Asciminib-Based Induction Therapy for the Treatment of Newly Diagnosed Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
A Phase 1 Study of Asciminib-Based Induction Therapy in Newly Diagnosed Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL)
This phase I trial studies the side effects and best dose of asciminib when given together with intrathecal (IT) chemotherapy, dexamethasone, vincristine, and blinatumomab (asciminib-based induction therapy) in treating patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). Asciminib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as methotrexate and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Dexamethasone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Vincristine is in a class of medications called vinca alkaloids. It works by stopping cancer cells from growing and dividing and may kill them. Blinatumomab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Giving asciminib-based induction therapy may be safe, tolerable, and/or effective in treating patients with newly diagnosed Ph+ ALL.
調査の概要
状態
状態
条件
条件
介入・治療
介入・治療
詳細な説明
PRIMARY OBJECTIVES:
I. Evaluate the safety of asciminib as the tyrosine kinase inhibitor (TKI) backbone in a chemotherapy minimizing induction strategy (dexamethasone, vincristine, IT chemotherapy, blinatumomab).
II. Determine the recommended phase 2 dose (RP2D) of asciminib induction therapy as the TKI backbone in a reduced intensity strategy for the treatment of Ph+ ALL.
SECONDARY OBJECTIVES:
I. To evaluate the safety and tolerability of an asciminib-based therapy for post-dose limiting toxicity (DLT) cycles.
II. To evaluate the complete molecular response (CMR) rate after 1 and 3 cycles.
III. To evaluate the complete response (CR)/CR with incomplete hematologic recovery (CRi) rate after 1 and 3 cycles.
IV. To evaluate 1-year overall survival and event-free survival rates. V. To evaluate the rate of transplant in patients achieving CR in cycle 1 (CR1).
VI. To evaluate the pharmacokinetics of asciminib in this combination. VII. To evaluate minimal residual disease negativity (MRD-) by clonoSEQ assay (clonoSEQ) after first and third cycles of induction.
EXPLORATORY OBJECTIVE:
I. Evaluate novel molecular minimal residual disease (MRD) status in plasma and cerebrospinal fluid (CSF).
OUTLINE: This is a dose-escalation study of asciminib in combination with triple IT chemotherapy, dexamethasone, vincristine, and blinatumomab.
Patients receive asciminib orally (PO) once daily (QD) or twice daily (BID) on days 1-28 of cycles 1 and 2 and days 1-42 of cycle 3, triple IT chemotherapy (methotrexate, cytarabine, corticosteroid) on days 1 and 14 of cycles 1 and 2 and days 1, 14, 28, and 42 of cycle 3, dexamethasone PO on days 1-4 and 11-14 of cycle 1, vincristine intravenously (IV) on days 1 and 14 of cycle 1, and blinatumomab IV on days 1-28 of cycle 2 and days 15-42 of cycle 3. Cycles repeat every 28 days (cycles 1 and 2) or 42 days (cycle 3) for up to 3 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo echocardiography (ECHO) during screening, blood and CSF sample collection on study, and bone marrow aspiration throughout the study.
After completion of study treatment, patients are followed up at 30 days and then every 3 months for up to 1 year post-treatment start.
研究の種類
研究の種類
入学 (推定)
入学
段階
段階
- フェーズ 1
連絡先と場所
研究場所
-
-
California
-
Duarte、California、アメリカ、91010
- City of Hope Medical Center
-
主任研究者:
- Paul Koller
-
コンタクト:
- Paul Koller
- 電話番号:626-218-2405
- メール:pkoller@coh.org
-
-
参加基準
適格基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
Inclusion Criteria:
-
Documented informed consent of the participant and/or legally authorized representative
-
Newly diagnosed Ph+ or BCR-ABL1-positive ALL, as defined by the 2025 National Comprehensive Cancer Network (NCCN) guidelines
-
Age: ≥ 18 years
-
Eastern Cooperative Oncology Group (ECOG) ≤ 2
-
Total bilirubin ≤ 1.5 X upper limit of normal (ULN) (unless has Gilbert's disease)
-
Aspartate aminotransferase (AST) ≤ 3.0 x ULN
-
Alanine aminotransferase (ALT) ≤ 3.0 x ULN
-
Serum creatinine < 1.5 mg/dL
-
Left ventricular ejection fraction (LVEF) ≥ 50%
- Note: To be performed within 28 days prior to day 1 of protocol therapy
-
Normal QT interval corrected per Fridericia method (QTcF) on screening electrocardiogram, defined as QTcF of ≤ 480 msec
-
Oxygen (O2) saturation > 90% on room air
-
Seronegative for HIV antigen (Ag)/antibody (Ab) combo, hepatitis C virus (HCV), active hepatitis B virus (HBV) (surface antigen negative)
- If seropositive for HIV, HCV or HBV, nucleic acid quantitation must be performed. Viral load must be undetectable. HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
-
Meets other institutional and federal requirements for infectious disease titer requirements
- Note Infectious disease testing to be performed within 28 days prior to day 1 of protocol therapy
-
Women of childbearing potential (WOCBP): negative urine or serum pregnancy test
- If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
-
Agreement by females and males of childbearing potential to use a highly effective method of birth control or abstain from heterosexual activity for the course of the study through at least 120 days after the last dose of protocol therapy
- Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for > 1 year (women only)
Exclusion Criteria:
-
Prior treatment failure with asciminib
-
Prior/current treatment with any systemic anticancer therapy (including but not limited to any TKI) and/or radiotherapy for ALL, with the exception of an optional prephase therapy or chemotherapy induction (no more than one cycle), which should be discussed with the principal investigator (PI). Patients who have received no more than two weeks of chemotherapy (not including steroids) with or without other TKIs are considered minimally pretreated and still eligible
-
Currently taking drugs that are known to have a risk of causing prolonged corrected QT interval (QTc) or torsades depointes (TdP) (unless these can be changed to acceptable alternatives or discontinued)
-
Taking any medications or herbal supplements that are known to be strong inhibitors or strong inducers of cytochrome P450 (CYP)3A4 within at least 14 days before the first dose of study drug
-
Cardiac or cardiac repolarization abnormality, including any of the following:
- History within 6 months prior to starting study treatment of myocardial infarction (MI), or coronary artery bypass graft (CABG)
- Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), complete left bundle branch block, high-grade atrioventricular (AV) block (e.g., bifascicular block, Mobitz type II and third-degree AV block)
-
A history of cerebrovascular events such as stroke or transient ischemic attack
-
Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome. Treatment with any investigational products within 30 days before enrollment or 6 half-lives of the agent, whichever is shorter
-
History of pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis
-
Uncontrolled hypertriglyceridemia (triglycerides > 450 mg/dL)
-
Clinically significant uncontrolled illness
-
Active infection not responding to treatment at the time of starting therapy
-
Autoimmune disease with potential central nervous system (CNS) involvement
-
Other active malignancy. Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
-
Females only: Pregnant or breastfeeding
-
Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures
-
Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)
研究計画
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:なし
- 介入モデル:単一グループの割り当て
- マスキング:なし(オープンラベル)
アーム数
武器と介入
参加者グループ / アーム参加者グループ / アーム |
介入・治療介入・治療 |
|---|---|
|
実験的:Treatment (asciminib-based induction therapy)
Patients receive asciminib PO QD or BID on days 1-28 of cycles 1 and 2 and days 1-42 of cycle 3, triple IT chemotherapy on days 1 and 14 of cycles 1 and 2 and days 1, 14, 28, and 42 of cycle 3, dexamethasone PO on days 1-4 and 11-14 of cycle 1, vincristine IV on days 1 and 14 of cycle 1, and blinatumomab IV on days 1-28 of cycle 2 and days 15-42 of cycle 3. Cycles repeat every 28 days (cycles 1 and 2) or 42 days (cycle 3) for up to 3 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo ECHO during screening, blood and CSF sample collection on study, and bone marrow aspiration throughout the study. |
Given IV
他の名前:
Undergo ECHO
他の名前:
Given PO
他の名前:
Given IV
他の名前:
Undergo bone marrow aspiration Given IT
他の名前:
Undergo blood and CSF sample collection
他の名前:
Given PO
他の名前:
Given IT Given IT
他の名前:
|
この研究は何を測定していますか?
主要な結果の測定
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 toxicity (DLT)
時間枠:Up to 2 cycles (Cycle length = 28 days)
|
Toxicity will be graded according to the National Cancer Institute-Common Terminology Criteria for Adverse Events (CTCAE), version (v) 6.0. |
Up to 2 cycles (Cycle length = 28 days)
|
|
Maximum tolerated dose/recommended phase 2 dose
時間枠:Up to the 18.6 months
|
Will be established using a phase 1 queue 3+3 design. |
Up to the 18.6 months
|
二次結果の測定
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Incidence of adverse events (AEs)
時間枠:Up to 30 days after last dose of study drug
|
The number and percent of patients with AEs will be tabulated by system organ class and preferred term using the Medical Dictionary for Regulatory Activities preferred term dictionary. Observed toxicities will be summarized for the DLT period (cycles 1 and 2), as well as for each of the 3 cycles, in terms of type (organ affected or laboratory determination), severity, time of onset, duration, frequency, probable association with each study treatment and reversibility or outcome. Listings and narratives will be provided for deaths on study, DLTs/unacceptable toxicities, serious adverse events, and events leading to discontinuation of treatment. Descriptive summaries will also be generated for AE data assessed and graded according to the CTCAE v6.0 grading system. |
Up to 30 days after last dose of study drug
|
|
Complete molecular response
時間枠:At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
Will be assessed in accordance with the 2025 National Comprehensive Cancer Network (NCCN) guidelines at each dose level and disease assessment timepoint (cycles 1 and 3). Rates and 95% Clopper-Pearson binomial confidence interval (CI) will be calculated. |
At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
|
Complete response (CR)/CR with incomplete hematologic recovery
時間枠:At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
Will be assessed in accordance with the 2025 NCCN guidelines at each dose level and disease assessment timepoint (cycles 1 and 3). Rates and 95% Clopper-Pearson binomial CI will be calculated. |
At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
|
Minimal residual disease negativity
時間枠:At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
Will be assessed in accordance with the 2025 NCCN guidelines at each dose level and disease assessment timepoint (cycles 1 and 3). Rates and 95% Clopper-Pearson binomial CI will be calculated. |
At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
|
Rate of allogeneic hematopoietic cell transplantation
時間枠:Up to 1 cycle (Cycle length = 28 days)
|
Rates and 95% Clopper-Pearson binomial CI will be calculated. |
Up to 1 cycle (Cycle length = 28 days)
|
|
Overall survival (OS)
時間枠:From first study dose to death from any cause or last contact date, whichever comes first, assessed up to 1 year
|
Median OS and 1-year OS rate, will be estimated using the product-limit method of Kaplan-Meier product-limit method and the 95% C.I. of the survival curve will be calculated by the Brookmeyer-Crowley method with log-log transformation. |
From first study dose to death from any cause or last contact date, whichever comes first, assessed up to 1 year
|
|
Progression-free survival (PFS)
時間枠:Up to 1 year
|
Median PFS and 1-year PFS rate, will be estimated using the product-limit method of Kaplan-Meier product-limit method and the 95% C.I. of the survival curve will be calculated by the Brookmeyer-Crowley method with log-log transformation. |
Up to 1 year
|
|
Concentration of asciminib (Pharmacokinetics)
時間枠:Up to 98 days (Cycle 1 and 2 are 28 days each, Cycle 3 is 42 days)
|
Will be assessed over time by descriptive and graphical statistics. |
Up to 98 days (Cycle 1 and 2 are 28 days each, Cycle 3 is 42 days)
|
協力者と研究者
協力者
協力者
捜査官
捜査官
- 主任研究者:Paul Koller、City of Hope Medical Center
研究記録日
主要日程の研究
研究開始 (推定)
研究開始
一次修了 (推定)
一次修了
研究の完了 (推定)
研究の完了
試験登録日
最初に提出
最初に提出
QC基準を満たした最初の提出物
QC基準を満たした最初の提出物
最初の投稿 (実際)
最初の投稿
学習記録の更新
投稿された最後の更新 (実際)
投稿された最後の更新
QC基準を満たした最後の更新が送信されました
QC基準を満たした最後の更新が送信されました
最終確認日
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
- ホルモン
- ホルモン、ホルモン代替品、ホルモン拮抗薬
- 硫黄化合物
- 有機化学物質
- 複素環化化合物、1リング
- 複素環化化合物
- 複素環化化合物、2リング
- 複素環化化合物、融合リング
- 調査手法
- 臨床検査技術
- 診断技術と手順
- 診断
- 核酸、ヌクレオチド、およびヌクレオシド
- 炭化水素
- 炭化水素、周期的
- アルカロイド
- 炭化水素、芳香
- 多環式化合物
- インドール
- シチジン
- ピリミジンヌクレオシド
- ピリミジン
- 妊娠
- 妊娠
- ステロイド
- 融合リング化合物
- ステロイド、フッ素化
- ベンゼン誘導体
- スルホン酸
- 硫酸
- ヌクレオシド
- 羽毛
- プテリジン
- 妊娠症
- ヴィンカアルカロイド
- セコロガニントリプタミンアルカロイド
- インドールアルカロイド
- インドリジジン
- インドリジン
- アラビノヌクレオシド
- アミノプテリン
- ベンゼンスルホン酸塩
- アリルスルホン酸塩
- アリールスルホン酸
- デキサメタゾン
- メトトレキサート
- シタラビン
- ビンクリスチン
- ドベシル酸カルシウム
- 標本処理
- Blinatumomab
- デキサメタゾン21-リン酸
- N、n-dicyclohexyl-isoborneol-10-スルホンアミド
- メルフォス
- Auricularum
- デキサメタゾン酢酸
- 副腎皮質ホルモン
- アシミニブ
その他の研究ID番号
その他の研究ID番号
- 25618
- P30CA033572 (米国 NIH グラント/契約)
- NCI-2026-07296 (レジストリ識別子:CTRP (Clinical Trial Reporting Program))
- 25618 (その他の識別子:City of Hope Medical Center)
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
米国FDA規制機器製品の研究
米国で製造され、米国から輸出された製品。
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。