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

2026年5月22日 更新者: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.

調査の概要

詳細な説明

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.

研究の種類

介入

入学 (推定)

12

段階

  • フェーズ 1

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究連絡先

研究連絡先のバックアップ

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

  • 大人
  • 高齢者

健康ボランティアの受け入れ

いいえ

説明

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

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか?

デザインの詳細

  • 主な目的:処理
  • 割り当て:非ランダム化
  • 介入モデル:並列代入
  • マスキング:なし(オープンラベル)

武器と介入

参加者グループ / アーム
介入・治療
アクティブコンパレータ: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.
アクティブコンパレータ: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.
アクティブコンパレータ: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.

この研究は何を測定していますか?

主要な結果の測定

結果測定
メジャーの説明
時間枠
Incidence of dose-limiting toxicities (DLT occurring within 28 days of CAR-T cell infusion)
時間枠:Within 28 days of CAR-T cell infusion
DLT
Within 28 days of CAR-T cell infusion

二次結果の測定

結果測定
メジャーの説明
時間枠
Manufacturing success rate
時間枠: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
時間枠:At 24 months
Treatment adverse events related to the administration of CART-cells
At 24 months
ORR per IMWG criteria
時間枠:At 24 months
Overall response rate
At 24 months
DOR
時間枠:At day 24 months
Duration of Response
At day 24 months
PFS
時間枠:At 24 months
Progression-Free Survival
At 24 months
OS
時間枠:At 24 months
Overall Survival
At 24 months

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出版物と役立つリンク

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一般刊行物

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (推定)

2026年9月1日

一次修了 (推定)

2028年9月30日

研究の完了 (推定)

2028年12月30日

試験登録日

最初に提出

2026年5月13日

QC基準を満たした最初の提出物

2026年5月22日

最初の投稿 (実際)

2026年5月28日

学習記録の更新

投稿された最後の更新 (実際)

2026年5月28日

QC基準を満たした最後の更新が送信されました

2026年5月22日

最終確認日

2026年5月1日

詳しくは

本研究に関する用語

医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

はい

米国FDA規制機器製品の研究

いいえ

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