CClinicalTrials.gg
TerminatedNCT03447769Canopy-AUpdated Oct 9, 2024Results posted

Brief Title: Study of Efficacy and Safety of Canakinumab as Adjuvant Therapy in Adult Subjects With Stages AJCC/UICC v. 8 II-IIIA and IIIB (T>5cm N2) Completely Resected Non-small Cell Lung Cancer Acronym: CANOPY-A

A Phase 3 interventional study of Canakinumab and Placebo in Non-Small Cell Lung Cancer, sponsored by Novartis Pharmaceuticals. Terminated at 283 sites in 41 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-10-09.

Sponsored by Novartis Pharmaceuticals · Phase 3, Interventional, and Treatment

Why this study was terminated
Upon review of the primary analysis results, the benefit-risk was assessed by the Steering Committee Members with the decision that the study will be closed. No new safety signals were observed for canakinumab.
Phase
Phase 3
Study type
Interventional
Enrollment
1,382
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

The primary purpose of the study was to compare the efficacy and safety of canakinumab versus placebo as adjuvant therapy in adult subjects with stages II -IIIA according to the 8th edition of the American Joint Committee on Cancer (AJCC)/Union for International Cancer Control (UICC) and the subset of IIIB (T>5cm N2 disease) completely resected (R0) non-small cell lung cancer (NSCLC).

Read the detailed description

This was a phase III, multicenter, randomized, double-blind study to evaluate the efficacy and safety of canakinumab as adjuvant therapy in adult patients with stages AJCC/UICC v.8 II-IIIA and IIIB (T>5 cm N2) completely resected (R0) NSCLC.

Approximately 1500 patients were planned to be randomized 1:1 to canakinumab, 200 mg subcutaneously (s.c.) every 3 weeks or matching placebo s.c. every 3 weeks. Patients were planned to continue their assigned treatment until they completed 18 cycles (cycle= 21 days) or experienced any one of the following: non-small cell lung cancer (NSCLC) disease recurrence as determined by Investigator; unacceptable toxicity that precluded further treatment; treatment discontinuation at the discretion of the Investigator or patient; start of a new antineoplastic therapy; death, or loss to follow-up, whichever occurred first. All patients who discontinued from the study treatment were to be followed up every 12 weeks for survival until the final OS analysis or death, loss to follow-up or withdrawal of consent for survival follow-up.

02

Conditions studied

  • Non-Small Cell Lung Cancer

Keywords

  • Non-Small Cell Lung Cancer
  • NSCLC
  • ACZ885
  • canakinumab
  • adjuvant
  • AJCC/UICC v. 8 stages II-IIIA and IIIB (T>5cm N2)
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  • Had completely resected (R0) NSCLC AJCC/UICC v. 8 stage IIA-IIIA and IIIB (N2 disease only) OR had NSCLC Stage IIA-IIIA, IIIB (N2 disease only) and were candidates for complete resection surgery.
  • Cisplatin-based chemotherapy was mandatory for all subjects (Exception: In subjects with stage IIA disease with no nodal involvement, cisplatin-based chemotherapy could be administered if recommended by the treating physician). When required, a minimum of two cycles of cisplatin-based chemotherapy was mandatory, after which additional therapies could be given based upon local clinical practice and/or guidelines. Typically, chemotherapy was initiated within 60 days of surgery.
  • Radiation therapy was allowed if indicated as per local guidelines or practice.
  • Had recovered from all toxicities related to prior systemic therapy to grade ≤ 1 (CTCAE v 5.0). Exception to this criterion: subjects with any grade of alopecia and grade 2 or less neuropathy were allowed to enter the study
  • Had ECOG performance status (PS) of 0 or 1

Key Exclusion Criteria:

  • Had unresectable or metastatic disease, positive microscopic margins on the pathology report, and/or gross disease remaining at the time of surgery
  • Had received any neoadjuvant therapy
  • Had presence or history of a malignant disease, other than the resected NSCLC, that had been diagnosed and/or required therapy within the past 3 years Exceptions to this exclusion included the following: completely resected basal cell and squamous cell skin cancers, completely resected carcinoma in situ of any type and hormonal maintenance for breast and prostate cancer > 3 years.
  • Had a history of current diagnosis of cardiac disease
  • Had uncontrolled diabetes
  • Had known active or recurrent hepatic disorder including cirrhosis, hepatitis B and C (positive or indeterminate central laboratory results)
  • Subjects had to be evaluated for tuberculosis as per local treatment guidelines or clinical practice. Subjects with active tuberculosis were not eligible.
  • Had suspected or proven immunocompromised state as described in the protocol
  • Had live and attenuated vaccination within 3 months prior to first dose of study drug (e.g. MMR, Yellow Fever, Rotavirus, Smallpox, etc.).
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
1,382 participants (actual)

Study arms

  • Experimental
    canakinumab

    Participants received 200mg of canakinumab subcutaneously every 3 weeks for up to 18 cycles (approximately 54 weeks)

    Drug: Canakinumab

  • Placebo comparator
    Placebo

    Participants received canakinumab placebo subcutaneously every 3 weeks for up to 18 cycles (approximately 54 weeks)

    Drug: Placebo

Interventions

  • DrugCanakinumab

    200 mg of canakinumab administered subcutaneously on day 1 of every 21-day cycle for 18 cycles. Canakinumab solution for injection was provided by Novartis as ready-to-use pre-filled syringes to be administered by study personnel.

    Also known as: ACZ885

  • DrugPlacebo

    Placebo administered subcutaneously on day 1 of every 21-day cycle for 18 cycles. Placebo solution for injection was provided by Novartis as ready-to-use pre-filled syringes to be administered by study personnel.

05

What researchers measure

Primary outcomes

  1. Disease Free Survival (DFS) by Local Investigator

    DFS is the time from the date of randomization to the date of the first documented NSCLC disease recurrence as assessed by local investigator radiologically or death due to any cause. Disease recurrence included diagnoses of new primary lung malignancies. Clinical deterioration was not considered as a recurrence of disease. In case of non-conclusive radiological evidence, a biopsy assessment was performed to confirm NSCLC recurrence. The median DFS was estimated using the Kaplan-Meier method. DFS was censored if no DFS event was observed prior to the analysis cut-off date or subjects who received any subsequent anti-neoplastic therapy for NSCLC. The censoring date was the date of last assessment before the cut-off date or NSCLC related anti-neoplastic therapy date.

    Time frame: Up to approximately 4 years

Secondary outcomes

  1. Overall Survival (OS)

    Overall Survival (OS) is the time from the date of randomization to the date of death due to any cause. The OS was censored at the latest date the subject was known to be alive. The OS distribution was estimated using the Kaplan-Meier method, and Kaplan-Meier medians and 95% confidence intervals of the medians were presented for each treatment group.

    Time frame: Up to approximately 4.3 years

  2. Overall Survival (OS) in PD-L1 Subgroups

    Overall Survival (OS) is the time from the date of randomization to the date of death due to any cause. The OS was censored at the latest date the subject was known to be alive. The OS distribution was estimated using the Kaplan-Meier method, and Kaplan-Meier curves, medians and 95% confidence intervals of the medians were presented for each treatment group. OS analysis was performed by programmed cell death-ligand 1 (PD-L1) expression status: PD-L1 \<1%, PD-L1 ≥1% and \<49%, and PD-L1 ≥50%.

    Time frame: Up to approximately 4.3 years

  3. Overall Survival (OS) in CD8 Subgroups

    Overall Survival (OS) is the time from the date of randomization to the date of death due to any cause. The OS was censored at the latest date the subject was known to be alive. The OS distribution was estimated using the Kaplan-Meier method, and Kaplan-Meier medians and 95% confidence intervals of the medians were presented for each treatment group. OS analysis was performed by CD8 subgroups with the median of baseline CD8 expression as cut-off.

    Time frame: up to approximately 4.3 years

  4. Lung Cancer Specific Survival (LCSS)

    LCSS is defined as the time from date of randomization to the date of death due to lung cancer. The LCSS distribution was estimated using the Kaplan-Meier method, and Kaplan-Meier medians and 95% confidence intervals of the medians were presented for each treatment group.

    Time frame: Up to approximately 4.3 years

  5. Disease Free Survival (DFS) by Local Investigator in PD-L1 Subgroups

    DFS is the time from the date of randomization to the date of the first documented NSCLC disease recurrence as assessed by local investigator radiologically or death due to any cause. Disease recurrence included diagnoses of new primary lung malignancies. Clinical deterioration was not considered as a recurrence of disease. In case of non-conclusive radiological evidence, a biopsy assessment was performed to confirm NSCLC recurrence. The median DFS was estimated using the Kaplan-Meier method. DFS was censored if no DFS event was observed prior to the analysis cut-off date or subjects who received any subsequent anti-neoplastic therapy for NSCLC. The censoring date was the date of last assessment before the cut-off date or NSCLC related anti-neoplastic therapy date. DFS analysis was performed by baseline programmed cell death-ligand 1 (PD-L1) expression status: PD-L1 \<1%, PD-L1 ≥1% and \<49%, and PD-L1 ≥50%.

    Time frame: Up to approximately 4 years

  6. Disease Free Survival (DFS) by Local Investigator in CD8 Subgroups

    DFS is the time from the date of randomization to the date of the first documented NSCLC disease recurrence as assessed by local investigator radiologically or death due to any cause. Disease recurrence included diagnoses of new primary lung malignancies. Clinical deterioration was not considered as a recurrence of disease. In case of non-conclusive radiological evidence, a biopsy assessment was performed to confirm NSCLC recurrence. The median DFS was estimated using the Kaplan-Meier method. DFS was censored if no DFS event was observed prior to the analysis cut-off date or subjects who received any subsequent anti-neoplastic therapy for NSCLC. The censoring date was the date of last assessment before the cut-off date or NSCLC related anti-neoplastic therapy date. DFS analysis was performed by CD8 subgroups with the median of baseline CD8 expression as cut-off.

    Time frame: Up to approximately 4 years

  7. Canakinumab Serum Concentrations

    Serum concentrations of canakinumab were determinded using an ELISA method.

    Time frame: Cycle 1 on day 1 (pre-dose), day 8 and 15; Cycle 2, 4, 6, 9 and 12 on day 1 (pre-dose). Cycle=21 days

  8. Canakinumab Anti-drug Antibody (ADA) Prevalence at Baseline

    Canakinumab ADA prevalence at baseline was calculated as the percentage of participants who had an ADA positive result at baseline

    Time frame: Baseline

  9. Canakinumab ADA Incidence

    Canakinumab ADA incidence on-treatment was calculated as the percentage of participants who were treatment-induced ADA positive (post-baseline ADA positive with ADA-negative sample at baseline) and treatment-boosted ADA positive (post-baseline ADA positive with titer that was at least the fold titer change greater than the ADA-positive baseline titer)

    Time frame: From baseline up to 130 days after end of treatment, assessed up to approx. 1.5 years

  10. Time to Definitive 10 Point Deterioration Symptom Scores of Pain,Cough and Dyspnea Per European Organization for Research and Treatment of Cancer Quality of Life (EORTC QLQ)- Lung Cancer (LC) 13 Questionnaire

    The Lung Cancer module of the EORTC's quality of life questionnaire (EORTC QLQ-LC13) was used in conjunction with the EORTC QLQ-C30 and provided information on an additional 13 items specifically related to lung cancer. The lung cancer module incorporated one multi-item scale to assess dyspnea, and 9 single items assessing pain, coughing, sore mouth, dysphagia, peripheral neuropathy, alopecia, and hemoptysis. All of the domain scores ranged from 0 to 100. A high score indicated a high level of symptoms. The time to definitive 10 point deterioration symptom scores of pain, cough and dyspnea was defined as the time from the date of randomization to the date of event, which was defined as at least 10 points relative to baseline worsening of the EORTC QLQ-LC13 symptom score with no later change below this threshold or death due to any cause, whichever occurred earlier.

    Time frame: From baseline up to approximately 4 years

  11. Time to Definitive 10 Point Deterioration of Global Health Status/Quality of Life (QoL), Shortness of Breath and Pain Per EORTC QLQ-C30 Questionnaire

    The EORTC QLQ-C30 was a questionnaire developed to assess the health-related quality of life of cancer participants. It assessed 15 domains consisting of 5 functional domains (physical, role, emotional, cognitive, social) and 9 symptom domains (fatigue, nausea and vomiting, pain, dyspnea, insomnia, appetite loss, constipation, diarrhea, financial difficulties) and a global health status/QoL scale. All domain scores ranged from 0 to 100. A high score for the functional or global health status scales indicated a high level of functioning or QoL; a high score for a symptom scale indicated a high level of symptoms. The time to definitive 10 point deterioration of global health status/QoL, shortness of breath and pain was defined as the time from the date of randomization to the date of event, which was defined as at least 10 points relative to baseline worsening of the EORTC QLQ-C30 score with no later change below this threshold or death due to any cause, whichever occured earlier.

    Time frame: From baseline up to approximately 4 years

  12. Time to First 10 Point Deterioration for Symptom Scores of Pain, Cough and Dyspnea Per EORTC QLQ-LC13 Questionnaire

    The Lung Cancer module of the EORTC's quality of life questionnaire (EORTC QLQ-LC13) was used in conjunction with the EORTC QLQ-C30 and provided information on an additional 13 items specifically related to lung cancer. The lung cancer module incorporated one multi-item scale to assess dyspnea, and 9 single items assessing pain, coughing, sore mouth, dysphagia, peripheral neuropathy, alopecia, and hemoptysis. All of the domain scores ranged from 0 to 100. A high score indicated a high level of symptoms. The time to first 10 point deterioration symptom scores of pain, cough and dyspnea was defined as the time from the date of randomization to the first onset of at least 10 points absolute increase from baseline (worsening) in symptoms scores or death due to any cause, whichever occurred earlier.

    Time frame: From baseline up to approximately 4 years

  13. Time to First 10 Point Deterioration of Global Health Status/QoL, Shortness of Breath and Pain Per EORTC QLQ-C30 Questionnaire

    The EORTC QLQ-C30 was a questionnaire developed to assess the health-related quality of life of cancer participants. It assessed 15 domains consisting of 5 functional domains (physical, role, emotional, cognitive, social) and 9 symptom domains (fatigue, nausea and vomiting, pain, dyspnea, insomnia, appetite loss, constipation, diarrhea, financial difficulties) and a global health status/QoL scale. All domain scores ranged from 0 to 100. A high score for the functional or global health status scales indicated a high level of functioning or QoL; a high score for a symptom scale indicated a high level of symptoms. The time to first 10 point deterioration of global health status/QoL, shortness of breath and pain scores was defined as the time from the date of randomization to the first onset of at least 10 points absolute increase from baseline (worsening) in symptoms scores or death due to any cause, whichever occurred earlier.

    Time frame: From baseline up to approximately 4 years

  14. Change From Baseline in the Utility Score of the EuroQoL- 5 Dimension- 5 Level (EQ-5D-5L)

    EQ-5D-5L was a standardized questionnaire that measured health-related QoL. EQ-5D-5L consisted of 2 components: a health state profile and a visual analogue scale. The health state profile included five dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression, each with five levels ranging from 1 (no problems) to 5 (extreme problems). The EQ-5D-5L health state profile responses were converted into single index utility score, ranging from -1 to 1, where lower scores representing a higher level of dysfunction. Published weights are available enabling the calculation of the utility score. A positive change from baseline indicated improvement. This endpoint was assessed throughout the study, including safety and efficacy follow-up (FU) visits. Safety FU visits: every 4 weeks after end of treatment up to 130 days post-last dose. Efficacy FU visits: at 18, 24, 30, 36 and 48 months post-randomization (if no recurrence observed during treatment or safety FU)

    Time frame: Baseline, every 3 weeks for 14 months; end of treatment; every 4 weeks up to 130 days post-treatment; at 18,24,30,36 and 48 months post-randomization (if no recurrence); 7 and 28 days post-disease progression, up to approx. 4 years.

06

Results

Posted Jan 30, 2024

Participant flow

The study was conducted across 290 centers in 41 countries. A total of 1830 subjects were screened of which 1382 participants were randomized to treatment on a 1:1 basis.

Participant flow — Overall Study
MilestoneCanakinumabPlacebo
Started693689
Treated692689
Completed414420
Not completed279269
Withdrew: Progressive disease138148
Withdrew: Study terminated by sponsor6044
Withdrew: Adverse event3431
Withdrew: Patient decision2727
Withdrew: Protocol deviation46
Withdrew: Death27
Withdrew: Technical problems10
Withdrew: Lost to follow-up01
Withdrew: Physician decision135

Outcome measures

PrimaryDisease Free Survival (DFS) by Local Investigator

DFS is the time from the date of randomization to the date of the first documented NSCLC disease recurrence as assessed by local investigator radiologically or death due to any cause. Disease recurrence included diagnoses of new primary lung malignancies. Clinical deterioration was not considered as a recurrence of disease. In case of non-conclusive radiological evidence, a biopsy assessment was performed to confirm NSCLC recurrence. The median DFS was estimated using the Kaplan-Meier method. DFS was censored if no DFS event was observed prior to the analysis cut-off date or subjects who received any subsequent anti-neoplastic therapy for NSCLC. The censoring date was the date of last assessment before the cut-off date or NSCLC related anti-neoplastic therapy date.

Time frame:
Up to approximately 4 years
Reported as:
Median · Months
Disease Free Survival (DFS) by Local Investigator
MonthsCanakinumabPlacebo
Disease Free Survival (DFS) by Local Investigator35.02 (28.55 to NA)29.73 (23.72 to NA)
Statistical analysis
  • Canakinumab vs Placebo · Stratified log-rank test · p = 0.258 (1-sided p-value) · Hazard ratio (hr): 0.94 · 95% CI 0.78 to 1.14The HR for DFS was calculated, along with its 95% confidence interval, from a stratified Cox model using the same stratification factors as those for the log-rank test.
SecondaryOverall Survival (OS)

Overall Survival (OS) is the time from the date of randomization to the date of death due to any cause. The OS was censored at the latest date the subject was known to be alive. The OS distribution was estimated using the Kaplan-Meier method, and Kaplan-Meier medians and 95% confidence intervals of the medians were presented for each treatment group.

Time frame:
Up to approximately 4.3 years
Reported as:
Median · Months
Overall Survival (OS)
MonthsCanakinumabPlacebo
Overall Survival (OS)51.12 (46.95 to NA)NA (NA to NA)
SecondaryOverall Survival (OS) in PD-L1 Subgroups

Overall Survival (OS) is the time from the date of randomization to the date of death due to any cause. The OS was censored at the latest date the subject was known to be alive. The OS distribution was estimated using the Kaplan-Meier method, and Kaplan-Meier curves, medians and 95% confidence intervals of the medians were presented for each treatment group. OS analysis was performed by programmed cell death-ligand 1 (PD-L1) expression status: PD-L1 \<1%, PD-L1 ≥1% and \<49%, and PD-L1 ≥50%.

Time frame:
Up to approximately 4.3 years
Reported as:
Median · Months
Overall Survival (OS) in PD-L1 Subgroups
MonthsCanakinumabPlacebo
PD-L1 <1%NA (NA to NA)NA (NA to NA)
PD-L1 ≥1% and <49%46.95 (22.11 to NA)NA (NA to NA)
PD-L1 ≥50%51.12 (NA to NA)NA (NA to NA)
SecondaryOverall Survival (OS) in CD8 Subgroups

Overall Survival (OS) is the time from the date of randomization to the date of death due to any cause. The OS was censored at the latest date the subject was known to be alive. The OS distribution was estimated using the Kaplan-Meier method, and Kaplan-Meier medians and 95% confidence intervals of the medians were presented for each treatment group. OS analysis was performed by CD8 subgroups with the median of baseline CD8 expression as cut-off.

Time frame:
up to approximately 4.3 years
Reported as:
Median · Months
Overall Survival (OS) in CD8 Subgroups
MonthsCanakinumabPlacebo
CD8 < median46.95 (32.23 to NA)NA (NA to NA)
CD8 ≥ median51.12 (NA to NA)NA (NA to NA)
SecondaryLung Cancer Specific Survival (LCSS)

LCSS is defined as the time from date of randomization to the date of death due to lung cancer. The LCSS distribution was estimated using the Kaplan-Meier method, and Kaplan-Meier medians and 95% confidence intervals of the medians were presented for each treatment group.

Time frame:
Up to approximately 4.3 years
Reported as:
Median · Months
Lung Cancer Specific Survival (LCSS)
MonthsCanakinumabPlacebo
Lung Cancer Specific Survival (LCSS)51.12 (44.71 to NA)NA (NA to NA)
SecondaryDisease Free Survival (DFS) by Local Investigator in PD-L1 Subgroups

DFS is the time from the date of randomization to the date of the first documented NSCLC disease recurrence as assessed by local investigator radiologically or death due to any cause. Disease recurrence included diagnoses of new primary lung malignancies. Clinical deterioration was not considered as a recurrence of disease. In case of non-conclusive radiological evidence, a biopsy assessment was performed to confirm NSCLC recurrence. The median DFS was estimated using the Kaplan-Meier method. DFS was censored if no DFS event was observed prior to the analysis cut-off date or subjects who received any subsequent anti-neoplastic therapy for NSCLC. The censoring date was the date of last assessment before the cut-off date or NSCLC related anti-neoplastic therapy date. DFS analysis was performed by baseline programmed cell death-ligand 1 (PD-L1) expression status: PD-L1 \<1%, PD-L1 ≥1% and \<49%, and PD-L1 ≥50%.

Time frame:
Up to approximately 4 years
Reported as:
Median · Months
Disease Free Survival (DFS) by Local Investigator in PD-L1 Subgroups
MonthsCanakinumabPlacebo
PD-L1 <1%30.72 (23.52 to NA)NA (23.03 to NA)
PD-L1 ≥1% and <49%30.42 (21.42 to NA)NA (17.05 to NA)
PD-L1 ≥50%46.95 (19.45 to NA)NA (22.31 to NA)
Statistical analysis
  • Canakinumab vs Placebo · Stratified log-rank test · p = 0.676 (1-sided p-value) · Hazard ratio (hr): 1.09 · 95% CI 0.76 to 1.58The HR for DFS was calculated, along with its 95% confidence interval, from a stratified Cox model using the same stratification factors as those for the log-rank test
  • Canakinumab vs Placebo · Stratified log-rank test · p = 0.036 (1-sided p-value) · Hazard ratio (hr): 0.60 · 95% CI 0.34 to 1.05The HR for DFS was calculated, along with its 95% confidence interval, from a stratified Cox model using the same stratification factors as those for the log-rank test
  • Canakinumab vs Placebo · Stratified log-rank test · p = 0.823 (1-sided p-value) · Hazard ratio (hr): 1.33 · 95% CI 0.73 to 2.43The HR for DFS was calculated, along with its 95% confidence interval, from a stratified Cox model using the same stratification factors as those for the log-rank test
SecondaryDisease Free Survival (DFS) by Local Investigator in CD8 Subgroups

DFS is the time from the date of randomization to the date of the first documented NSCLC disease recurrence as assessed by local investigator radiologically or death due to any cause. Disease recurrence included diagnoses of new primary lung malignancies. Clinical deterioration was not considered as a recurrence of disease. In case of non-conclusive radiological evidence, a biopsy assessment was performed to confirm NSCLC recurrence. The median DFS was estimated using the Kaplan-Meier method. DFS was censored if no DFS event was observed prior to the analysis cut-off date or subjects who received any subsequent anti-neoplastic therapy for NSCLC. The censoring date was the date of last assessment before the cut-off date or NSCLC related anti-neoplastic therapy date. DFS analysis was performed by CD8 subgroups with the median of baseline CD8 expression as cut-off.

Time frame:
Up to approximately 4 years
Reported as:
Median · Months
Disease Free Survival (DFS) by Local Investigator in CD8 Subgroups
MonthsCanakinumabPlacebo
CD8 < median26.58 (20.67 to NA)NA (25.03 to NA)
CD8 ≥ median46.95 (28.81 to NA)NA (23.89 to NA)
Statistical analysis
  • Canakinumab vs Placebo · Stratified log-rank test · p = 0.872 (1-sided p-value) · Hazard ratio (hr): 1.22 · 95% CI 0.87 to 1.72The HR for DFS was calculated, along with its 95% confidence interval, from a stratified Cox model using the same stratification factors as those for the log-rank test.
  • Canakinumab vs Placebo · Stratified log-rank test · p = 0.303 (1-sided p-value) · Hazard ratio (hr): 0.91 · 95% CI 0.62 to 1.33The HR for DFS was calculated, along with its 95% confidence interval, from a stratified Cox model using the same stratification factors as those for the log-rank test.
SecondaryCanakinumab Serum Concentrations

Serum concentrations of canakinumab were determinded using an ELISA method.

Time frame:
Cycle 1 on day 1 (pre-dose), day 8 and 15; Cycle 2, 4, 6, 9 and 12 on day 1 (pre-dose). Cycle=21 days
Reported as:
Mean · ug/ml
Canakinumab Serum Concentrations
ug/mlCanakinumab
Cycle 1 Day 10 ± 0
Cycle 1 Day 818.1 ± 6.53
Cycle 1 Day 1516.9 ± 5.43
Cycle 2 Day 115.0 ± 4.91
Cycle 4 Day 129.7 ± 10.3
Cycle 6 Day 134.7 ± 13.0
Cycle 9 Day 137.1 ± 14.5
Cycle 12 Day 138.6 ± 15.5
SecondaryCanakinumab Anti-drug Antibody (ADA) Prevalence at Baseline

Canakinumab ADA prevalence at baseline was calculated as the percentage of participants who had an ADA positive result at baseline

Time frame:
Baseline
Reported as:
Count of participants · Participants
Canakinumab Anti-drug Antibody (ADA) Prevalence at Baseline
ParticipantsCanakinumab
Canakinumab Anti-drug Antibody (ADA) Prevalence at Baseline8
SecondaryCanakinumab ADA Incidence

Canakinumab ADA incidence on-treatment was calculated as the percentage of participants who were treatment-induced ADA positive (post-baseline ADA positive with ADA-negative sample at baseline) and treatment-boosted ADA positive (post-baseline ADA positive with titer that was at least the fold titer change greater than the ADA-positive baseline titer)

Time frame:
From baseline up to 130 days after end of treatment, assessed up to approx. 1.5 years
Reported as:
Count of participants · Participants
Canakinumab ADA Incidence
ParticipantsCanakinumab
Canakinumab ADA Incidence7
SecondaryTime to Definitive 10 Point Deterioration Symptom Scores of Pain,Cough and Dyspnea Per European Organization for Research and Treatment of Cancer Quality of Life (EORTC QLQ)- Lung Cancer (LC) 13 Questionnaire

The Lung Cancer module of the EORTC's quality of life questionnaire (EORTC QLQ-LC13) was used in conjunction with the EORTC QLQ-C30 and provided information on an additional 13 items specifically related to lung cancer. The lung cancer module incorporated one multi-item scale to assess dyspnea, and 9 single items assessing pain, coughing, sore mouth, dysphagia, peripheral neuropathy, alopecia, and hemoptysis. All of the domain scores ranged from 0 to 100. A high score indicated a high level of symptoms. The time to definitive 10 point deterioration symptom scores of pain, cough and dyspnea was defined as the time from the date of randomization to the date of event, which was defined as at least 10 points relative to baseline worsening of the EORTC QLQ-LC13 symptom score with no later change below this threshold or death due to any cause, whichever occurred earlier.

Time frame:
From baseline up to approximately 4 years
Reported as:
Median · Months
Time to Definitive 10 Point Deterioration Symptom Scores of Pain,Cough and Dyspnea Per European Organization for Research and Treatment of Cancer Quality of Life (EORTC QLQ)- Lung Cancer (LC) 13 Questionnaire
MonthsCanakinumabPlacebo
PainNA (35.45 to NA)NA (NA to NA)
CoughNA (35.06 to NA)NA (34.99 to NA)
Dyspnea28.88 (23.10 to 34.96)34.99 (23.13 to NA)
SecondaryTime to Definitive 10 Point Deterioration of Global Health Status/Quality of Life (QoL), Shortness of Breath and Pain Per EORTC QLQ-C30 Questionnaire

The EORTC QLQ-C30 was a questionnaire developed to assess the health-related quality of life of cancer participants. It assessed 15 domains consisting of 5 functional domains (physical, role, emotional, cognitive, social) and 9 symptom domains (fatigue, nausea and vomiting, pain, dyspnea, insomnia, appetite loss, constipation, diarrhea, financial difficulties) and a global health status/QoL scale. All domain scores ranged from 0 to 100. A high score for the functional or global health status scales indicated a high level of functioning or QoL; a high score for a symptom scale indicated a high level of symptoms. The time to definitive 10 point deterioration of global health status/QoL, shortness of breath and pain was defined as the time from the date of randomization to the date of event, which was defined as at least 10 points relative to baseline worsening of the EORTC QLQ-C30 score with no later change below this threshold or death due to any cause, whichever occured earlier.

Time frame:
From baseline up to approximately 4 years
Reported as:
Median · Months
Time to Definitive 10 Point Deterioration of Global Health Status/Quality of Life (QoL), Shortness of Breath and Pain Per EORTC QLQ-C30 Questionnaire
MonthsCanakinumabPlacebo
Global health status/QoL34.99 (29.93 to NA)35.15 (35.15 to NA)
Shortness of breathNA (NA to NA)35.15 (34.99 to NA)
Pain29.93 (28.29 to 35.22)36.44 (34.99 to NA)
SecondaryTime to First 10 Point Deterioration for Symptom Scores of Pain, Cough and Dyspnea Per EORTC QLQ-LC13 Questionnaire

The Lung Cancer module of the EORTC's quality of life questionnaire (EORTC QLQ-LC13) was used in conjunction with the EORTC QLQ-C30 and provided information on an additional 13 items specifically related to lung cancer. The lung cancer module incorporated one multi-item scale to assess dyspnea, and 9 single items assessing pain, coughing, sore mouth, dysphagia, peripheral neuropathy, alopecia, and hemoptysis. All of the domain scores ranged from 0 to 100. A high score indicated a high level of symptoms. The time to first 10 point deterioration symptom scores of pain, cough and dyspnea was defined as the time from the date of randomization to the first onset of at least 10 points absolute increase from baseline (worsening) in symptoms scores or death due to any cause, whichever occurred earlier.

Time frame:
From baseline up to approximately 4 years
Reported as:
Median · Months
Time to First 10 Point Deterioration for Symptom Scores of Pain, Cough and Dyspnea Per EORTC QLQ-LC13 Questionnaire
MonthsCanakinumabPlacebo
Pain35.15 (26.58 to NA)NA (23.06 to NA)
Cough15.44 (10.38 to 23.06)15.01 (9.69 to NA)
Dyspnea4.17 (3.42 to 5.55)4.86 (3.48 to 6.97)
SecondaryTime to First 10 Point Deterioration of Global Health Status/QoL, Shortness of Breath and Pain Per EORTC QLQ-C30 Questionnaire

The EORTC QLQ-C30 was a questionnaire developed to assess the health-related quality of life of cancer participants. It assessed 15 domains consisting of 5 functional domains (physical, role, emotional, cognitive, social) and 9 symptom domains (fatigue, nausea and vomiting, pain, dyspnea, insomnia, appetite loss, constipation, diarrhea, financial difficulties) and a global health status/QoL scale. All domain scores ranged from 0 to 100. A high score for the functional or global health status scales indicated a high level of functioning or QoL; a high score for a symptom scale indicated a high level of symptoms. The time to first 10 point deterioration of global health status/QoL, shortness of breath and pain scores was defined as the time from the date of randomization to the first onset of at least 10 points absolute increase from baseline (worsening) in symptoms scores or death due to any cause, whichever occurred earlier.

Time frame:
From baseline up to approximately 4 years
Reported as:
Median · Months
Time to First 10 Point Deterioration of Global Health Status/QoL, Shortness of Breath and Pain Per EORTC QLQ-C30 Questionnaire
MonthsCanakinumabPlacebo
Global health status/QoL9.23 (7.10 to 11.76)9.07 (7.62 to 11.76)
Shortness of breath29.14 (23.03 to NA)NA (23.13 to NA)
Pain5.49 (4.21 to 6.90)5.62 (4.17 to 7.62)
SecondaryChange From Baseline in the Utility Score of the EuroQoL- 5 Dimension- 5 Level (EQ-5D-5L)

EQ-5D-5L was a standardized questionnaire that measured health-related QoL. EQ-5D-5L consisted of 2 components: a health state profile and a visual analogue scale. The health state profile included five dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression, each with five levels ranging from 1 (no problems) to 5 (extreme problems). The EQ-5D-5L health state profile responses were converted into single index utility score, ranging from -1 to 1, where lower scores representing a higher level of dysfunction. Published weights are available enabling the calculation of the utility score. A positive change from baseline indicated improvement. This endpoint was assessed throughout the study, including safety and efficacy follow-up (FU) visits. Safety FU visits: every 4 weeks after end of treatment up to 130 days post-last dose. Efficacy FU visits: at 18, 24, 30, 36 and 48 months post-randomization (if no recurrence observed during treatment or safety FU)

Time frame:
Baseline, every 3 weeks for 14 months; end of treatment; every 4 weeks up to 130 days post-treatment; at 18,24,30,36 and 48 months post-randomization (if no recurrence); 7 and 28 days post-disease progression, up to approx. 4 years.
Reported as:
Mean · Score on a scale
Change From Baseline in the Utility Score of the EuroQoL- 5 Dimension- 5 Level (EQ-5D-5L)
Score on a scaleCanakinumabPlacebo
Week 30.0 ± 0.110.0 ± 0.12
Week 60.0 ± 0.130.0 ± 0.12
Week 90.0 ± 0.130.0 ± 0.13
Week 120.0 ± 0.120.0 ± 0.12
Week 150.0 ± 0.130.0 ± 0.12
Week 180.0 ± 0.130.0 ± 0.13
Week 210.0 ± 0.130.0 ± 0.15
Week 240.0 ± 0.140.0 ± 0.14
Week 270.0 ± 0.130.0 ± 0.14
Week 300.0 ± 0.130.0 ± 0.14
Week 330.0 ± 0.130.0 ± 0.14
Week 360.0 ± 0.140.0 ± 0.15
Week 390.0 ± 0.150.0 ± 0.13
Week 420.0 ± 0.150.0 ± 0.14
Week 450.0 ± 0.140.0 ± 0.14
Week 480.0 ± 0.140.0 ± 0.15
Week 510.0 ± 0.130.0 ± 0.14
Week 540.0 ± 0.180.1 ± 0.12
Week 570.0-0.1
Week 60-0.2-0.1
Week 630.0—
Week 690.0—
Safety FU 10.0 ± 0.150.0 ± 0.14
Safety FU 20.0 ± 0.140.0 ± 0.14
Safety FU 30.0 ± 0.140.0 ± 0.15
Safety FU 40.0 ± 0.150.0 ± 0.16
Safety FU 50.0 ± 0.150.0 ± 0.14
Efficacy FU 10.0 ± 0.160.0 ± 0.14
Efficacy FU 20.0 ± 0.150.0 ± 0.16
Efficacy FU 30.0 ± 0.140.0 ± 0.15
Efficacy FU 40.0 ± 0.130.0 ± 0.15
Efficacy FU 50.0 ± 0.10-0.1 ± 0.17
7 days post disease progression-0.1 ± 0.16-0.1 ± 0.22
28 days post disease progression-0.1 ± 0.18-0.1 ± 0.18
Post-hocAll Collected Deaths

Pre-treatment deaths were collected from day of participant's informed consent to the day before first dose of study medication. On-treatment deaths were collected from start of treatment to 130 days after last dose. Post-treatment follow-up deaths were collected from day 131 after last dose of study treatment to end of study.

Time frame:
Pre-treatment: Up to 28 days prior to treatment. On-treatment: Up to approx. 1.5 years. Post-treatment follow-up: Up to approx. 4.3 years
Reported as:
Number · Participants
All Collected Deaths
ParticipantsCanakinumabPlacebo
Pre-treatment deaths00
On-treatment deaths917
Post-treatment follow-up deaths5351
All deaths6268

Adverse events

Collected over Pre-treatment: from study consent to the day before first dose, up to 28 days. On-treatment: from first dose of study treatment to 130 days after last dose of study medication, up to approx. 1.5 years. Post-treatment follow-up: from 131 days after last dose of study medication until the end of the study, up to approx. 4.3 years.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
All Participants - Pre-treatment0/1,382 (0%)——
Canakinumab - On-treatment9/692 (1.3%)141/692 (20.4%)465/692 (67.2%)
Canakinumab - Post-treatment Follow-up53/671 (7.9%)——
Placebo - On-treatment17/689 (2.5%)146/689 (21.2%)455/689 (66%)
Placebo - Post-treatment Follow-up51/662 (7.7%)——
Most frequent serious events
Showing 10 of 154
Most frequent serious events
EventAll Participants - Pre-treatmentCanakinumab - On-treatmentCanakinumab - Post-treatment Follow-upPlacebo - On-treatmentPlacebo - Post-treatment Follow-up
COVID-19Infections and infestations—48/692—48/689—
PneumoniaInfections and infestations—13/692—9/689—
DyspnoeaRespiratory, thoracic and mediastinal disorders—7/692—2/689—
Myocardial infarctionCardiac disorders—1/692—4/689—
COVID-19 pneumoniaInfections and infestations—1/692—4/689—
Cerebrovascular accidentNervous system disorders—4/692—4/689—
CholecystitisHepatobiliary disorders—1/692—3/689—
C-reactive protein increasedInvestigations—0/692—3/689—
Intervertebral disc protrusionMusculoskeletal and connective tissue disorders—0/692—3/689—
Chronic obstructive pulmonary diseaseRespiratory, thoracic and mediastinal disorders—1/692—3/689—
Most frequent other events
Showing 10 of 24
Most frequent other events
EventAll Participants - Pre-treatmentCanakinumab - On-treatmentCanakinumab - Post-treatment Follow-upPlacebo - On-treatmentPlacebo - Post-treatment Follow-up
CoughRespiratory, thoracic and mediastinal disorders—89/692—108/689—
ArthralgiaMusculoskeletal and connective tissue disorders—74/692—88/689—
FatigueGeneral disorders—70/692—60/689—
DyspnoeaRespiratory, thoracic and mediastinal disorders—67/692—50/689—
Alanine aminotransferase increasedInvestigations—65/692—49/689—
Weight increasedInvestigations—63/692—48/689—
Back painMusculoskeletal and connective tissue disorders—61/692—56/689—
HeadacheNervous system disorders—31/692—60/689—
DiarrhoeaGastrointestinal disorders—57/692—48/689—
Aspartate aminotransferase increasedInvestigations—53/692—37/689—

Baseline characteristics

Age, Continuous
Age, Continuous(Years)CanakinumabPlaceboTotal
Mean61.5 ± 8.9061.6 ± 9.0061.6 ± 8.95
Sex: Female, Male
Sex: Female, Male(Participants)CanakinumabPlaceboTotal
Female263257520
Male430432862
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)CanakinumabPlaceboTotal
White393391784
Asian248236484
Black or African American347
American Indian or Alaska Native055
Multiple011
Missing4952101
07

Study locations

283 sites
  • Highlands Oncology Group .
    Fayetteville, Arkansas 72703, United States
  • Cancer and Blood Specialty Clinic
    Los Alamitos, California 90720, United States
  • University of California at Los Angeles
    Los Angeles, California 90095, United States
  • VA Palo Alto Health Care System CRLX030A2301
    Palo Alto, California 94304-1207, United States
  • Sansum Clinic
    Santa Barbara, California 93105, United States
  • Rocky Mountain Cancer Centers Denver-Mdtn(Bone&MarrowTransp)
    Longmont, Colorado 80501, United States
  • Florida Cancer Specialists
    Fort Myers, Florida 33901, United States
  • Advanced Medical Specialties Drug Ship - 2
    Miami, Florida 33176, United States
  • Florida Cancer Affiliates of Ocala
    Ocala, Florida 34474, United States
  • Florida Cancer Specialists North
    Saint Petersburg, Florida 33705, United States
  • Florida Cancer Specialists
    West Palm Beach, Florida 33401, United States
  • Rush University Medical Center Regulatory
    Chicago, Illinois 60612, United States
  • Cancer Center of Kansas Dept.ofCancerCtr.ofKansas
    Wichita, Kansas 67214-3728, United States
  • VA Nebraska-W IA Health Care System .
    Omaha, Nebraska 68105, United States
  • Louis Stokes Cleveland Department of Veterans Affairs MC .
    Cleveland, Ohio 44106, United States
  • Oncology Associates of Oregon, PC
    Eugene, Oregon 97401-8122, United States
  • Chattanooga Oncology and Hematology Associates PC Chattanooga Oncology
    Chattanooga, Tennessee 37404, United States
  • Sarah Cannon Research Institute
    Nashville, Tennessee 37203, United States
  • Texas Oncology MamieMcFaddenWardCtr
    Dallas, Texas 75246, United States
  • Virginia Cancer Specialists Fairfax Northern Virginia
    Fairfax, Virginia 22031, United States
  • Oncology and Hematology Associates of Southwest Virginia Inc .
    Salem, Virginia 24153, United States
  • Novartis Investigative Site
    Caba, Buenos Aires C1426ANZ, Argentina
  • Novartis Investigative Site
    Mar del Plata, Buenos Aires B7600FZN, Argentina
  • Novartis Investigative Site
    Rosario, Santa Fe S2000DSV, Argentina
  • Novartis Investigative Site
    Rosario, Sante Fe S200KZE, Argentina
  • Novartis Investigative Site
    Cordoba, X5016KEH, Argentina
  • Novartis Investigative Site
    Graz, 8036, Austria
  • Novartis Investigative Site
    Klagenfurt, 9020, Austria
  • Novartis Investigative Site
    Krems, A-3500, Austria
  • Novartis Investigative Site
    Vienna, A 1090, Austria
  • Novartis Investigative Site
    Teresina, Piaui 64049-200, Brazil
  • Novartis Investigative Site
    Londrina, PR 86015-520, Brazil
  • Novartis Investigative Site
    Barretos, SP 14784 400, Brazil
  • Novartis Investigative Site
    Sao Paulo, SP 01246 000, Brazil
  • Novartis Investigative Site
    Sao Paulo, SP 04014-002, Brazil
  • Novartis Investigative Site
    Sofia, 1303, Bulgaria
  • Novartis Investigative Site
    Sofia, 1407, Bulgaria
  • Novartis Investigative Site
    Sofia, 1756, Bulgaria
  • Novartis Investigative Site
    Sofia, 1784, Bulgaria
  • Novartis Investigative Site
    Edmonton, Alberta T6G 1Z2, Canada
  • Novartis Investigative Site
    Moncton, New Brunswick E1C 6Z8, Canada
  • Novartis Investigative Site
    Halifax, Nova Scotia B3H 1V7, Canada
  • Novartis Investigative Site
    Montreal, Quebec H2W 1T8, Canada
  • Novartis Investigative Site
    Quebec, GIV 4G5, Canada
  • Novartis Investigative Site
    Santiago, 7500006, Chile
  • Novartis Investigative Site
    Santiago, 7500921, Chile
  • Novartis Investigative Site
    Hefei, Anhui 230001, China
  • Novartis Investigative Site
    Beijing, Beijing 100039, China
  • Novartis Investigative Site
    Chongqing, Chongqing 400037, China
  • Novartis Investigative Site
    Guangzhou, Guangdong 510000, China
  • Novartis Investigative Site
    Shenzhen, Guangdong 518020, China
  • Novartis Investigative Site
    Zhanjing, Guangong 524000, China
  • Novartis Investigative Site
    Zunyi, Guizhou 563000, China
  • Novartis Investigative Site
    Zhengzhou, Henan 450008, China
  • Novartis Investigative Site
    Nanjing, Jiangsu 210008, China
  • Novartis Investigative Site
    Suzhou, Jiangsu 215004, China
  • Novartis Investigative Site
    Chang Chun, Jilin 130021, China
  • Novartis Investigative Site
    Shenyang, Liaoning 110011, China
  • Novartis Investigative Site
    Jinan, Shandong 250117, China
  • Novartis Investigative Site
    Shanghai, Shanghai 200032, China
  • Novartis Investigative Site
    Shanghai, Shanghai 200433, China
  • Novartis Investigative Site
    XI An, Shanxi 710061, China
  • Novartis Investigative Site
    Shenyang, Shengyang 110041, China
  • Novartis Investigative Site
    Chengdu, Sichuan 610041, China
  • Novartis Investigative Site
    Hangzhou, Zhejiang 310003, China
  • Novartis Investigative Site
    Hangzhou, Zhejiang 310022, China
  • Novartis Investigative Site
    Beijing, 100036, China
  • Novartis Investigative Site
    Changsha, 410013, China
  • Novartis Investigative Site
    Chengdu, 610044, China
  • Novartis Investigative Site
    Fujian, 350001, China
  • Novartis Investigative Site
    Tianjin, 300000, China
  • Novartis Investigative Site
    Tianjin, 300052, China
  • Novartis Investigative Site
    Bogota, 110221, Colombia
  • Novartis Investigative Site
    Ostrava Vitkovice, 703 84, Czechia
  • Novartis Investigative Site
    Prague 2, 128 21, Czechia
  • Novartis Investigative Site
    Marseille cedex 20, Bouches Du Rhone 13915, France
  • Novartis Investigative Site
    Le Mans, Cedex 09 72037, France
  • Novartis Investigative Site
    Montpellier cedex 5, Herault 34059, France
  • Novartis Investigative Site
    Amiens, 80054, France
  • Novartis Investigative Site
    Angers Cedex 9, 49933, France
  • Novartis Investigative Site
    Avignon Cedex, 84902, France
  • Novartis Investigative Site
    Bayonne, 64109, France
  • Novartis Investigative Site
    Brest, 29200, France
  • Novartis Investigative Site
    Bron, 69677, France
  • Novartis Investigative Site
    Clermont-Ferrand, 63000, France
  • Novartis Investigative Site
    Creteil, 94000, France
  • Novartis Investigative Site
    La Rochelle, 17019, France
  • Novartis Investigative Site
    La Seyne sur mer, 83500, France
  • Novartis Investigative Site
    Lille, 59000, France
  • Novartis Investigative Site
    Paris, 75014, France
  • Novartis Investigative Site
    Paris, 75231, France
  • Novartis Investigative Site
    Pessac Cedex, 33604, France
  • Novartis Investigative Site
    Poitiers, 86000, France
  • Novartis Investigative Site
    Saint-Herblain Cédex, 44805, France
  • Novartis Investigative Site
    Strasbourg Cedex, 67091, France
  • Novartis Investigative Site
    Suresnes, 92150, France
  • Novartis Investigative Site
    Toulouse, 31400, France
  • Novartis Investigative Site
    Batumi, 6000, Georgia
  • Novartis Investigative Site
    Tbilisi, 0144, Georgia
  • Novartis Investigative Site
    Tbilisi, 0159, Georgia

Showing the first 100 of 283 sites across 41 countries.

08

References and documents

Study documents

  • Study protocol · Feb 3, 2022
  • Statistical analysis plan · Mar 3, 2023

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data will be available according to the process described on www.clinicalstudydatarequest.com.

09

Registry details

Key details

Study ID
NCT03447769
Lead sponsor
Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
Feb 27, 2018
Start date
Mar 16, 2018
Primary completion
Mar 17, 2022
Completion
Feb 7, 2023
Results posted
Jan 30, 2024
Last update
Oct 9, 2024

Study contacts

Novartis Pharmaceuticals
study director · Novartis Pharmaceuticals

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.

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