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- Ensayo clínico NCT07838714
Dual-Targeted Umbilical Cord Blood CAR-T Cells Against CD19 and CD22 for the Treatment of Relapsed or Refractory Acute B-Cell Lymphoblastic Leukemia
A Single-Center Phase I Clinical Trial of Dual-Targeted Umbilical Cord Blood CAR-T Cells Against CD19 and CD22 for the Treatment of Relapsed or Refractory Acute B-Cell Lymphoblastic Leukemia
This investigator-initiated, prospective, single-arm, phase I dose-escalation trial aims to evaluate the safety and tolerability of anti-CD19/CD22 dual-target cord blood-derived CAR-T cells in adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia who have failed prior therapies, with the goal of reducing antigen-escape relapse and overcoming poor autologous T-cell fitness through the use of universally available umbilical cord blood T cells.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
Patients with relapsed/refractory B-cell acute lymphoblastic leukemia who meet eligibility criteria will receive fludarabine (30 mg/m²/d) and cyclophosphamide (300 mg/m²/d) for three consecutive days as lymphodepleting conditioning, followed by a single intravenous infusion of anti-CD19/CD22 dual-target cord blood-derived CAR-T cells. Dose assignment follows a "3+3" escalation design across three dose levels (4×10⁶, 8×10⁶, and 12×10⁶ CAR-T/kg, ±20%). Dose-limiting toxicity will be assessed during the first 14 days post-infusion to determine the maximum tolerated dose. After CAR-T infusion, patients will be monitored for safety and efficacy at protocol-specified time points through 3 months, with long-term follow-up continuing up to 15 years post-infusion. A total of 18 patients will be enrolled.
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 1
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Weiyang Liu
- Número de teléfono: +86 13817506393
- Correo electrónico: lwy21758760@sjtu.edu.cn
Ubicaciones de estudio
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Shanghai Municipality
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Shanghai, Shanghai Municipality, Porcelana, 200025
- Reclutamiento
- Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
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Contacto:
- Jin Wang, PhD
- Número de teléfono: +86 13524945202
- Correo electrónico: jinwang@shsmu.edu.cn
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
Inclusion Criteria:
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The patient or their legally authorized guardian has signed the informed consent form (ICF), indicating understanding of the purpose and procedures of this clinical trial and willingness to participate.
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Age between 18 and 75 years, male or female.
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Diagnosis of relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-ALL) according to the Chinese Guidelines for Diagnosis and Treatment of Adult Acute Lymphoblastic Leukemia (2024 edition).
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Leukemic cells confirmed to express CD19 and/or CD22 by flow cytometry.
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ECOG performance status 0-2.
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Life expectancy ≥12 weeks.
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Adequate organ function at screening, meeting all of the following laboratory criteria:
- Hematology: absolute neutrophil count (ANC) ≥1×10⁹/L; absolute lymphocyte count (ALC) ≥0.3×10⁹/L; platelet count ≥20×10⁹/L; hemoglobin ≥60 g/L.
- Hepatic function: ALT and AST ≤2.5× upper limit of normal (ULN); total bilirubin ≤1.5× ULN.
- Renal function: creatinine clearance (CrCl) ≥40 mL/min (by Cockcroft-Gault formula).
- Coagulation: fibrinogen ≥1.0 g/L; activated partial thromboplastin time (APTT) ≤1.5× ULN; prothrombin time (PT) ≤1.5× ULN.
- Oxygen saturation >91%.
- Left ventricular ejection fraction (LVEF) ≥50%.
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The subject and their spouse agree to use effective contraceptive measures (excluding rhythm method) from ICF signing through 1 year after CAR-T cell infusion.
Exclusion Criteria:
- Active graft-versus-host disease (GVHD) or autoimmune disease requiring long-term immunosuppressive therapy.
- Use of therapeutic doses of corticosteroids (defined as prednisone or equivalent >20 mg/day) within 7 days prior to screening. Physiologic replacement, topical, and inhaled steroids are permitted.
- Hypertension not controllable with medication.
- Severe cardiac disease, including but not limited to: unstable angina, myocardial infarction (within 6 months prior to screening), congestive heart failure (NYHA class ≥III), or severe arrhythmia.
- Unstable systemic disease as judged by the investigator, including but not limited to: severe hepatic, renal, or metabolic disease requiring medication.
- Malignancy other than B-ALL within 5 years prior to screening, except for adequately treated carcinoma in situ of the cervix, basal cell or squamous cell skin cancer, localized prostate cancer after radical surgery, or ductal carcinoma in situ of the breast after radical surgery.
- History of solid organ transplantation.
- Planned surgery within 2 weeks after study treatment (subjects scheduled for local anesthesia surgery may participate).
- Receipt of other interventional investigational drugs within 1 month prior to ICF signing.
- Uncontrolled active infection.
- Positive for HBsAg, or positive for HBcAb with detectable HBV DNA in peripheral blood; positive for HCV antibody with detectable HCV RNA; positive for HIV antibody; positive for CMV DNA; positive for syphilis serology.
- Pregnant or breastfeeding women.
- Psychiatric illness, consciousness disorder, or central nervous system disease.
- Other conditions deemed unsuitable for enrollment by the investigator.
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)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Experimental: Anti-CD19/CD22 Dual-Target Cord Blood CAR-T Cells
A single intravenous infusion of anti-CD19/CD22 dual-target umbilical cord blood-derived CAR-T cells administered on Day 0, following lymphodepleting chemotherapy with fludarabine (30 mg/m²/day) and cyclophosphamide (300 mg/m²/day) for three consecutive days. Dose escalation follows a 3+3 design across three dose levels (4.0×10⁶, 8.0×10⁶, and 12.0×10⁶ CAR-T/kg, ±20%). All subjects receive the same investigational product; no comparator arm is included. |
Genetically modified umbilical cord blood-derived T cells expressing chimeric antigen receptors targeting both CD19 and CD22. Administered as a single intravenous infusion on Day 0 following lymphodepleting chemotherapy. Dose levels: 4.0×10⁶, 8.0×10⁶, and 12.0×10⁶ CAR-T/kg (±20%) according to 3+3 dose escalation design. 30 mg/m² intravenous infusion over 30 minutes once daily for 3 consecutive days as lymphodepleting preconditioning prior to CAR-T cell infusion. 300 mg/m² intravenous infusion over 30 minutes once daily for 3 consecutive days as lymphodepleting preconditioning prior to CAR-T cell infusion. |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Incidence and Severity of Dose-Limiting Toxicity (DLT)
Periodo de tiempo: Within 14 days after CAR-T cell infusion
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DLT is defined as treatment-related adverse events occurring within 14 days post-infusion. Non-hematologic DLT: Grade ≥3 toxicity not reducible to ≤ Grade 1 within 72 hours. Hematologic DLT: Grade 4 toxicity (excluding lymphopenia) persisting >21 days, not attributable to underlying disease. Predefined exclusion criteria include tumor lysis syndrome, electrolyte disturbances, hypogammaglobulinemia, transient laboratory abnormalities, febrile neutropenia, and others per protocol. CRS/ICANS graded per ASTCT 2019, aGVHD per modified Glucksberg, other AEs per CTCAE v5.0. |
Within 14 days after CAR-T cell infusion
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Objective Response Rate (ORR)
Periodo de tiempo: 1 and 3 months post-infusion
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ORR is the proportion of subjects achieving CR/CRi at 1 and 3 months post-infusion. Efficacy assessed by bone marrow examination per international acute leukemia response criteria. |
1 and 3 months post-infusion
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Minimal Residual Disease (MRD) Negativity Rate
Periodo de tiempo: 1 and 3 months post-infusion
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MRD negativity rate is the proportion of subjects achieving MRD-negative status by flow cytometry at 1 and 3 months post-infusion. |
1 and 3 months post-infusion
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Complete Remission (CR) Rate
Periodo de tiempo: 1 and 3 months post-infusion
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CR rate is the proportion achieving CR. Efficacy assessed by bone marrow examination per international acute leukemia response criteria. |
1 and 3 months post-infusion
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Progression-Free Survival (PFS)
Periodo de tiempo: From infusion up to 24 months
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PFS is defined as time from infusion to disease progression, relapse, or death. Median PFS and 12-/24-month PFS rates will be estimated by Kaplan-Meier method. |
From infusion up to 24 months
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Overall Survival (OS)
Periodo de tiempo: From infusion up to 24 months
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OS is defined as time from infusion to death from any cause. Median OS and 12-/24-month OS rates will be estimated by Kaplan-Meier method. |
From infusion up to 24 months
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CAR-T Cell Expansion and Persistence
Periodo de tiempo: Up to 90 days post-infusion
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CAR-T cell levels and vector copy number (VCN) in peripheral blood will be measured. |
Up to 90 days post-infusion
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CAR-T Cell Expansion and Persistence
Periodo de tiempo: Up to 90 days post-infusion
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PK parameters: Cmax(Peak Plasma Concentration) |
Up to 90 days post-infusion
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CAR-T Cell Expansion and Persistence
Periodo de tiempo: Up to 90 days post-infusion
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PK parameters: Tmax |
Up to 90 days post-infusion
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CAR-T Cell Expansion and Persistence
Periodo de tiempo: Up to 90 days post-infusion
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PK parameters: AUC₀-₂₈d, AUC₀-₉₀d. |
Up to 90 days post-infusion
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CAR-T Cell Expansion and Persistence
Periodo de tiempo: At 3 and 6 months
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Proportion of subjects with detectable CAR-T cells at 3 and 6 months, and duration of persistence will be recorded. |
At 3 and 6 months
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Colaboradores e Investigadores
Patrocinador
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
- Neoplasias
- Enfermedades del sistema inmunológico
- Infecciones
- Enfermedades virales
- Neoplasias por tipo histológico
- Infecciones por virus de ADN
- Enfermedades linfáticas
- Trastornos linfoproliferativos
- Trastornos inmunoproliferativos
- Linfoma No Hodgkin
- Linfoma de células B
- Linfoma
- Infecciones por el virus de Epstein-Barr
- Infecciones por herpesviridae
- Infecciones por virus tumorales
- Enfermedades hemic y linfáticas
- Linfoma de Burkitt
- Químicos orgánicos
- Hidrocarburos
- Mostaza de fosforamida
- Compuestos de mostaza de nitrógeno
- Compuestos de mostaza
- Hidrocarburos, halogenados
- Fosforamidas
- Compuestos organofosforados
- Ciclofosfamida
- fludarabina
Otros números de identificación del estudio
- RJ-ALL 2026-B01
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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