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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.

연구 개요

상태

모병

정황

개입 / 치료

상세 설명

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.

연구 유형

중재적

등록 (추정된)

10

단계

  • 2 단계
  • 1단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 연락처 백업

연구 장소

      • León, 스페인, 24008
        • 아직 모집하지 않음
        • Hospital Universitario de León
      • Madrid, 스페인, 28040
        • 아직 모집하지 않음
        • Hospital Universitario Fundación Jiménez Diaz
      • Murcia, 스페인, 30120
        • 아직 모집하지 않음
        • Hospital Clínico Universitario Virgen de la Arrixaca
      • Salamanca, 스페인, 37007
        • 아직 모집하지 않음
        • Hospital Universitario de Salamanca
      • Seville, 스페인, 41013
        • 아직 모집하지 않음
        • Hospital Universitario Virgen del Rocio
    • Barcelona
      • Badalona, Barcelona, 스페인, 08916
        • 아직 모집하지 않음
        • Hospital Germans Trias i Pujol
    • Galicia
      • Santiago de Compostela, Galicia, 스페인, 15706
        • 아직 모집하지 않음
        • Hospital Clínico Universitario de Santiago
    • Navarre
      • Pamplona, Navarre, 스페인, 31008
        • 모병
        • Clinica Universidad de Navarra

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

18년 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

Inclusion Criteria:

  1. Ability and willingness to provide written informed consent.
  2. 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.
  3. 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.
  4. 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.
  5. Evidence of refractory or treatment-resistant lupus nephritis according to GLOSEN criteria.
  6. Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m² during the screening period.
  7. 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.
  8. 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.
  9. Adequate venous access and no contraindication to leukapheresis.
  10. 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.
  11. Completion of recommended vaccinations, including SARS-CoV-2 vaccination/immunization, before study treatment.
  12. Ability and willingness to comply with all study procedures and follow-up requirements.

Exclusion Criteria:

  1. Planned initiation of renal replacement therapy during the study period or eGFR <30 mL/min/1.73 m².

  2. 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.
  3. Active infection requiring treatment during screening or before lymphodepletion.

  4. Positive screening for HIV, hepatitis C virus, hepatitis B virus, or evidence of active tuberculosis.

  5. Grade ≥2 thromboembolic event within 4 weeks before screening.

  6. History of progressive multifocal leukoencephalopathy (PML) or symptoms suggestive of PML.

  7. Requirement for systemic glucocorticoids at doses ≥30 mg/day prednisone equivalent.

  8. Previous treatment with anti-CD19 CAR-T therapy.

  9. Known hypersensitivity or contraindication to TranspoCART19, fludarabine, cyclophosphamide, bendamustine, or required concomitant medications.

  10. Concurrent systemic autoimmune disease requiring immunosuppressive therapy independent of SLE treatment.

  11. Current or previous malignancy, except adequately treated non-melanoma skin cancer, carcinoma in situ, or malignancy in complete remission for more than 3 years.

  12. Previous solid organ transplantation, hematopoietic stem cell transplantation, or bone marrow transplantation.

  13. Planned major surgery within one year after study treatment.

  14. Receipt of live vaccines within 30 days before administration of the investigational product.

  15. Current drug or alcohol abuse that may interfere with study participation.

  16. Any severe or uncontrolled medical or psychiatric condition that, in the investigator's opinion, would increase risk or interfere with study participation.

  17. Pregnancy or breastfeeding.

  18. Women of childbearing potential unwilling to use highly effective contraception throughout the study period.

  19. Sexually active men unwilling to use condoms and follow reproductive precautions required by the protocol.

  20. Participation in another interventional clinical trial within 90 days before informed consent or receipt of another investigational product within the protocol-specified washout period.

  21. Inability or unwillingness to provide informed consent or comply with study requirements.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 해당 없음
  • 중재 모델: 단일 그룹 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: 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.

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Number of Participants With Adverse Events and Serious Adverse Events Following TranspoCART19 Infusion.
기간: Day 0 through Day 28 after infusion (extended through Day 42 for prolonged cytopenias).

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).

2차 결과 측정

결과 측정
측정값 설명
기간
Number of Participants Achieving Complete or Partial Clinical and Immunological Remission.
기간: Day 56 after TranspoCART19 infusion.

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.
기간: 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.
기간: 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
기간: 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.
기간: 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.
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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.
기간: 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.
기간: 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.
기간: 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.
기간: 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.
기간: Baseline through 24 months after infusion

Changes from baseline in LupusQoL scores.

Baseline through 24 months after infusion

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

수사관

  • 수석 연구원: Alberto Ortiz Arduan, Hospital Universitario Fundación Jiménez Díaz. IIS-FJD.

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2026년 9월 23일

기본 완료 (추정된)

2029년 9월 1일

연구 완료 (추정된)

2030년 9월 1일

연구 등록 날짜

최초 제출

2026년 8월 25일

QC 기준을 충족하는 최초 제출

2026년 8월 28일

처음 게시됨 (실제)

2026년 9월 2일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 10월 1일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 9월 30일

마지막으로 확인됨

2026년 9월 1일

추가 정보

이 연구와 관련된 용어

기타 연구 ID 번호

  • CART-NEL
  • ICI24/00034 (기타 보조금/기금 번호: Instituto de Salud Carlos III)
  • 2025-522449-23-00 (씨티스)
  • 2025 (미국 NIH 보조금/계약)

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

아니

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .