Preoperative Durvalumab, Cisplatin, Gemcitabine With or Without Futibatinib Targeted Therapy for the Treatment of Resectable Intrahepatic Cholangiocarcinoma, OPTIC Trial
Phase II, Open Label, Non-Randomized, Study of Optimal Preoperative Therapy for Intrahepatic Cholangiocarcinoma (OPTIC)
調査の概要
状態
条件
詳細な説明
PRIMARY OBJECTIVE:
I. To assess the feasibility and safety of a novel treatment strategy that includes conducting next generation sequencing (NGS) on a preoperative tissue biopsy to administer prior to surgical resection.
SECONDARY OBJECTIVES:
I. To assess the radiological response rate (RRR) according to Response Evaluation Criteria in Solid Tumors 1.1 (RECIST 1.1) and advanced image analysis (radiomics) in patients with resectable intrahepatic cholangiocarcinoma (IHCC).
II. To assess the degree of pathologic response in the surgical specimen of patients with resectable intrahepatic cholangiocarcinoma (IHCC).
III. To assess response by measuring circulating tumor DNA (CT-DNA) dynamics in the blood samples of patients with resectable intrahepatic cholangiocarcinoma (IHCC).
IV. To determine the R0 resection rate post-surgery in patients with resectable intrahepatic cholangiocarcinoma (IHCC).
V. To determine progression-free survival (PFS) in patients with resectable intrahepatic cholangiocarcinoma (IHCC).
VI. To determine recurrence-free survival (RFS) in patients with resectable intrahepatic cholangiocarcinoma (IHCC).
VII. To identify patients' overall survival (OS) rate in patients with resectable intrahepatic cholangiocarcinoma (IHCC).
EXPLORATORY OBJECTIVE:
I. To assess the circulating and tumor immune microenvironment in tissue and blood samples.
OUTLINE:
Patients receive durvalumab intravenously (IV) over 60 minutes on day 1 and cisplatin IV over 60 minutes and gemcitabine IV over 30 minutes on days 1 and 8 of one 21-day cycle in the absence of disease progression or unacceptable toxicity while awaiting for NGS results.
After obtaining NGS molecular results, patients with FGFR2 fusion, rearrangement, or activating mutation are assigned to Arm A, while patients without FGFR2 fusion, rearrangement, or activating mutation are assigned to Arm B.
ARM A: Patients receive futibatinib orally (PO) once daily (QD) on days 1-28 of each cycle. Cycles repeat every 28 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo magnetic resonance imaging (MRI), computed tomography (CT) and blood sample collection throughout the trial.
ARM B: Patients continue receiving durvalumab IV over 60 minutes on day 1 and cisplatin IV over 60 minutes and gemcitabine IV over 30 minutes on days 1 and 8 of each cycle. Cycles repeat every 21 days for up to 3 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo MRI, CT and blood sample collection throughout the trial.
After completion of study treatment, patients are followed up every 3 months for 3 years.
研究の種類
入学 (推定)
段階
- フェーズ2
連絡先と場所
研究連絡先
- 名前:Study Coordinator
- 電話番号:3126951301
- メール:cancertrials@northwestern.edu
研究場所
-
-
Illinois
-
Chicago、Illinois、アメリカ、60611
- Northwestern University
-
主任研究者:
- Shishir K. Maithel, MD
-
コンタクト:
- Shishir K. Maithel, MD
- 電話番号:404-617-7936
- メール:Shishir.maithel@nm.org
-
-
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:
Patients must have a confirmed biopsy proven diagnosis of resectable intrahepatic cholangiocarcinoma (IHCC) confined to the liver, bile duct, and /or regional lymph nodes confirmed by high-quality cross-sectional imaging by CT or MRI of the chest and MRI of the abdomen, and pelvis performed within 42 days (6 weeks) prior to enrollment. (MRI protocol: With and without gadolineum with T1 and T2 weighted sequences)
- Note: Distant extrahepatic disease is not allowed
- Note: When using CTs in addition to MRI, a high resolution triple phase thin-cut, CT with intravenous +/- oral contrast is the preferred imaging technique for assessing radiographic tumor response
- Patients must have measurable disease per RECIST 1.1
- Patients must be treatment naïve
- Patients must be age ≥ 18 years
- Patient with CORE biopsy for diagnosis that is sufficient enough to do NGS
- Patients must exhibit an Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
- Absolute neutrophil count (ANC) ≥ 1,000/mcL
- Hemoglobin (Hgb) ≥ 8 g/dL (blood transfusion allowed up to 7 days prior to starting on study drug)
Serum total bilirubin ≤ 2 X Institutional upper limit of normal (ULN)
- For Gilbert's disease: these value does not apply and treatment will be done per treating physician
Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT]) ≤ 5 x institutional ULN
- For Gilbert's disease: these value does not apply and treatment will be done per treating physician
Alanine aminotransferase (ALT) (serum glutamic-pyruvic transaminase [SGPT]) ≤ 5 x institutional ULN
- For Gilbert's disease: these value does not apply and treatment will be done per treating physician
- Creatinine ≤ 1.5 X Institutional ULN
- Calcium-phosphorus product < 55 mg^2 /dL2 (5.5mg/dL)
- Total corrected serum calcium within local normal limits
- Inorganic phosphorus within local normal limits
- For patients with a known history of human immunodeficiency virus (HIV), infected patients on effective anti-retroviral therapy must have a viral load undetectable for 6 months prior to registration. Please note this lab is not a requirement for eligibility, however, if it has been completed previously as part of the patient's health care, it should be documented for eligibility
- For patients with a known history of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
- Patients with a known history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with a known HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load. Please note this lab is not a requirement for eligibility, however if it has been completed previously as part of the patient's health care, it should be documented for eligibility
- The futibatinib and other therapeutic agents used in this trial are known to be teratogenic. For this reason, patients of child-bearing potential (POCBP) and their partners with sperm-producing reproductive capacity must agree to use adequate contraception from time of informed consent, for the duration of study participation, and for 11 and 14 months for POCBP and men respectively, following completion of study drug therapy. Should a POCBP become pregnant or suspect they are pregnant while they or their partner are participating in this study, they should inform their treating physician immediately. Patients with sperm-producing reproductive capacity (PWSPRC) treated or enrolled on this protocol must also agree to use adequate contraception with partners of childbearing potential from 28 days prior to starting gemcitabine/cisplatin/durvalumab (including dose interruptions) for the duration of study participation, and 11 and 14 months for POCBP and men respectively, months after completion of study drug administration.
Male subjects must practice true abstinence or agree to use a condom during sexual contact with a female of childbearing potential or a pregnant female while on treatment (including during dose interruptions) with gemcitabine/cisplatin/durvalumab and for 14 months following, even if he has undergone a successful vasectomy.
Note: A POCBP is any patient (regardless of gender, sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) with an egg-producing reproductive tract who meets the following criteria:
- Has not undergone a hysterectomy or bilateral oophorectomy
Has had menses at any time in the preceding 12 consecutive months (and therefore has not been naturally postmenopausal for > 12 months)
- POCBP must have a negative pregnancy test prior to registration on study
- Patients must have the ability to understand and the willingness to sign a written informed consent document and comply with the study requirements
- Patients must have the ability to swallow, retain and absorb oral medications
Exclusion Criteria:
- Patients with peripheral neuropathy of grade 2 or greater by Common Terminology Criteria for Adverse Events (CTCAE) 5.0. In CTCAE version 5.0 grade 2 sensory neuropathy is defined as "moderate symptoms; limiting instrumental activities of daily living (ADLs)
- Patients who are receiving any other investigational agents
Patients who have current evidence of corneal or retinal disorder/keratopathy including, but not limited to, bullous/band keratopathy, inflammation or ulceration, keratoconjunctivitis, confirmed by ophthalmic examination
- Note: Patients with asymptomatic ophthalmic conditions assessed by the investigator to pose minimal risk for study participation may be enrolled in the study
- Patients who have used medications known to prolong the QT interval and/or are associated with a risk of Torsades de Pointes (TdP) 7 days prior to first dose of study drug
- Patients who have clinically significant cardiac disease including any of the following: Congestive heart failure requiring treatment (New York Heart Association grade ≥ 2), or uncontrolled hypertension (refer to the European Society of Cardiology and European Society of Hypertension guidelines (Williams et al 2018)
Patients with corrected QT interval using Fridericia formula (QTcF) > 470 msec (males and females)
- Note: If the QTcF is > 470 msec in the first electrocardiogram (ECG), a total of 3 ECGs separated by at least 5 minutes should be performed. If the average of these 3 consecutive results for QTcF is ≤ 470 msec, the patient meets eligibility in this regard
- Patient with known history of congenital long QT syndrome
- Patients who have current evidence of concerning endocrine alterations of calcium/phosphate homeostasis, eg, parathyroid disorders, history of parathyroidectomy, tumor lysis, tumoral calcinosis etc., in the opinion of the investigator OR and taking medications which increase serum phosphorus and/or calcium concentration
Patients who have abnormal calcium or phosphorus, or calcium-phosphorus product ≥ 55 mg^2 /dL2:
- Inorganic phosphorus above local normal limits
- Total corrected serum calcium above local normal limits
Patients who are currently receiving or are planning to receive during participation in this study, treatment with agents that are known strong inducers or inhibitors of CYP3A4
- Note: Patients are not permitted to receive enzyme-inducing anti-epileptic drugs
Patients with known central nervous system (CNS) disease, except for treated brain metastasis. Patients with CNS metastases treated by neurosurgical resection or brain biopsy performed within 3 months prior to day 1 will be excluded.
- Note: Treated brain metastases are defined as having no evidence of progression or hemorrhage after treatment and no ongoing requirement for dexamethasone, as ascertained by clinical examination and brain imaging (MRI or CT) during the screening period. Anticonvulsants (stable dose) are allowed. Treatment for brain metastases may include whole brain radiotherapy (WBRT), radiosurgery (radiosurgery [RS]; gamma knife, linear accelerator [LINAC], or equivalent) or a combination as deemed appropriate by the treating physician
Patients who have the following would be excluded (as they would be contraindicated from receiving the trial drugs)
- Patients with history of ototoxicity or hearing issues (contraindicated for cisplatin administration)
Patients with the following should not receive immunotherapy
- History or active autoimmune disease
- Previous immunotherapy use
- Require immunosuppressive medications greater than 10mg of prednisone
- History of pneumonitis or interstitial lung disease
- History of grade ≥ 2 myocarditis
- Prior anaphylactic reaction to immune checkpoint inhibitors (ICI)
- Greater than grade 2 neuropathy
- Previous fibroblast growth factor receptor (FGFR) drug use
- Patients who have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to any of the study drugs
Patients who have an concurrent severe and/or uncontrolled medical conditions which could compromise participation in the study, including, but not limited to any of the following:
- Uncontrolled diabetes
- Ongoing or active infection requiring systemic treatment
- Symptomatic congestive heart failure
- Unstable angina pectoris
- Unstable symptomatic arrhythmia
- Myocardial infarction within 6 months of enrollment into the study
- Serious active, uncontrolled infection after inadequate biliary drainage if tumor obstructing bile duct
- Psychiatric illness/social situations that would limit compliance with study requirements
- Any other illness or condition that the treating investigator feels would interfere with study compliance or would compromise the patient's safety or study endpoints
- Patients with active other primary malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessment of the investigational regimen (at treating physician's discretion)
- Patients who are pregnant or nursing
- Patients with active alcohol use or illicit drug use that would, in the opinion of the principal investigator (PI) or a sub investigator (sub-I), prevent the subject from complying with the study protocol and/or endanger the subject during their participation in the study
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: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 |
|---|---|
|
実験的:Arm A (durvalumab, cisplatin, gemcitabine, futibatinib)
Patients receive durvalumab IV over 60 minutes on day 1 and cisplatin IV over 60 minutes and gemcitabine IV over 30 minutes on days 1 and 8 of one 21-day cycle in the absence of disease progression or unacceptable toxicity while awaiting for NGS results.
Patients receive futibatinib PO QD on days 1-28 of each cycle.
Cycles repeat every 28 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity.
Patients undergo MRI, CT and blood sample collection throughout the trial.
|
MRIを受ける
他の名前:
与えられた IV
他の名前:
与えられた IV
他の名前:
CTを受ける
他の名前:
与えられた IV
他の名前:
与えられたPO
他の名前:
Undergo blood and previously collected tissue sample collection
他の名前:
|
|
アクティブコンパレータ:Arm B (durvalumab, cisplatin, gemcitabine)
Patients receive durvalumab IV over 60 minutes on day 1 and cisplatin IV over 60 minutes and gemcitabine IV over 30 minutes on days 1 and 8 of one 21-day cycle in the absence of disease progression or unacceptable toxicity while awaiting for NGS results.
Patients continue receiving durvalumab IV over 60 minutes on day 1 and cisplatin IV over 60 minutes and gemcitabine IV over 30 minutes on days 1 and 8 of each cycle.
Cycles repeat every 21 days for up to 3 cycles in the absence of disease progression or unacceptable toxicity.
Patients undergo MRI, CT and blood sample collection throughout the trial.
|
MRIを受ける
他の名前:
与えられた IV
他の名前:
与えられた IV
他の名前:
CTを受ける
他の名前:
与えられた IV
他の名前:
Undergo blood and previously collected tissue sample collection
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
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Completion of all preoperative testing and therapy
時間枠:Up to 3 years
|
Treatment completion is defined as completion of next generation sequencing testing and all preoperative and operative therapy.
Will record the completion of all therapy rate, along with the exact 95% binomial confidence interval.
|
Up to 3 years
|
|
Incidence of Treatment-Emergent Adverse Events during all preoperative testing and therapy
時間枠:Up to 3 years
|
Unacceptable toxicity is defined as any grade 3 or higher toxicities by National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 that result in a treatment delay of > 28 days.
|
Up to 3 years
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Radiological response rate
時間枠:Up to 3 years
|
Will be assessed by according to Response Evaluation Criteria in Solid Tumors 1.1 (RECIST 1.1) and defined as the percentage of patients who will have complete response, partial response or stable disease after the neoadjuvant therapy.
|
Up to 3 years
|
|
Pathologic response rate (PRR)
時間枠:At the time of surgery
|
Will be defined as the degree of treatment effect to preoperative therapy.
Pathologic complete response (pCR) will be defined as no viable tumor cells (0% viable tumor, 100% necrosis or fibrosis).
Absent response will be defined as 100% viable tumor cells with no evidence of tumor regression.
Partial response will be defined as degree of necrosis or fibrosis in the tumor bed, rounded to the nearest 10% interval.
This is an endpoint to assess the pathologic response to futibatinib in those with FGFR2 fusions / rearrangements / activating mutations compared to cytotoxic chemotherapy in those without FGFR2 fusions/alterations.
Pathological response will be measured by a three-tier response rate using pathologic (p) complete response = 0% tumor cells, no response = no histologic evidence of tumor regression and partial response = % necrosis and evident tumor regression.
PRR will be calculated by Northwestern's pathologists with subspecialty training in gastrointestinal pathology.
|
At the time of surgery
|
|
Response by measuring circulating tumor deoxyribonucleic acid in the blood samples of patients with resectable intrahepatic cholangiocarcinoma
時間枠:Up to 3 years
|
Up to 3 years
|
|
|
R0 resection rate
時間枠:Up to 7 days after last dose of study drug
|
Up to 7 days after last dose of study drug
|
|
|
Progression-free survival (PFS)
時間枠:From the time of enrollment to either the day of first documented disease progression or death from any cause, assessed up to 3 years
|
Disease progression is defined as progressive disease per RECIST 1.1, other documented clinical or radiographical progression per physician judgement, or death due to disease.
If disease progression or death from any cause is not observed prior to initiating subsequent anti-cancer therapy or completing study participation, the PFS will be censored as the last available disease assessment.
|
From the time of enrollment to either the day of first documented disease progression or death from any cause, assessed up to 3 years
|
|
Recurrence-free survival (RFS)
時間枠:Time that elapses between time of surgery and either the day of first documented disease recurrence or death from any cause, assessed up to 3 years
|
Disease recurrence is defined as documented clinical or radiographical progression per physician judgment, or death due to disease.
Disease progression/recurrence, if it occurs, will be noted by the treating clinician.
RFS data will be collected from baseline (day of surgery) until the subject experiences disease recurrence, initiates subsequent anti-cancer therapy, completes study participation, or experiences death from any cause, whichever comes first.
|
Time that elapses between time of surgery and either the day of first documented disease recurrence or death from any cause, assessed up to 3 years
|
|
Overall survival
時間枠:From enrollment to the date of any cause death from or to the date of last follow-up if patients are alive, assessed up to 3 years
|
From enrollment to the date of any cause death from or to the date of last follow-up if patients are alive, assessed up to 3 years
|
協力者と研究者
スポンサー
捜査官
- 主任研究者:Shishir K Maithel, MD、Northwestern University
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
- アミノ酸、ペプチド、およびタンパク質
- タンパク質
- 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
- 調査手法
- 臨床検査技術
- 診断技術と手順
- 診断
- 抗体
- 免疫グロブリン
- 免疫タンパク質
- 血液タンパク質
- 血清グロブリン
- グロブリン
- 無機化学物質
- 塩素化合物
- 窒素化合物
- デオキシシチジン
- シチジン
- ピリミジンヌクレオシド
- ピリミジン
- 要素
- 金属
- 化学技術、分析
- スペクトル分析
- 金属、重い
- 免疫グロブリンアイソタイプ
- 硫化物
- 陰イオン
- イオン
- 電解質
- 硫化水素
- プラチナ化合物
- 遷移要素
- ゲムシタビン
- 免疫グロブリンG
- シスプラチン
- クエン酸1,2-ジアミノシクロヘキサン白金II
- 標本処理
- 磁気共鳴分光法
- Durvalumab
- ジスルフィド
- 白金
- futibatinib
その他の研究ID番号
- NU 25I12 (その他の識別子:Northwestern University)
- P30CA060553 (米国 NIH グラント/契約)
- NCI-2026-03371 (レジストリ識別子:CTRP (Clinical Trial Reporting Program))
- STU00226069
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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