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Trastuzumab Deruxtecan in Participants With HER2-mutated Metastatic Non-small Cell Lung Cancer (NSCLC)

Phase 2
Completed
Conditions
Non-Small Cell Lung Cancer
Interventions
Registration Number
NCT04644237
Lead Sponsor
Daiichi Sankyo
Brief Summary

This study was designed to evaluate the safety and efficacy of trastuzumab deruxtecan in HER2-mutated metastatic non-small cell lung cancer (NSCLC) participants who had disease recurrence or progression during/after at least one regimen of prior anticancer therapy (second line or later) that must have contained a platinum-based chemotherapy drug.

Detailed Description

This randomized, two-arm, phase 2, multicenter study will evaluate the safety and efficacy of 5.4 mg/kg and 6.4 mg/kg trastuzumab deruxtecan administered every 3 weeks (Q3W) in participants with HER2-mutated metastatic NSCLC. Each participant is expected to receive approximately 14 months of trastuzumab deruxtecan treatment. The primary endpoint of the study will be confirmed objective response rate (blinded independent central review). Secondary endpoints will include, but not limited to, disease control rate, duration of response, progression-free survival, objective response rate (investigator), overall survival, and safety.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
152
Inclusion Criteria
  • Written informed consent
  • Men or women ≥18 years, follow local regulatory requirements if the legal age of the consent for study participation is >18 years
  • Pathologically documented metastatic NSCLC with a known activating HER2 mutation. Note: A HER2 mutation documented only from a liquid biopsy samples cannot be used for enrollment.
  • Had previous treatment including platinum therapy in the metastatic/locally advanced setting and not amenable to curative surgery or radiation. Participant must have progressed during or after the last treatment regimen or discontinued because of unacceptable toxicity.
  • Presence of at least 1 measurable lesion confirmed by the blinded Independent Central Review based on RECIST version 1.1
  • Willing and able to provide an archival tumor tissue sample. A fresh biopsy is required if an archival tumor tissue sample cannot be supplied. Resection and core needle biopsy are acceptable. Fine needle aspirates or cell block are not acceptable.
  • Eastern Cooperative Oncology Group performance status 0 to 1
  • Left ventricular ejection fraction ≥ 50% within 28 days before randomization Resection and core needle biopsy are acceptable - Adequate organ function as specified in protocol within 14 days before randomization
  • Adequate treatment washout period before randomization
  • Participants of reproductive/childbearing potential agree to use a highly effective form of contraception (or avoid intercourse) during study period and up to 7 months (females) and 4 months (males) after last study dose
  • Males should not freeze or donate sperm throughout the study period up to at least 4 months after last study dose; females should not donate or retrieve ova for their own use throughout the study period and up to at least 7 months after last study dose
  • Life expectancy 3 months or more
Exclusion Criteria
  • Known driver mutation in the epidermal growth factor receptor (EGFR), BRAF, or MET exon 14 gene or a known anaplastic lymphoma kinase (ALK), ROS1, RET, or NTRK fusion
  • Medical history of myocardial infarction within 6 months before randomization, symptomatic congestive heart failure (CHF) (New York Heart Association Class II to IV). Participants with troponin levels above upper limit of normal at screening (as defined by the manufacturer) and without any myocardial infarction (MI)-related symptoms should have a cardiologic consultation before randomization to rule out MI
  • Corrected QT interval (QTcF) prolongation > 470 msec (females) or >450 msec (males) based on average of the triplicate12-lead electrocardiogram at screening
  • History of non-infectious interstitial lung disease (ILD)/pneumonitis that required steroids, current ILD/pneumonitis, or where suspected ILD/pneumonitis cannot be ruled out by imaging at screening
  • Spinal cord compression or clinically active central nervous system metastases, defined as untreated and symptomatic, or requiring therapy with corticosteroids or anticonvulsants to control associated symptoms
  • Multiple primary malignancies within 3 years, except adequately resected non-melanoma skin cancer, curatively treated in-situ disease, or other solid tumors curatively treated
  • History of severe hypersensitivity reactions to either the drug substances or inactive ingredients in the drug product
  • History of severe hypersensitivity reactions to other monoclonal antibodies
  • Uncontrolled infection requiring IV antibiotics, antivirals, or antifungals
  • Substance abuse or any other medical conditions such as clinically significant cardiac or psychological conditions, that may, in the opinion of the investigator, interfere with the participant's participation in the clinical study or evaluation of the clinical study results
  • Known human immunodeficiency virus (HIV) infection
  • Known active, clinically relevant liver disease (eg, active hepatitis B, or active hepatitis C) such as those with serologic evidence of viral infection within 28 days of Cycle 1, Day 1
  • Unresolved toxicities from previous anticancer therapy, defined as toxicities (other than alopecia) not yet resolved to Grade ≤ 1 or baseline
  • Pregnant, breastfeeding, or planning to become pregnant
  • Otherwise considered inappropriate for the study by the Investigator
  • Lung-specific intercurrent clinically significant illnesses including, but not limited to, any underlying pulmonary disorder (eg. pulmonary emboli within three months of the study randomization, severe asthma, severe COPD, restrictive lung disease, pleural effusion, etc.)
  • Any autoimmune, connective tissue or inflammatory disorders (e.g., Rheumatoid arthritis, Sjogren's, sarcoidosis etc.) where there is documented, or a suspicion of pulmonary involvement at the time of screening
  • Prior complete pneumonectomy
  • Had prior treatment with any agent, including an antibody drug conjugate (ADC), containing a chemotherapeutic agent targeting topoisomerase I

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Trastuzumab deruxtecan 6.4 mg/kgTrastuzumab deruxtecanParticipants will be randomized to receive trastuzumab deruxtecan 6.4 mg/kg administered by intravenous infusion every 3 weeks (Q3W).
Trastuzumab deruxtecan 5.4 mg/kgTrastuzumab deruxtecanParticipants will be randomized to receive trastuzumab deruxtecan 5.4 mg/kg administered by intravenous infusion every 3 weeks (Q3W).
Primary Outcome Measures
NameTimeMethod
Percentage of Participants With Confirmed Objective Response Rate by Blinded Independent Central Review Following Intravenous Administration of Trastuzumab Deruxtecan in Participants With Metastatic Non-small Cell Lung Cancer9 months after the last participant is randomized to data cut off, up to approximately 21 months

Confirmed objective response rate (ORR), defined as the proportion of participants with complete response (CR) or partial response (PR), was assessed by blinded independent central review (BICR) based on Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1. CR was defined as a disappearance of all target lesions and PR was defined as at least a 30% decrease in the sum of diameters of target lesions.

Secondary Outcome Measures
NameTimeMethod
Pharmacokinetic Parameter Area Under the Serum Concentration-Time Curve (AUC) for Trastuzumab Deruxtecan, Total Anti-HER2 Antibody, and Active Metabolite MAAA-1181aCycle 1 Day 1; Cycle 2 Day 1, and Cycle 3 Day 1: pre- and post-dose; Cycle 1 Day 8: 7 days post-dose; Cycle 1 Day 15: 14 days post-dose; Cycle 4 Day 1 and Cycle 6 Day 1: pre-dose (each cycle is 21 days)
Percentage of Participants With Confirmed Objective Response Rate by Investigator Following Intravenous Administration of Trastuzumab Deruxtecan in Participants With Metastatic Non-small Cell Lung CancerTumors9 months after the last participant is randomized or later to data cut off, up to approximately 35 months

Confirmed objective response rate (ORR), defined as the percentage of participants with complete response (CR) or partial response (PR), will be assessed by the Investigator based on Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1. CR was defined as a disappearance of all target lesions and PR was defined as at least a 30% decrease in the sum of diameters of target lesions.

Duration of Response Following Intravenous Administration of Trastuzumab Deruxtecan in Participants With Metastatic Non-small Cell Lung Cancer9 months after the last participant is randomized or later to data cut off, up to approximately 35 months

Duration of response (DoR) is defined as the time from the initial response (complete response \[CR\] or partial response \[PR\]) until documented tumor progression or death from any cause. DoR is only defined for participants who achieved confirmed CR or PR. CR was defined as a disappearance of all target lesions and PR was defined as at least a 30% decrease in the sum of diameters of target lesions.

The Percentage of Participants Reporting Treatment-emergent Adverse Events Following Intravenous Administration of Trastuzumab Deruxtecan in Participants With Metastatic Non-small Cell Lung Cancer Tumors9 months after the last participant is randomized or later to data cut off, up to approximately 35 months
Pharmacokinetic Parameter Minimum Observed Concentration (Ctrough) for Trastuzumab Deruxtecan, Total Anti-HER2 Antibody, and Active Metabolite MAAA-1181aCycle 1 Day 1; Cycle 2 Day 1, and Cycle 3 Day 1: pre- and post-dose; Cycle 1 Day 8: 7 days post-dose; Cycle 1 Day 15: 14 days post-dose; Cycle 4 Day 1 and Cycle 6 Day 1: pre-dose (each cycle is 21 days)
Disease Control Rate Following Intravenous Administration of Trastuzumab Deruxtecan in Participants With Metastatic Non-small Cell Lung Cancer9 months after the last participant is randomized or later to data cut off, up to approximately 35 months

Disease control rate (DCR) is the sum of complete response (CR), partial response (PR), and stable disease (SD) rates. CR was defined as a disappearance of all target lesions, PR was defined as at least a 30% decrease in the sum of diameters of target lesions, and stable disease (SD) was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD; at least a 20% increase in the sum of diameters of target lesions.

Progression-free Survival Following Intravenous Administration of Trastuzumab Deruxtecan in Participants With Metastatic Non-small Cell Lung Cancer9 months after the last participant is randomized or later to data cut off, up to approximately 35 months

Progression-free survival (PFS) is defined as the time from date of randomization until first objective radiographic tumor progression or death from any cause, based on blinded independent central review (BICR) and investigator assessment.

Overall Survival Following Intravenous Administration of Trastuzumab Deruxtecan in Participants With Metastatic Non-small Cell Lung Cancer Tumors9 months after the last participant is randomized or later to data cut off, up to approximately 35 months

Overall survival (OS) is defined as the time from date of randomization until death from any cause.

Pharmacokinetic Parameter Maximum Serum Concentration (Cmax) for Trastuzumab Deruxtecan, Total Anti-HER2 Antibody, and Active Metabolite MAAA-1181aCycle 1 Day 1; Cycle 2 Day 1, and Cycle 3 Day 1: pre- and post-dose; Cycle 1 Day 8: 7 days post-dose; Cycle 1 Day 15: 14 days post-dose; Cycle 4 Day 1 and Cycle 6 Day 1: pre-dose (each cycle is 21 days)
Incidence of Anti-Drug Antibodies (ADA) Following Intravenous Administration of Trastuzumab Deruxtecan in Participants With Metastatic Non-small Cell Lung Cancer TumorsPre-dose on Day 1 of Cycles 1, 2 and 4, and then every 4 cycles (each cycle is 21 days)
Time to Deterioration in European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life Questionnaire (QLQ-C30) ScoresOn Day 1 of every cycle (each cycle is 21 days), and at end of treatment visit 40-day follow-up visit

The EORTC QLQ-C30 consists of 30 questions assessing global health-related quality of life, five aspects of subject functioning (physical, emotional, role, cognitive, and social), three symptom scales (fatigue, nausea and vomiting, and pain), and six single-items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). All of the scales and single-item measures range in score from 0 to 100. Higher scores for functioning scales and global health status indicate a better level of functioning while higher scores on the symptom and single-item scales indicate a higher level of symptoms.

Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life Questionnaire (QLQ-C30) and EORTC Quality of Life Questionnaire for Lung Cancer Trials (QLQ-LC13) ScoresOn Day 1 of every cycle (each cycle is 21 days), and at end of treatment visit 40-day follow-up visit

The EORTC QLQ-C30 consists of 30 questions assessing global health-related quality of life, five aspects of subject functioning (physical, emotional, role, cognitive, and social), three symptom scales (fatigue, nausea and vomiting, and pain), and six single-items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). All of the scales and single-item measures range in score from 0 to 100. Higher scores for functioning scales and global health status indicate a better level of functioning while higher scores on the symptom and single-item scales indicate a higher level of symptoms. The QLQ-LC13 is a 13-item questionnaire designed to assess lung cancer-related symptoms and treatment side effects. The scales ranges from 1=not at all to 4=very much. The summation of scores range from 0 to 100, where higher scores represent increasing symptoms levels.

scales.

Trial Locations

Locations (50)

Washington University School of Medicine

🇺🇸

Saint Louis, Missouri, United States

University Health Network

🇨🇦

Toronto, Canada

Princess Alexandra Hospital

🇦🇺

Woolloongabba, Australia

St John of God Subiaco Hospital

🇦🇺

Subiaco, Australia

Centre Leon Berard

🇫🇷

Lyon, France

Assistance Publique Hopitaux de Marseille AP-HM, Hopital NORD

🇫🇷

Marseille, France

CHU Nantes

🇫🇷

Saint-Herblain, France

CHU toulouse - hôpital Larrey

🇫🇷

Toulouse, France

SC Oncologia, AOU Policlinico Modena

🇮🇹

Modena, Italy

Gustav Roussy

🇫🇷

Villejuif, France

National Cancer Central Hospital

🇯🇵

Chuo Ku, Japan

National Cancer Center Hospital East

🇯🇵

Kashiwa, Japan

Aichi Cancer Center Hospital

🇯🇵

Chikusa, Japan

Okayama University Hospital

🇯🇵

Okayama, Japan

Kindai University Hospital

🇯🇵

Ōsaka-sayama, Japan

Seoul National University Bundang Hospital

🇰🇷

Seongnam, Korea, Republic of

Chungbuk National University Hospital

🇰🇷

Chungbuk, Korea, Republic of

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

Hospital Universitario Vall d'Hebron

🇪🇸

Barcelona, Spain

Asan Medical Center

🇰🇷

Seoul, Korea, Republic of

Netherlands Cancer Institute

🇳🇱

Amsterdam, Netherlands

Clinica Universidad de Navarra

🇪🇸

Madrid, Spain

Erasmus MC

🇳🇱

Rotterdam, Netherlands

Hospital Universitario Fundacion Jimenez Diaz

🇪🇸

Madrid, Spain

Hospital 12 de Octubre

🇪🇸

Madrid, Spain

Clinica Univ. de Navarra - P

🇪🇸

Pamplona, Spain

Hospital Universitari i Politècnic La Fe

🇪🇸

Valencia, Spain

National Cheng Kung University Hospital NCKUH

🇨🇳

Tainan, Taiwan

University of Colorado Denver - Anschutz Medical Campus

🇺🇸

Aurora, Colorado, United States

AdventHealth Orlando

🇺🇸

Orlando, Florida, United States

H. Lee Moffitt Cancer Center & Research Institute

🇺🇸

Tampa, Florida, United States

Norton Cancer Institute

🇺🇸

Louisville, Kentucky, United States

Massachusetts General Hospital (MGH) - Hematology/Oncology

🇺🇸

Boston, Massachusetts, United States

Dana-Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Henry Ford Cancer Institute/Henry Ford Hospital

🇺🇸

Detroit, Michigan, United States

Virgina Cancer Specialists

🇺🇸

Fairfax, Virginia, United States

University of Michigan

🇺🇸

Detroit, Michigan, United States

Istituto Nazionale Tumori Fondazione G. Pascale di Napoli Strutturadi Oncologia

🇮🇹

Napoli, Italy

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

Chang Gung Memorial Hospital CGMH - LinKou Branch

🇨🇳

Taoyuan, Taiwan

Hopital Pontchaillou

🇫🇷

Rennes, France

Azienda Ospedaliero-Universitaria San Luigi Gonzaga

🇮🇹

Orbassano, Italy

Chung Shan Medical University Hospital

🇨🇳

Taichung, Taiwan

Taichung Veterans General Hospital

🇨🇳

Taichung, Taiwan

Sarah Cannon Research Institute

🇺🇸

Nashville, Tennessee, United States

Ospedale San Luca

🇮🇹

Lucca, Italy

IRCCS Istituto Europeo di Oncologia

🇮🇹

Milano, Italy

Humanitas Cancer Center Istituto Clinico Humanitas

🇮🇹

Rozzano, Italy

National Hospital Organization Kyushu Cancer Center

🇯🇵

Fukuoka, Japan

National Taiwan University Hospital

🇨🇳

Taipei City, Taiwan

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