Asciminib and Inotuzumab Ozogamicin for Relapsed or Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia (R/R Ph+ B-ALL)
A Phase I Open-label, Multicenter Trial of Asciminib (ABL001) Added to Inotuzumab Ozogamicin (Besponsa®) in Patients With Relapsed or Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia
This is a multi-center phase 1 open-label study to determine the safety of ABL001 and INO in patients with relapsed or refractory Philadelphia chromosome-positive b-cell acute lymphoblastic leukemia.
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
상세 설명
This study will test a combination of two drugs for people with Philadelphia chromosome-positive B-cell acute lymphoblastic leukemia (Ph-positive B-ALL), a fast-growing blood cancer. Asciminib is a targeted oral drug that is FDA-approved for chronic myeloid leukemia, but is not FDA-approved for Ph-positive B-ALL. Inotuzumab ozogamicin (also called Besponsa) is an FDA-approved treatment that uses an antibody (a protein) to deliver a small amount of chemotherapy directly to leukemia cells. It is commonly used when leukemia has come back after earlier treatment (relapsed) or has not improved with treatment (refractory). This study aims to find the safest dose of asciminib when it is given together with inotuzumab ozogamicin. The goal is to see whether this combination can provide a more effective and better tolerated treatment option for people whose leukemia has returned or has not responded to previous therapies.
연구 유형
연구 유형
등록 (추정된)
등록
단계
단계
- 1단계
연락처 및 위치
연구 연락처
연구 연락처
- 이름: Research Nurse Navigator
- 전화번호: 212-342-5162
- 이메일: cancerclinicaltrials@cumc.columbia.edu
연구 장소
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Florida
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Miami, Florida, 미국, 33136
- 아직 모집하지 않음
- University of Miami Sylvester Comprehensive Cancer Center
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연락하다:
- Terrence Bradley, MD
- 전화번호: (305) 243-5302
- 이메일: tbradley@med.miami.edu
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Illinois
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Chicago, Illinois, 미국, 60805
- 아직 모집하지 않음
- University of Chicago Comprehensive Cancer Center
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연락하다:
- Anand Ashwin Patel, MD
- 전화번호: (708) 229-6001
- 이메일: Anand.patel@bsd.uchicago.edu
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New York
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New York, New York, 미국, 10032
- 모병
- Columbia University Irving Medical Center
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연락하다:
- Research Nurse Navigator
- 전화번호: 646-317-5077
- 이메일: cancerclinicaltrials@cumc.columbia.edu
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Washington
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Seattle, Washington, 미국, 98109
- 아직 모집하지 않음
- University of Washington Fred Hutchinson Comprehensive Cancer Center
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연락하다:
- Ryan Cassaday, MD
- 전화번호: (855) 557-0555
- 이메일: rcassada@fredhutch.edu
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
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Documented and confirmed CD22+ R/R Ph+ B-ALL according to the World Health Organization (WHO) classification
- Relapsed ALL is defined as the detection of ≥ 5% blasts in the bone marrow with or without non-central nervous system (CNS) extramedullary disease
- Refractory ALL is defined as the failure to obtain a CRc after at least two courses of intensive induction or immunotherapy such as blinatumomab, or to CAR-T cells. Persistent MRD without ≥ 5% blasts in the bone marrow will not be sufficient for inclusion
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Disease must be positive for Ph (p190 or p210 transcript) by cytogenetics or PCR
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Disease must be positive for CD22, defined as any percentage of leukemic blasts expressing CD22
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Disease must be positive for t(9;22) by Cytogenetics and FISH. Subjects with complex translocations resulting in BCR::ABL1 fusion protein are eligible.
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BCR-ABL mutational panel performed within 90 days prior to start of treatment, including the following 4 mutations: T315I and M244V, A337V, or F359C/I/V mutations. Specific panel may be determined per institution, however must include all aforementioned mutations. Note: T315I mutated patients are only eligible once Dose Level 3 is open to enrollment
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Subject must have recovered (no worse than Grade 1 severity) from effects of all previous treatments.
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Able to provide informed consent
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Age ≥18 years of age
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Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 2 or Karnofsky ≥60%
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Adequate organ function defined by the following parameters:
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5x the upper limit of normal (ULN)
- Total bilirubin ≤1.5x the ULN unless due to known Gilbert's disease or hemolysis due to blood transfusion
- For patients with Gilbert's disease: total bilirubin ≤ 3x the ULN
- Calculated glomerular filtration rate (GFR) of ≥ 15 mL/min/1.73 m2
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Female patients of childbearing potential must not be nursing or planning to become pregnant
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Female patients of childbearing potential require a negative urine or serum pregnancy test within 30 days of study therapy.
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Female patients of childbearing potential must be willing to use at least 1 method of highly effective contraception during the study until 8 months after last dose of inotuzumab ozogamicin, or 1 week after the last dose of asciminib, whichever is later.
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Male patients who are sexually active must be willing to use condoms and refrain from sperm donation during the study until 5 months after last dose of inotuzumab ozogamicin
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Willing to provide pretreatment bone marrow aspirate and biopsies samples as well as subsequent bone marrow aspirate and biopsies samples during the study.
Exclusion Criteria:
- Presence of severe and/or uncontrollable concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, active or uncontrolled infection)
- All prior Philadelphia chromosome-directed TKI therapy must be discontinued at least five days prior to study entry; corticosteroids and hydroxyurea are permitted during this period
- Treatment with chemotherapy, radiotherapy, or biologic therapy within 3 weeks prior to treatment Note: Hydroxyurea or corticosteroids is permitted; Previous treatment with ≥1 BCR-ABL-directed TKI
- Prior allogeneic stem cell transplant < 90 days prior to anticipated treatment start
- Patients with active GVHD (graft versus host disease) and/or receiving immunosuppressive drugs for GVHD
- Patients receiving calcineurin inhibitors < 4 weeks prior to anticipated treatment start
- Ph-directed TKI within 5 days of treatment start
- Prior exposure to INO
- Prior exposure to asciminib
- The presence of mixed phenotype acute leukemia or CML in lymphoid blast phase
- Presence of BCR-ABL1 M244V, A337V, or F359C/I/V mutations other n-lobe known to confer clinical resistance to asciminib as detected by ABL1 kinase mutational panel
- Patients with T315I mutation as detected by ABL1 kinase mutational panel are excluded from asciminib dose levels lower than 200mg BID
- Clinical suspicion for active CNS involvement by ALL; previously treated CNS involvement is allowed
- Second malignancy requiring treatment within 6 months of study enrollment, with the exception of non-melanoma skin cancers or cancers requiring hormonal therapy only
- Active, uncontrolled infection
- Human immunodeficiency virus (HIV) not controlled by standard therapy
- Active hepatitis B or C infection. Participants whose infections are controlled with antiviral therapy are allowed.
- Patients with end stage renal disease requiring dialysis
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 순차적 할당
- 마스킹: 없음(오픈 라벨)
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
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실험적: Single-Arm
All participants enrolled to the treatment arm of the study will receive Asciminib in combination with Inotuzumab Ozogamicin. INO is to be dosed in a fractionated schedule as follows:
Asciminib will be administered at a dose of 40, 60, 80, 160, or 200 mg twice daily. |
Asciminib is a first-in-class STAMP (Specifically Targeting the ABL Myristoyl Pocket), and is an approved TKI in newly-diagnosed and previously-treated chronic myeloid leukemia (CML). It is a non-chemotherapeutic, oral agent which specifically and potently targets the mutant Philadelphia chromosome driven by t(9;22). Due to its novel mechanism of action as a STAMP, asciminib is active in the setting of mutations in the BCR-ABL ATP-binding pocket including the T315I gatekeeper mutations, which confers resistance to first- and second-generation tyrosine kinase inhibitors (TKIs). Asciminib has not yet been studied in Philadelphia-chromosome positive B-ALL.
다른 이름들:
Inotuzumab ozogamicin (INO) is an antibody-drug conjugate which targets CD22, an antigen present in >90% of B-ALL cases. INO is FDA approved and a standard-of-care treatment in relapsed/refractory B-ALL. The drug is approved and active in Ph-positive and -negative disease.
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Number of adverse events
기간: 36 Months
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The safety profile of asciminib added to inotuzumab ozogamicin (INO) will be evaluated by the rates of adverse events categorized by the NCI Common Terminology Criteria for Adverse Events [CTCAE] v5.0. |
36 Months
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Maximum Tolerated Dose (MTD) of asciminib
기간: 36 Months
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The MTD of asciminib will be determined using a dose escalation strategy. The MTD is defined as the highest dose that be administered without causing dose-limiting toxicities in 75% of participants. |
36 Months
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2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Composite Complete Response (CRc) rate
기간: 36 Months
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Composite complete response (CRc) is defined as the rate of complete response (CR) + complete response with incomplete hematologic recovery (CRi), with complete metabolic response by PET/CT by the end of consolidation, according to the European LeukemiaNet (ELN) 2024 ALL criteria. |
36 Months
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Duration of remission (DOR)
기간: 36 Months
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DOR is defined as the time from CRc until relapse or death from any cause, whichever is first |
36 Months
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Overall survival (OS)
기간: 36 Months
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OS is defined as the time from study entry until death from any cause |
36 Months
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Event-free survival (EFS)
기간: 36 Months
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EFS is defined as the time from randomization to an event which may include disease progression, discontinuation of the treatment for any reason, or death |
36 Months
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Rate of molecular response (MR)
기간: 36 Months
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The rate of molecular response (MR) is assessed by clearance of measurable disease <0.1% (MR3) and <0.01% (MR4) as measured by BCR::ABL1 PCR in the bone marrow with complete metabolic response by PET/CT by the end of consolidation. |
36 Months
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공동 작업자 및 조사자
수사관
수사관
- 수석 연구원: Sunil Iyer, MD, Columbia University
연구 기록 날짜
연구 주요 날짜
연구 시작 (추정된)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
- 병리학적 과정
- 신생물
- 질병 속성
- 면역계 질환
- 감염
- 바이러스 질환
- 조직학적 유형에 따른 신생물
- DNA 바이러스 감염
- 림프계 질환
- 림프 증식 장애
- 면역증식성 장애
- 림프종, 비호지킨
- 림프종, B세포
- 림프종
- 엡스타인-바 바이러스 감염
- 헤르페스바이러스과 감염
- 종양 바이러스 감염
- 병리학적 상태, 징후 및 증상
- 헴 및 림프병
- 회귀
- 버킷 림프종
- 아미노산, 펩티드 및 단백질
- 단백질
- 탄수화물
- 글리코 사이드
- 항체, 모노클로 날, 인간화
- 항체, 모노클로 날
- 항체
- 면역 글로불린
- 면역 단백질
- 혈액 단백질
- 혈청 글로불린
- 글로불린
- 아미노 글리코 시드
- Calicheamicins
- 이노투주맙 오조가미신
- 아스 시미 닙
기타 연구 ID 번호
기타 연구 ID 번호
- ACYY0048
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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