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NCT03292133

A Study of EGF816 and Gefitinib in TKI-naïve EGFR-mutant Non-Small Cell Lung Cancer

Terminated Phase 2 Results posted Last updated 10 June 2025
What this trial tests

Phase 2 trial testing EGF816 in Lung Cancer in 11 participants. Terminated before completion.

Timeline
31 October 2017
Primary endpoint
31 December 2021
4 March 2025

Quick facts

Lead sponsorMassachusetts General Hospital
PhasePhase 2
StatusTerminated
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment11
Start date31 October 2017
Primary completion31 December 2021
Estimated completion4 March 2025
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Massachusetts General Hospital

Who can join

18 and older, any sex, with Lung Cancer. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

9 Months Progression Free Rate Primary · 9 months

The percentage of participants that are free from objective disease progression or death at 9 months after the start treatment. Progression is evaluated using Response Evaluation Criteria In Solid Tumors (RECIST) 1.1. Progressive Disease (PD): At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the 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. (Note: the appearance of one or more new lesions is also

GroupValue95% CI
EGF816 + Gefitinib7
Overall Response Rate (ORR) Secondary · 4 years and 4 months

The number of participants that achieve either a complete response (CR) or a partial response (PR). Response is evaluated using Response Evaluation Criteria In Solid Tumors (RECIST) 1.1. * Complete Response (CR): Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. * Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. All CRs and PRs must be confirmed by a second assessment not earlier than 4 weeks after the criter

GroupValue95% CI
EGF816 + Gefitinib8
Median Overall Survival Secondary · 6 years

Overall survival is defined as time from the start of treatment until death or loss to follow up. Overall survival will be analyzed using the Kaplan-Meier method.

GroupValue95% CI
EGF816 + Gefitinib35.76.18 – NA
Median Progression Free Survival (PFS) Secondary · 4.2 years

PFS is defined as the time from start of treatment to the time of objective disease progression, death, or loss to follow up. Progression is evaluated using Response Evaluation Criteria In Solid Tumors (RECIST) 1.1. Progressive Disease (PD): At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the 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. (Note: the appearance of one or more new lesions is also co

GroupValue95% CI
EGF816 + Gefitinib20.865.42 – 39.57
Summary of Treatment-related Adverse Events Secondary · Start of treatment through 30 days after the end of treatment (max 4 years and 5 months)

Treatment-related adverse events, defined as those at least possibly related to study treatment, are summarized below as a measure of patient safety and treatment tolerability. Subjects were evaluated for adverse events by the Common Terminology Criteria for Adverse Events (CTCAE) v4. For each treatment-related event, subjects are counted below by the highest grade they experienced on study per CTCAE v 4.0 to demonstrate the range and severity of treatment-related toxicities documented on study. Grade refers to the severity of the toxicity, with Grade 1 corresponding to mild toxicity, Grade 2

Anemia
GroupValue95% CI
EGF816 + Gefitinib1
EGF816 + Gefitinib0
EGF816 + Gefitinib0
EGF816 + Gefitinib10
Cardiac disorders - other, specify
GroupValue95% CI
EGF816 + Gefitinib0
EGF816 + Gefitinib1
EGF816 + Gefitinib0
EGF816 + Gefitinib10
Dry eye
GroupValue95% CI
EGF816 + Gefitinib1
EGF816 + Gefitinib0
EGF816 + Gefitinib0
EGF816 + Gefitinib10
Eye Pain
GroupValue95% CI
EGF816 + Gefitinib1
EGF816 + Gefitinib0
EGF816 + Gefitinib0
EGF816 + Gefitinib10
Abdominal Pain
GroupValue95% CI
EGF816 + Gefitinib1
EGF816 + Gefitinib0
EGF816 + Gefitinib0
EGF816 + Gefitinib10
Bloating
GroupValue95% CI
EGF816 + Gefitinib1
EGF816 + Gefitinib0
EGF816 + Gefitinib0
EGF816 + Gefitinib10
Diarrhea
GroupValue95% CI
EGF816 + Gefitinib6
EGF816 + Gefitinib3
EGF816 + Gefitinib2
EGF816 + Gefitinib0
Dry mouth
GroupValue95% CI
EGF816 + Gefitinib3
EGF816 + Gefitinib0
EGF816 + Gefitinib0
EGF816 + Gefitinib8

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events are collected from informed consent until 30 days after the end of treatment (maximum 4 years and 5 months). Patients were monitored for survival up to 6 years.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

EGF816 + Gefitinib
Serious: 2/11 (18%)
Deaths: 4/11

Serious adverse events (2 terms)

ReactionSystemEGF816 + Gefitinib
FeverGeneral disorders
Pericardial effusionCardiac disorders
Other adverse events (104 terms — click to expand)

ReactionSystemEGF816 + Gefitinib
DiarrheaGastrointestinal disorders
Rash maculo-papularSkin and subcutaneous tissue disorders
DyspneaRespiratory, thoracic and mediastinal disorders
Rash acneiformSkin and subcutaneous tissue disorders
CoughRespiratory, thoracic and mediastinal disorders
FatigueGeneral disorders
Alanine aminotransferase increasedInvestigations
Dry skinSkin and subcutaneous tissue disorders
FeverGeneral disorders
HypertensionVascular disorders
PruritusSkin and subcutaneous tissue disorders
Aspartate aminotransferase increasedInvestigations
Back painMusculoskeletal and connective tissue disorders
Dry mouthGastrointestinal disorders
ParonychiaInfections and infestations
ChillsGeneral disorders
Gastroesophageal reflux diseaseGastrointestinal disorders
Mucositis oralGastrointestinal disorders
MyalgiaMusculoskeletal and connective tissue disorders
Pain in extremityMusculoskeletal and connective tissue disorders
Pain of skinSkin and subcutaneous tissue disorders
AlopeciaSkin and subcutaneous tissue disorders
ArthralgiaMusculoskeletal and connective tissue disorders
Cholesterol highInvestigations
DizzinessNervous system disorders
DyspepsiaGastrointestinal disorders
Erythema multiformeSkin and subcutaneous tissue disorders
FloatersEye disorders
Gastrointestinal disorders - Other, specify: mouth soresGastrointestinal disorders
HeadacheNervous system disorders
HyperkalemiaMetabolism and nutrition disorders
NauseaGastrointestinal disorders
Neutrophil count decreasedInvestigations
PainGeneral disorders
Sore throatRespiratory, thoracic and mediastinal disorders
Abdominal painGastrointestinal disorders
Allergic reactionImmune system disorders
AnemiaBlood and lymphatic system disorders
AnorexiaMetabolism and nutrition disorders
AnxietyPsychiatric disorders

Most-reported serious reactions: Fever, Pericardial effusion.

Data from ClinicalTrials.gov NCT03292133 adverse events section.

Sponsor's own description

This research study is studying a combination of drugs as a possible treatment for EGFR mutation-positive lung cancer. The drugs involved in this study are: * EGF816 * Gefitinib

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Targeted therapy in advanced non-small cell lung cancer: current advances and future trends.
    Majeed U, Manochakian R, Zhao Y, Lou Y. · · 2021 · cited 193× · PMID 34238332 · DOI 10.1186/s13045-021-01121-2
  2. EGFR first- and second-generation TKIs-there is still place for them in <i>EGFR</i>-mutant NSCLC patients.
    Karachaliou N, Fernandez-Bruno M, Bracht JWP, Rosell R. · · 2019 · cited 84× · PMID 35117062 · DOI 10.21037/tcr.2018.10.06
  3. Structural Insight and Development of EGFR Tyrosine Kinase Inhibitors.
    Amelia T, Kartasasmita RE, Ohwada T, Tjahjono DH. · · 2022 · cited 83× · PMID 35164092 · DOI 10.3390/molecules27030819
  4. The advance of the third‑generation EGFR‑TKI in the treatment of non‑small cell lung cancer (Review).
    Cheng Z, Cui H, Wang Y, Yang J, et al · · 2024 · cited 37× · PMID 38063215 · DOI 10.3892/or.2023.8675
  5. Drug resistance of targeted therapy for advanced non-small cell lung cancer harbored EGFR mutation: from mechanism analysis to clinical strategy.
    Zhao Y, Wang H, He C. · · 2021 · cited 26× · PMID 34661758 · DOI 10.1007/s00432-021-03828-8
  6. Young lung cancer: from diagnosis to survivorship.
    Florez N, Kiel L, Kaufman R, LoPiccolo J, et al · · 2025 · PMID 40641929 · DOI 10.3389/fonc.2025.1570143

Verify or expand the search:

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Trials by the same sponsor.

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