A Study of LP-118 In Combination With Ponatinib, Dexamethasone And Blinatumomab For Adults With Newly-Diagnosed, BCR::ABL1-Positive Acute Lymphoblastic Leukemia (ALL)
A Phase I Study of the Bcl-2/Bcl-XL Inhibitor LP-118 In Combination With Ponatinib, Dexamethasone And Blinatumomab For Adults With Newly-Diagnosed, Philadelphia-Chromosome/BCR::ABL1-Positive Acute Lymphoblastic Leukemia
調査の概要
詳細な説明
Philadelphia-Chromosome/BCR::ABL1-Positive (Ph+) acute lymphoblastic leukemia (ALL) is a type of blood cancer that happens when a specific genetic change occurs in the DNA of certain blood cells, leading to uncontrolled growth of cells. Ph+ ALL is a more aggressive form of leukemia compared to other types but with specific medications called tyrosine kinase inhibitors, treatment outcomes have significantly improved. Unfortunately, some patients still do experience relapsed disease, when their leukemia comes back after treatment, which is challenging to treat. Therefore, research is ongoing to determine ways to improve therapy and outcomes for patients with Ph+ ALL.
The purpose of this study is to learn more about LP-118 and its side effects and decide on acceptable doses when combined with Food and Drug Administration (FDA) approved therapy for adult patients with Ph+ ALL. All participants will receive standard of care treatment for newly diagnosed Ph+ ALL consisting of the FDA approved drugs ponatinib, dexamethasone, intrathecal (injection into the spinal canal, also called "spinal tap") and systemic methotrexate and blinatumomab. This study will investigate different dose levels of LP-118 when given with his treatment combination to determine the most effective doses that can be safely given to patients with Ph+ ALL. This means that enrolled patients will receive progressive increasing or decreasing doses of LP-118 until the highest effective and safe dose is determined, while closely monitoring patients for any side effects or reactions. The highest dose is the dose that is the most effective dose that can be safely given to patients. This study will also preliminarily investigate if the addition of LP-118 improves treatment responses and outcomes for patients. If this trial is successful, the effectiveness of LP-118 added to this treatment regimen to treat Ph+ ALL will be studied in the next phase of clinical trials.
研究の種類
入学 (推定)
段階
- フェーズ 1
連絡先と場所
研究連絡先
- 名前:Dianna Auman, RN
- 電話番号:336-716-1122
- メール:Dianna.Roesler@Advocatehealth.org
研究場所
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North Carolina
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Charlotte、North Carolina、アメリカ、28204
- Atrium Health Levine Cancer
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コンタクト:
- Katarzyna Wydrzynska-Gruner, RN
- 電話番号:704-355-2000
- メール:katarzyna.wydrzynskagruner@advocatehealth.org
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副調査官:
- Thomas Knight, MD
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Winston-Salem、North Carolina、アメリカ、27157
- Atrium Health Wake Forest Baptist Comprehensive Cancer Center
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主任研究者:
- Madelyn Burkart, MD
-
コンタクト:
- Dianna Auman, RN
- 電話番号:336-716-1122
- メール:Dianna.Roesler@Advocatehealth.org
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-
参加基準
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
説明
Inclusion Criteria:
- Ability to understand and willingness to sign an IRB-approved informed consent.
- Age ≥ 18 years at the time of consent.
- ECOG Performance Status (PS) ≤ 2.
- Histological or cytological confirmation of newly diagnosed CD19-positive Philadelphia-chromosome/BCR::ABL1-positive ALL.
- Creatinine clearance: ≥60 mL/min, determined by the Cockroft-Gault formula, or measured by a 24-hour urine collection.
- Bilirubin ≤ 1.5 × upper limit of normal (ULN) - Unless liver abnormalities considered due to Gilbert's syndrome or of non-hepatic origin i.e., leukemic involvement. For patients with Gilbert's syndrome, bilirubin ≤1.5 x of their baseline bilirubin level will be required.
- Aspartate aminotransferase (AST) - Unless liver abnormalities considered due to Gilbert's syndrome or of non-hepatic origin i.e., leukemic involvement. For patients with Gilbert's syndrome, bilirubin ≤1.5 x of their baseline bilirubin level will be required.
- Alanine aminotransferase (ALT) - Unless liver abnormalities considered due to Gilbert's syndrome or of non-hepatic origin i.e., leukemic involvement. For patients with Gilbert's syndrome, bilirubin ≤1.5 x of their baseline bilirubin level will be required.
- Individuals of childbearing potential (ICBP) must have a negative serum pregnancy test. NOTE: Individuals who may become pregnant are considered to have childbearing potential unless they are surgically infertile (have undergone a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or are postmenopausal (at least 12 consecutive months with no menses without an alternative medical cause).
- ICBP must be willing to use two forms of contraception, one of which must be a barrier method and the other must be a highly effective contraceptive method from the time of informed consent until 6 months after study treatment discontinuation. Male participants with female partners of reproductive potential will need to agree to use contraception methods described above during study treatment and for at least 6 months after completion of all study treatment.
- Male participants must agree to refrain from sperm donation during study treatment and for at least 6 months after completion of all study treatment.
- Ability to ingest oral medications without a malabsorption condition, known dysphagia, short-gut syndrome, gastroparesis, or other conditions that may limit the ingestion or gastrointestinal absorption, distribution, metabolism and excretion of drugs administered orally, per the enrolling investigator.
Exclusion Criteria:
- Any prior treatment for ALL except for a single dose of intrathecal (IT) chemotherapy, corticosteroids, hydroxyurea, a single dose of vincristine, cytarabine, leukapheresis, and/or a BCR::ABL1-targeted tyrosine kinase inhibitor. Permitted prior treatment is limited to a duration of no longer than 14 days. Permitted prior treatment must be stopped at least 24 hours prior to starting study therapy.
- Women who are pregnant, nursing, or who plan to become pregnant while in the study and for at least 6 months after the last administration of all study treatment. NOTE: breast milk cannot be stored for future use while the mother is being treated on study. Pregnant participants are excluded from this study because ponatinib, blinatumomab, and methotrexate have the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with ponatinib, blinatumomab, and methotrexate breastfeeding should be discontinued if the patient is treated with ponatinib, blinatumomab, and methotrexate.
- Active second malignancy except for localized prostate cancer, basal cell or squamous cell carcinoma of the skin and carcinoma in situ of the skin or cervix.
- Unstable or severe uncontrolled medical condition in the opinion of the enrolling investigator (e.g., unstable cardiac function or unstable pulmonary condition; uncontrolled infection).
- Participants with known history of hepatitis B virus, hepatitis C virus, or human immunodeficiency virus (HIV) are eligible if no evidence of active viral replication by blood testing (i.e. negative viral loads). HIV positive participants must be on active anti-retroviral therapy and willing to continue therapy during study treatment.
- Uncontrolled cardiac disease as determined by the enrolling investigator.
- Major surgery, as determined by the enrolling investigator, within 2 weeks before enrollment.
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:順次割り当て
- 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 |
|---|---|
|
実験的:Combination of LP-118, ponatinib, dexamethasone, blinatumomab and methotrexate
LP-118 will be given as tablets of 10mg or 100mg during the induction II course only in combination with ponatinib, dexamethasone, methotrexate and blinatumomab. .
Ponatinib, dexamethasone and methotrexate dosing will remain fixed, whereas LP-118 dosing will be dependent on the dose level to which a participant is assigned.
|
s LP-118 dosing will be dependent on the dose level to which a participant is assigned:
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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 |
|---|---|---|
|
Estimation of the Maximum Tolerated Dose (MTD) for LP-118 (Dose Escalation)
時間枠:The DLT monitoring period is from Day 1 of Course 2 until the end of Course 2 (approximately 21 days).
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Binary variable indicating if a Dose Limiting Toxicity (DLT) occurred.
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The DLT monitoring period is from Day 1 of Course 2 until the end of Course 2 (approximately 21 days).
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Identification of the Recommended Phase 2 Dose (RP2D) LP-118 (Dose Expansion)
時間枠:The DLT monitoring period is from Day 1 of Course 2 until the end of Course 2 (approximately 21 days).
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Binary variable indicating if a Dose Limiting Toxicity (DLT) occurred.
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The DLT monitoring period is from Day 1 of Course 2 until the end of Course 2 (approximately 21 days).
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Overall Survival (OS).
時間枠:At most 10 years.
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The time from treatment administration to death from any cause, or until last contact if the patient has not died
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At most 10 years.
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Event-Free Survival (EFS).
時間枠:At most 10 years.
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Time from start of therapy until failure to achieve CR/CRi, relapse, or death from any cause or otherwise censored at the date of the last disease assessment.
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At most 10 years.
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Overall Complete Molecular Response (CMR) rate.
時間枠:At most 10 years.
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Binary variable indicating if a CMR at any time while on study.
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At most 10 years.
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Measurable Residual Disease (MRD) rate.
時間枠:Approximately 3 years.
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Binary variable indicating if MRD was achieved.
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Approximately 3 years.
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Complete clinical remission with incomplete count recovery (CRi)
時間枠:Approximately 3 years.
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Complete clinical remission with incomplete count recovery (CRi) defined as complete clinical remission except with ANC< 1000/μL and/or platelets <100,000/μL
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Approximately 3 years.
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Overall Response (OR) rate.
時間枠:Approximately 3 years.
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A binary variable indicating if a CR or CRi was achieved.
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Approximately 3 years.
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Relapsed-Free Survival (RFS).
時間枠:At most 10 years.
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Time from Complete Remission (CR) or Complete Remission with Incomplete Hematologic Recovery (CRi) to disease relapse or death (whichever occurs first) or otherwise censored at the date of the last disease assessment.
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At most 10 years.
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Isolated Central Nervous System (CNS) relapse rate.
時間枠:At most 10 years.
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Binary variable indicating if isolated CNS relapse occurred.
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At most 10 years.
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Complete Response (CR) rate.
時間枠:Approximately 3 years.
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Complete clinical remission defined as the disappearance of leukemia as indicated by <5% marrow blasts and the absence of peripheral blasts or extramedullary disease, with recovery of hematopoiesis defined by Absolute Neutrophil Count (ANC) ≥1000/μL and platelets ≥100,000/μL.
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Approximately 3 years.
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Occurrence of toxicities per Common Terminology Criteria V5.0
時間枠:Approximately 3 years.
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Binary variables indicating the occurrence of toxicities per Common Terminology Criteria V5.0.
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Approximately 3 years.
|
協力者と研究者
捜査官
- 主任研究者:Madelyn Burkart, MD、Wake Forest University Health Sciences
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
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
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- IRB00127277
- P30CA012197 (米国 NIH グラント/契約)
- ONC-LEUK-2405 (その他の識別子:Atrium Health Wake Forest Baptist Comprehensive Cancer Center)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
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
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