changes from baseline in LupusQoL scores. |
Baseline through 24 months after infusion
|
Colaboradores e Investigadores
Patrocinador
Patrocinador
Investigadores
Investigadores
- Investigador principal: Alberto Ortiz Arduan, Hospital Universitario Fundación Jiménez Díaz. IIS-FJD.
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Inicio del estudio
Finalización primaria (Estimado)
Finalización primaria
Finalización del estudio (Estimado)
Finalización del estudio
Fechas de registro del estudio
Enviado por primera vez
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Publicado por primera vez
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización publicada
Última actualización enviada que cumplió con los criterios de control de calidad
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
- Enfermedades urogenitales
- Enfermedades urogenitales masculinas
- Enfermedades Renales
- Enfermedades urológicas
- Enfermedades urogenitales femeninas
- Enfermedades urogenitales femeninas y complicaciones del embarazo
- Enfermedades del tejido conectivo
- Enfermedades del sistema inmunológico
- Glomerulonefritis
- Nefritis
- Enfermedades de la piel y del tejido conectivo
- Lupus Eritematoso Sistémico
- Nefritis lúpica
- Enfermedades autoinmunes
Otros números de identificación del estudio
Otros números de identificación del estudio
- CART-NEL
- ICI24/00034 (Otro número de subvención/financiamiento: Instituto de Salud Carlos III)
- 2025-522449-23-00 (Ctis)
- 2025 (Subvención/contrato del NIH de EE. UU.)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Información sobre medicamentos y dispositivos, documentos del estudio
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Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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