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

2026년 5월 22일 업데이트: 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.

연구 개요

상세 설명

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.

연구 유형

중재적

등록 (추정된)

12

단계

  • 1단계

연락처 및 위치

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연구 연락처

연구 연락처 백업

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

  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

아니

설명

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

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: 무작위화되지 않음
  • 중재 모델: 병렬 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
활성 비교기: 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.
활성 비교기: 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.
활성 비교기: 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.

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Incidence of dose-limiting toxicities (DLT occurring within 28 days of CAR-T cell infusion)
기간: Within 28 days of CAR-T cell infusion
DLT
Within 28 days of CAR-T cell infusion

2차 결과 측정

결과 측정
측정값 설명
기간
Manufacturing success rate
기간: 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
기간: At 24 months
Treatment adverse events related to the administration of CART-cells
At 24 months
ORR per IMWG criteria
기간: At 24 months
Overall response rate
At 24 months
DOR
기간: At day 24 months
Duration of Response
At day 24 months
PFS
기간: At 24 months
Progression-Free Survival
At 24 months
OS
기간: At 24 months
Overall Survival
At 24 months

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간행물 및 유용한 링크

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일반 간행물

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 (추정된)

2026년 9월 1일

기본 완료 (추정된)

2028년 9월 30일

연구 완료 (추정된)

2028년 12월 30일

연구 등록 날짜

최초 제출

2026년 5월 13일

QC 기준을 충족하는 최초 제출

2026년 5월 22일

처음 게시됨 (실제)

2026년 5월 28일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 5월 28일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 5월 22일

마지막으로 확인됨

2026년 5월 1일

추가 정보

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미국 FDA 규제 의약품 연구

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아니

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