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Study to Evaluate the Safety of UF-KURE-BCMA CAR T-Cells in Advanced Myeloma

22 de mayo de 2026 actualizado por: Kure Cells, INC

A Phase 1, Single-Arm, Open-Label Study to Evaluate the Safety of UF-KURE-BCMA Cells in Patients With Relapsed or Refractory Multiple Myeloma

The goal of this study is to evaluate the safety of a new type of CAR T-cell, UF-KURE-BCMA, for the treatment of patients with advanced multiple myeloma that has not responded to other therapies. The main question is whether the use of these new CAR T-cells is safe for patients with this condition. Secondarily, the study will also look at the response of myeloma to this therapy.

Descripción general del estudio

Descripción detallada

This is a Phase I, single-arm, open-label, dose-escalation study using a 3+3 design that evaluates the safety of UF (ultrafast)- KURE-BCMA for patients with relapsed/refractory multiple myeloma. Eligible patients are adults (≥18 years) with relapsed or refractory multiple myeloma after ≥3 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody. The study will accrue a total of 12 patients.

The primary objectives of this study are:

  • To determine the recommended Phase 2 dose (RP2D) of UF-KURE-BCMA
  • To establish the safety profile and identify dose-limiting toxicities (DLTs)

The secondary objectives:

  • Manufacturing feasibility
  • Overall response rate (ORR) per IMWG criteria
  • Duration of response (DOR)
  • Progression-free survival (PFS)
  • Overall survival (OS)

The exploratory objectives include:

  • CAR-T cell persistence and expansion
  • Replication-competent lentivirus (RCL) testing
  • Cytokine profiling
  • CAR-T cell phenotype characterization
  • Anti-BCMA CAR antibody development
  • Spatial multi-omics analysis of the bone marrow microenvironment

The study endpoints include:

Primary Endpoint Incidence of dose-limiting toxicities occurring within 28 days of CAR-T cell infusion Secondary Endpoints

  • Manufacturing success rate (≥75%)
  • Treatment-emergent adverse events
  • ORR, DOR, PFS, and OS per IMWG criteria

The Investigational Product is UF-KURE-BCMA, an autologous CAR-T cells manufactured using an ultra-fast process (approximately 17-20 hours), that targets specifically BCMA, a marker expressed on the surface of the myeloma cell. The study will evaluate 3 different dose levels:

  • Level -1: 3 × 10⁶ cells (≥50 kg) / 2 × 10⁶ cells (<50 kg)
  • Level 1 (Starting Dose): 10 × 10⁶ cells (≥50 kg) / 7 × 10⁶ cells (<50 kg)
  • Level 2: 15 × 10⁶ cells (≥50 kg) / 10 × 10⁶ cells (<50 kg) Prior to the cell infusion, the patients will receive a chemotherapy regimen (lymphodepleting regimen) that includes: Cyclophosphamide 300 mg/m² IV plus fludarabine 30 mg/m² IV, administered daily for 3 days (Days -4 to -2) The study will last 28 days, which is Dose-limiting toxicity (DLT) observation period. The follow-up period is 24 months with a long-term safety follow-up of 15 years.

The study endpoints:

Primary Endpoint Incidence of dose-limiting toxicities occurring within 28 days of CAR-T cell infusion Secondary Endpoints

  • Manufacturing success rate (≥75%)
  • Treatment-emergent adverse events
  • ORR, DOR, PFS, and OS per IMWG criteria

Statistical Methods A standard 3+3 dose-escalation design will be used. Safety and efficacy will be summarized using descriptive statistics. Time-to-event endpoints will be analyzed using the Kaplan-Meier method.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

12

Fase

  • Fase 1

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Copia de seguridad de contactos de estudio

  • Nombre: Ola Soliman, MD
  • Número de teléfono: 6478650773
  • Correo electrónico: olaselkadi@gmail.com

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Adulto
  • Adulto Mayor

Acepta Voluntarios Saludables

No

Descripción

Inclusion Criteria:

Subjects must meet ALL of the following criteria to be eligible for study enrollment:

  1. Age: ≥18 years at time of signing informed consent
  2. Diagnosis: Documented multiple myeloma meeting one of the following:

    • Relapsed disease: Progression after achieving at least minimal response (MR) to prior therapy
    • Refractory disease: Non-responsive or progressive disease while on therapy or within 60 days of last treatment (in subjects who achieved ≥MR on prior therapy)
  3. Prior Therapy:

    • Received ≥3 prior lines of anti-myeloma therapy
    • Prior therapy must include:

    At least one proteasome inhibitor At least one immunomodulatory drug (e.g., lenalidomide, pomalidomide, thalidomide) At least one anti-CD38 monoclonal antibody (e.g., daratumumab, isatuximab) o Prior anti-BCMA CAR-T therapy is permitted if subject achieved PFS ≥6 months post-infusion

  4. Measurable Disease: At least one of the following at screening (for response assessment eligibility):

    • Serum M-protein ≥0.5 g/dL by protein electrophoresis (SPEP)
    • Urine M-protein ≥200 mg/24 hours by protein electrophoresis (UPEP)
    • Serum free light chain (FLC) difference ≥10 mg/dL with abnormal FLC ratio Note: Subjects with non-measurable disease may enroll for safety assessment
  5. Performance Status: ECOG Performance Status 0-2 (see Appendix A)
  6. Organ Function: Adequate organ function as defined by:

    Hepatic:

    o Total bilirubin ≤2× institutional upper limit of normal (ULN), except subjects with Gilbert's syndrome

    • AST and ALT ≤2.5× institutional ULN

    Renal:

    o Calculated creatinine clearance ≥30 mL/min (Cockcroft-Gault formula)

    Cardiac:

    o Left ventricular ejection fraction (LVEF) ≥45% by echocardiogram or MUGA

    Pulmonary:

    o ≤Grade 1 dyspnea

    o Oxygen saturation ≥92% on room air

    o If PFTs performed: FEV₁ ≥50% predicted and DLCO ≥40% predicted (corrected for hemoglobin)

  7. Prior Therapy Washout:

    o ≥2 weeks since last radiation or systemic anti-myeloma therapy (standard agents)

    o ≥4 weeks since last investigational therapy

    o ≥6 weeks since autologous stem cell transplant

  8. Informed Consent: Ability to understand and willingness to provide written informed consent
  9. Contraception Requirements (for subjects of reproductive potential):

Female subjects:

o Women of childbearing potential must: Have negative serum pregnancy test at screening Agree to use highly effective contraception (failure rate <1% per year) from enrollment through 6 months post-CAR-T infusion

  • Acceptable methods: bilateral tubal ligation, male partner sterilization, hormonal contraceptives that inhibit ovulation, hormone-releasing IUD, copper IUD
  • Sexual abstinence is acceptable if consistent with subject's preferred lifestyle

Male subjects:

  • Must agree to use condom plus effective contraception if partner is of childbearing potential
  • Must refrain from sperm donation from enrollment through 6 months post-CAR-T infusion

Exclusion Criteria:

Subjects meeting ANY of the following criteria will be excluded:

  1. Disease-Specific Exclusions:

    • Active CNS involvement by multiple myeloma
    • Plasma cell leukemia
    • History of allogeneic hematopoietic stem cell transplantation
  2. Malignancy Exclusions:

    o Second active malignancy, except: Non-melanoma skin cancer Carcinoma in situ (cervix, bladder, breast) Stage 1 uterine cancer Localized prostate cancer

  3. Cardiovascular Exclusions:

    • New York Heart Association (NYHA) Class IV congestive heart failure
    • Unstable angina pectoris
    • Clinically significant cardiac arrhythmias
    • Myocardial infarction, stroke, or TIA within 6 months of enrollment
  4. Infectious Disease Exclusions:

    • Known HIV infection or AIDS-related illness
    • Active hepatitis B or C infection:

Positive HBsAg, or Positive anti-HBc or anti-HCV with detectable viral nucleic acid by PCR

  • Active infection requiring systemic therapy 5. Neurological Exclusions:
  • History of clinically relevant CNS pathology including:

Epilepsy or seizure disorders Paresis, aphasia Uncontrolled cerebrovascular disease Severe brain injury Dementia Parkinson's disease 6. Autoimmune Disease:

  • Active autoimmune disease requiring systemic immunosuppression >15 mg/day prednisone equivalent within past 6 months
  • Examples: rheumatoid arthritis, lupus

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Comparador activo: Lower Dose Level
Lower Dose (Level -1): 3 × 10⁶ cells (≥50 kg)// 2 × 10⁶ cells (<50 kg)
The patients will receive one of 3 dose levels as outlined above.
Comparador activo: Starting Dose Level
Starting Dose Level (Level 1) : 10 × 10⁶ cells (≥50 kg) / 7 × 10⁶ cells (<50 kg)
The patients will receive one of 3 dose levels as outlined above.
Comparador activo: Higher Dose Level
Higher Dose Level (Level 2): 15 × 10⁶ cells (≥50 kg) / 10 × 10⁶ cells (<50 kg)
The patients will receive one of 3 dose levels as outlined above.

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Incidence of dose-limiting toxicities (DLT occurring within 28 days of CAR-T cell infusion)
Periodo de tiempo: Within 28 days of CAR-T cell infusion
DLT
Within 28 days of CAR-T cell infusion

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Manufacturing success rate
Periodo de tiempo: 28 days post infusion
Manufacturing success rate is defined as manufacturing process leading to an adequate product per protocol standards. We expect this outcome to occur in ≥75% of the products manufactured.
28 days post infusion
Treatment-emergent adverse events
Periodo de tiempo: At 24 months
Treatment adverse events related to the administration of CART-cells
At 24 months
ORR per IMWG criteria
Periodo de tiempo: At 24 months
Overall response rate
At 24 months
DOR
Periodo de tiempo: At day 24 months
Duration of Response
At day 24 months
PFS
Periodo de tiempo: At 24 months
Progression-Free Survival
At 24 months
OS
Periodo de tiempo: At 24 months
Overall Survival
At 24 months

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Publicaciones Generales

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Estimado)

1 de septiembre de 2026

Finalización primaria (Estimado)

30 de septiembre de 2028

Finalización del estudio (Estimado)

30 de diciembre de 2028

Fechas de registro del estudio

Enviado por primera vez

13 de mayo de 2026

Primero enviado que cumplió con los criterios de control de calidad

22 de mayo de 2026

Publicado por primera vez (Actual)

28 de mayo de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

28 de mayo de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

22 de mayo de 2026

Última verificación

1 de mayo de 2026

Más información

Términos relacionados con este estudio

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

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