- ICH GCP
- 미국 임상 시험 레지스트리
- 임상시험 NCT04720313
NXC-201 (Formerly HBI0101) Multiple Myeloma
A Phase 1 Dose Escalation and Safety Study of NXC-201 (Formerly HBI0101) CART in BCMA-Expressing Multiple Myeloma Patients
It is a phase one study with dose escalation and safety CART in BCMA- Expressing Multiple Myeloma and AL amyloidosis Patients
연구 개요
상세 설명
The intention with NXC-201 (formerly HBI0101) CART is to follow the chimeric antigen receptor T-cells (CART) approach, as for approved products, but target the B cell maturation antigen (BCMA) rather than the CD19 antigen targeted by KYMRIAHTM (tisagenlecleucel) and YESCARTATM (axicabtagene ciloleucel).
Importantly, successful results from at least three clinical trials of a BCMA targeted CAR T therapy were published (Zhao 2018, Brundo 2018, Raje 2019), with excellent results obtained for relapsed or refractory multiple myeloma (MM) patients, that validate the approach.
연구 유형
등록 (추정된)
단계
- 1단계
연락처 및 위치
연구 장소
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Jerusalem
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Jerusalem, Jerusalem, 이스라엘, 91120
- Hadassah University Hospital
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
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≥18 years of age
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Voluntarily signed informed consent form (ICF)
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Eastern Cooperative Oncology Group (ECOG) performance status 0 - 2
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Diagnosis of MM with relapsed or refractory disease and have had at least 3 different prior lines of therapy including proteasome inhibitor, immunomodulatory therapy and at least one antibody therapy.
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Subjects must have measurable disease, including at least one of the criteria below:
- Serum M-protein greater or equal to 0.5 g/dL
- Urine M-protein greater or equal to 200 mg/24 h
- Serum free light chain (FLC) assay: involved FLC level greater or equal to 5 mg/dL (50 mg/L) provided serum FLC ratio is abnormal
- A biopsy-proven evaluable plasmacytoma
- Bone marrow plasma cells > 20% of total bone marrow cells
- Non secretory patient will be allowed provided they have measurable disease by PET-CT or bone marrow aspiration, as designated.
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Women of child-bearing potential (WCBP), must have a negative serum pregnancy test prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use effective methods of birth control throughout the study
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Recovery to ≤Grade 2 or baseline of any non-hematologic toxicities due to prior treatments, excluding alopecia and Grade 3 neuropathy
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Ability and willingness to adhere to the study visit schedule and all protocol requirements
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공부 계획
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: CART BCMA
The dose escalation phase (Part A) will include the following doses of CAR-positive (CAR+) T cells: 150×10^6, 450×10^6, 800×10^6 or 1200 ×10^6 The expansion phase (Part B) will include a dose between 450×10^6 to 800×10^6 CAR-positive (CAR+) T cells |
NXC-201 (formerly HBI0101) CART is defined as autologous T cells transduced ex-vivo with anti-BCMA CAR retroviral vector encoding the chimeric antigen receptor (CAR) targeted to human BCMA. The NXC-201 (formerly HBI0101) CART is provided fresh without cryopreservation. |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Determination of MTD
기간: 21 days
|
Part A: Determination of MTD Part B: Confirmation of selected dose tested (at or below MTD) ( safety ) |
21 days
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
The overall survival
기간: 2 years
|
according to the IMWG Uniform Response Criteria for Multiple Myeloma |
2 years
|
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The progression-free survival
기간: 2 years
|
according to the IMWG Uniform Response Criteria for Multiple Myeloma |
2 years
|
공동 작업자 및 조사자
협력자
수사관
- 수석 연구원: Polina Stepensky, prof., Hadassah university hospital of Jerusalem
간행물 및 유용한 링크
일반 간행물
- Kfir-Erenfeld S, Asherie N, Grisariu S, Avni B, Zimran E, Assayag M, Sharon TD, Pick M, Lebel E, Shaulov A, Cohen YC, Avivi I, Cohen CJ, Stepensky P, Gatt ME. Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis. Clin Cancer Res. 2022 Dec 1;28(23):5156-5166. doi: 10.1158/1078-0432.CCR-22-0637.
- Lebel E, Asherie N, Kfir-Erenfeld S, Grisariu S, Avni B, Elias S, Assayag M, Dubnikov-Sharon T, Pick M, Alexander-Shani R, Bessig N, Herr S, Shehadeh A, Ishtay A, Pimienta S, Vainstein V, Zimran E, Cohen Y, Avivi I, Cohen C, Stepensky P, Gatt ME. Efficacy and Safety of Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor T-Cell for the Treatment of Relapsed and Refractory AL Amyloidosis. J Clin Oncol. 2025 Jun 10;43(17):2007-2016. doi: 10.1200/JCO-24-02252. Epub 2024 Dec 9.
- Kfir-Erenfeld S, Asherie N, Lebel E, Vainstein V, Assayag M, Dubnikov Sharon T, Grisariu S, Avni B, Elias S, Alexander-Shani R, Bessig N, Shehadeh A, Ishtay A, Zelmanovich V, Zimran E, Pick M, Roziner I, Kenett RS, Cohen Y, Avivi I, Cohen CJ, Gatt ME, Stepensky P. Clinical evaluation and determinants of response to HBI0101 (BCMA CART) therapy in relapsed/refractory multiple myeloma. Blood Adv. 2024 Aug 13;8(15):4077-4088. doi: 10.1182/bloodadvances.2024012967.
- Asherie N, Kfir-Erenfeld S, Avni B, Assayag M, Dubnikov T, Zalcman N, Lebel E, Zimran E, Shaulov A, Pick M, Cohen Y, Avivi I, Cohen C, Gatt ME, Grisariu S, Stepensky P. Development and manufacture of novel locally produced anti-BCMA CAR T cells for the treatment of relapsed/refractory multiple myeloma: results from a phase I clinical trial. Haematologica. 2023 Jul 1;108(7):1827-1839. doi: 10.3324/haematol.2022.281628.
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
기타 연구 ID 번호
- MOH_2020-12-22_009584
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
There will be no IPD sharing
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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