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Phase II Safety and Efficacy Study of Crizotinib in East Asian Patients With ROS1 Positive, ALK Negative Advanced NSCLC

Phase 2
Completed
Conditions
ROS1 Proto Oncogene
Crizotinib
Non Small Cell Lung Cancer
Interventions
Registration Number
NCT01945021
Lead Sponsor
Pfizer
Brief Summary

To assess treatment effectiveness and safety of oral crizotinib administered to East Asian patients with Advanced Non-Small Cell Lung Cancer (NSCLC) that is confirmed to be positive for a ROS1 positive gene mutation (translocation or inversion) and confirmed negative for an ALK mutation

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
129
Inclusion Criteria
  • Histologically or cytologically proven diagnosis of NSCLC that is locally advanced or metastatic
  • treatment-naïve or have received no more than 3 systemic treatment regimen(s)
  • Positive for translocation or inversion events involving the ROS1 gene
  • Negative for translocation or inversion events involving the ALK gene
  • Patients with brain metastases are eligible if asymptomatic, or if treated, must be neurologically stable for at least 2 weeks and are not taking any contraindicated medications
  • Any prior treatment (chemotherapy, radiation [except for palliative], or surgery) must have been completed at least 2 weeks prior to initiation of study medication
  • At least 1 measurable tumor lesion as per RECIST v1.1
  • Female or male, 18 years of age or older
  • ECOG performance status 0 to 1
  • Adequate organ function
  • Signed and dated informed consent
  • Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including completion of the PRO measures
  • Agree to use effective contraception during the study period and for at least 90 days after completion of the study treatment
Exclusion Criteria
  • Current treatment on another therapeutic clinical trial
  • Prior therapy specifically directed against ALK or ROS1 fusion genes
  • Spinal cord compression unless treated with the patient attaining good pain control and stable or recovered neurologic function, carcinomatous meningitis, or leptomeningeal disease
  • known interstitial fibrosis or interstitial lung disease
  • myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, congestive heart failure, or cerebrovascular accident including transient ischemic attack within 3 months prior to start of study treatment
  • Ongoing cardiac dysrhythmias of NCI CTCAE v4.03 Grade >/=2, uncontrolled atrial fibrillation of any grade, or QTc >470 msec
  • Pregnant or breast feeding
  • Use of drugs or foods that are known potent CYP3A4 inhibitors or inducers
  • Use of other anti-cancer drugs including traditional Chinese medicine on the SFDA list
  • Evidence of active malignancy within last 3 years

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
CrizotinibCrizotinibSingle-arm trial whereby all consented, enrolled, eligible patients receive crizotinib
Primary Outcome Measures
NameTimeMethod
Independent Radiology Reviewed Overall Objective Response (ORR)Starting from the first dose study treatment until the first documented CR or PR (every 8 weeks then after 8 cycles at every 12 weeks in duration of 94.0 weeks)

Overall objective response (ORR) was defined as the number of patients with a best overall response of confirmed Complete Response or confirmed Partial Response according to RECIST v1.1 (as determined by Independent Radiology Review \[IRR\]), relative to the total population of response-evaluable participants. Per RECIST v1.1, CR: disappearance of all target lesions. Disappearance of all non-target lesions and normalization of tumor marker level. All lymph nodes must be non-pathological in size (\<10 mm short axis); PR: at least 30% decrease in sum of diameters of target lesions, taking as reference baseline sum diameters. Confirmed responses were those that persisted on repeat imaging at least 4 weeks after the initial documentation of response.

Secondary Outcome Measures
NameTimeMethod
IRR-Assessed Duration of Response (DR)From first documentation of objective tumor response to first documentation of objective PD or death due to any cause, whichever occurred first (every 8 weeks then after 8 cycles at every 12 weeks in duration of 151.3 weeks)

DR: time from first documentation of objective tumor response (CR or PR) to first documentation of objective progressive disease (PD) or to death due to any cause, whichever occurred first. If no progression or death on study was observed, or given antitumor treatment other than study drug, participants were censored on date of last on-study tumor assessment. RECIST v1.1, a) PD: \>=20% increase in sum of diameters of target lesions, taking as reference the smallest sum on study (this includes baseline sum if that is the smallest on study), sum must also demonstrate an absolute increase of \>=5 mm, appearance of 1 or more new lesions, unequivocal progression of existing non-target lesions; b) CR: disappearance of all target lesions. Disappearance of all non-target lesions and normalization of tumor marker level. All lymph nodes must be non-pathological in size (\<10 mm short axis); c) PR: \>=30% decrease in sum of diameters of target lesions, taking as reference baseline sum diameters.

IRR-Assessed Time to Tumor Response (TTR)From date of first dose of crizotinib to first documentation of objective response was observed (every 8 weeks then after 8 cycles at every 12 weeks in duration of 151.3 weeks)

TTR was defined as the time from the date of first dose to first documentation of objective tumor response (CR or PR), that was subsequently confirmed. For participants proceeding from PR to CR, the onset of PR was taken as the onset of response. RECIST v1.1 (as determined by IRR), a) CR: disappearance of all target lesions. Disappearance of all non-target lesions and normalization of tumor marker level. All lymph nodes must be non-pathological in size (\<10 mm short axis); b) PR: at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.

Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious AEs, Treatment Emergent Treatment Related AEs and SAEs, Grade 3 or 4 Treatment Emergent AEs and Grade 3 or 4 Treatment Emergent Treatment Related AEsBaseline up to 28 days after the last dose of study treatment (maximum up to 295.9 weeks)

Treatment-emergent AEs :between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pretreatment state. Relatedness to crizotinib was assessed by the investigator. Treatment-related AE: any untoward medical occurrence attributed to study drug in a participant who received study drug. Serious adverse event (SAE):an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Grade 3 (Severe) events=unacceptable or intolerable events, significantly interrupting usual daily activity, require systemic drug therapy/other treatment. Grade 4 (Life-threatening) events caused participant to be in imminent danger of death.

IRR Assessed Disease Control Rate (DCR) at 8 WeeksAt 8 weeks after the start of study treatment

DCR at 8 weeks was defined as the percentage of participants with a confirmed CR, confirmed PR, or stable disease (SD) at 8 weeks, respectively, relative to the total population of response evaluable participants. RECIST v1.1 (as determined by IRR), a) CR: disappearance of all target lesions. Disappearance of all non-target lesions and normalization of tumor marker level. All lymph nodes must be non-pathological in size (\<10 mm short axis); b) PR: at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.

IRR-Assessed Progression Free Survival (PFS)From the date of first dose of crizotinib until the first documentation of objective PD or death (every 8 weeks then after 8 cycles at every 12 weeks in duration of 151.3 weeks)

PFS was defined as the time from the date of the first dose of crizotinib to first documentation of objective PD or to death on study due to any cause, whichever occurred first. If no progression or death on study was observed, or given antitumor treatment other than study drug, participants were censored on date of last on-study tumor assessment. RECIST v1.1, PD: at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study (this includes baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered a sign of progression. Unequivocal progression of existing non-target lesions.

Overall Survival (OS)From date of the first dose of crizotinib until the date of death from any cause (up to 291.9 weeks)

OS was defined as the time from the date of the first dose of crizotinib to the date of death due to any cause. For participants still alive at the time of analysis, the OS time was censored on the last date the participants were known to be alive.

Number of Participants With a Shift in Hematology Laboratory Results From Baseline Grade </=2 to Worst Grade 3 or Grade 4Baseline up to 28 days after the last dose of study treatment (maximum up to 295.9 weeks)

Laboratory values included hemoglobin increased, anemia, platelet count decreased, leukocytosis, white blood cell decreased, lymphocyte count increased, lymphocyte count decreased and neutrophil count decreased. Laboratory values were defined as National Cancer Institute Common Toxicity Criteria for Adverse Events Version 3.0 (NCI CTCAE v3.0) grade 3 or higher.

Number of Participants With a Shift of Chemistry Laboratory Results From Baseline Grade </=2 to Worst Grade 3 or Grade 4Baseline up to 28 days after the last dose of study treatment (maximum up to 295.9 weeks)

Laboratory values included blood bilirubin increased, alanine aminotransferase increased, aspartate aminotransferase increased, alkaline phosphatase increased, hypoalbuminemia, hypernatremia, hyponatremia, hyperkalemia, hypokalemia, hypercalcemia, hypocalcemia, hypophosphatemia, Creatinine increased, hyperuricemia, hypermagnesemia, hypomagnesemia, hyperglycemia and hypoglycemia. Laboratory values were defined as National Cancer Institute Common Toxicity Criteria for Adverse Events Version 3.0 (NCI CTCAE v3.0) grade 3 or higher.

Change From Baseline to Cycle 60 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life (QOL) Questionnaire Core 30 (QLQ-C30) ScoresBaseline up to Cycle 60

The EORTC QLQ-C30 consists of 30 questions which are incorporated into 5 functional domains (physical, role, cognitive, emotional, and social); a global health status/global QOL scale; 3 symptom scales (fatigue, pain, nausea and vomiting scales); and 6 single items that assess the additional symptoms (dyspnea, appetite loss, sleep disturbance/insomnia, constipation, and diarrhea) and the perceived financial burden of treatment. All the scales and single-item scores ranged from 0 to 100, higher score is indicative of a higher response level (high score for a functional scale represents a high / healthy level of functioning; high score for the global health status / QoL represents a high QoL; a high score for a symptom scale / item represents a high level of symptomatology / problems).

Change From Baseline to Cycle 60 in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Lung Cancer Module 13 ScoresBaseline up to Cycle 60

The EORTC QLQ-LC13 consisted of 1 multi-item scale and 9 single items that assessed specific symptoms (dyspnea, cough, hemoptysis, and site-specific pain), side effects (sore mouth, dysphagia, neuropathy, and alopecia). Scores on each scale and item ranged from 0 to 100, higher score is indicative of a higher response level (a high score for a symptom scale / item represents a high level of symptomatology / problems).

Trial Locations

Locations (44)

National Taiwan University Hospital

🇨🇳

Taipei, Taiwan

Taipei Veterans General Hospital

🇨🇳

Taipei, Taiwan

National Cheng Kung University Hospital

🇨🇳

Tainan, Taiwan

Kinki University Hospital

🇯🇵

Osaka, Japan

Department of Medical Oncology, Cancer Institute and Hospital, Chinese Academy of Medical Sciences

🇨🇳

Chaoyang District, Beijing, China

Guangdong General Hospital

🇨🇳

Guangzhou, Guangdong, China

Department of Thoracic Oncology, Tianjin Medical University Cancer Institute and Hospital

🇨🇳

Hexi District, Tianjin, China

Taichung Veterans General Hospital

🇨🇳

Taichung, Taiwan

National Cancer Center Hospital East

🇯🇵

Chiba, Japan

The First Affiliated Hospital of Anhui Medical University, Department of Medical Oncology

🇨🇳

Hefei, Anhui, China

The Military General Hospital of Beijing PLA / Medical Oncology Dept.

🇨🇳

Dongcheng District, Beijing, China

307 Hospital of PLA/Department of Lung Cancer

🇨🇳

Fengtai District, Beijing, China

Beijing Cancer Hospital, Department of Thoracic Oncology

🇨🇳

Haidian District, Beijing, China

Beijing Chest Hospital

🇨🇳

Tongzhou District, Beijing, China

Chinese PLA General Hospital

🇨🇳

Haidian District, Beijing, China

Respiration department,the First Affiliated Hospital of Third Military Medical University, PLA

🇨🇳

Shapingba District, Chongqing, China

Department of Oncology, Jilin Provincial Cancer Hospital

🇨🇳

Changchun, Jilin, China

The First Affiliated Hospital of Guangzhou Medical College

🇨🇳

Guangzhou, Guangdong, China

SUN Yat-Sen University Cancer Center

🇨🇳

Guangzhou, Guangdong, China

Fujian Province Oncology Hospital

🇨🇳

Fuzhou, Fujian, China

The affiliated hospital of medical college Qingdao University / Medical oncology department

🇨🇳

Qingdao, Shandong, China

Hunan Provincial Tumor Hospital/Division of Oncology

🇨🇳

Changsha, Hunan, China

Department of Pulmonary Medicine, Shanghai Chest Hospital

🇨🇳

Shanghai, Shanghai, China

Shanghai Chest Hospital/Lung cancer clinical center

🇨🇳

Shanghai, Shanghai, China

Shanghai Pulmonary Hospital

🇨🇳

Shanghai, Shanghai, China

Zhongshan Hospital Fudan University / Respiratory Department

🇨🇳

Xuhui District, Shanghai, China

The First Affiliated Hospital of College of Medicine, Zhejiang University

🇨🇳

Hangzhou, Zhejiang, China

Sichuan Province Cancer Hospital/Department of Pulmonary Tumor

🇨🇳

Chengdu, Sichuan, China

Oncology Department, West China Hospital of Sichuan University

🇨🇳

Chengdu, Sichuan, China

Sir Run Run Shaw Hospital of College of Medicine of Zhejiang University, Center for Oncology

🇨🇳

Hangzhou, Zhejiang, China

Zhejiang Cancer Hospital

🇨🇳

Hangzhou, Zhejiang, China

Shizuoka Cancer Center

🇯🇵

Sunto-gun, Shizuoka, Japan

Aichi Cancer Center Hospital

🇯🇵

Aichi, Japan

NHO Shikoku Cancer Center

🇯🇵

Ehime, Japan

Hyogo Cancer Center

🇯🇵

Hyogo, Japan

NHO Kyushu Cancer Center

🇯🇵

Fukuoka, Japan

National Cancer Center Hospital

🇯🇵

Tokyo, Japan

Osaka City General Hospital

🇯🇵

Osaka, Japan

Tohoku University Hospital

🇯🇵

Miyagi, Japan

Cancer Institute Hospital of JFCR

🇯🇵

Tokyo, Japan

Asan Medical Center

🇰🇷

Seoul, Korea, Republic of

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

Yonsei University, Severance Hospital

🇰🇷

Seoul, Korea, Republic of

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