Trial Outcomes & Findings for Study Evaluating Efficacy and Safety of Capmatinib in Combination With Osimertinib in Adult Subjects With Non-small Cell Lung Cancers as Second Line Therapy (NCT NCT04816214)

NCT ID: NCT04816214

Last Updated: 2024-03-05

Results Overview

A DLT was defined as an adverse event (AE) or abnormal laboratory value assessed as unrelated to disease, progressive disease, inter-current illness, or concomitant medications, that despite optimal therapeutic intervention that occurred within the first 21 days of treatment with capmatinib in combination with osimertinib.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

6 participants

Primary outcome timeframe

Up to 21 Days

Results posted on

2024-03-05

Participant Flow

In the run-in part, participants were enrolled at 4 investigative sites in 3 countries. The study was terminated early based on Sponsor's decision unrelated to any safety concerns and the randomized part of the study was not initiated.

A total of 23 participants were screened of which 6 participants were enrolled into the run-in part of the study.

Participant milestones

Participant milestones
Measure
Run-in Part: Capmatinib + Osimertinib
Participants received a starting dose of capmatinib 400 mg, orally, twice daily (BID) in combination with osimertinib 80 mg, orally, once daily (QD)
Randomized Part: Capmatinib + Osimertinib
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Overall Study
STARTED
6
0
0
Overall Study
Full Analysis Set (FAS)
6
0
0
Overall Study
Safety Set
6
0
0
Overall Study
Pharmacokinetic (PK) Analysis Set
6
0
0
Overall Study
COMPLETED
0
0
0
Overall Study
NOT COMPLETED
6
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Run-in Part: Capmatinib + Osimertinib
Participants received a starting dose of capmatinib 400 mg, orally, twice daily (BID) in combination with osimertinib 80 mg, orally, once daily (QD)
Randomized Part: Capmatinib + Osimertinib
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Overall Study
Progressive Disease
4
0
0
Overall Study
Death
1
0
0
Overall Study
Study Terminated by Sponsor
1
0
0

Baseline Characteristics

Study Evaluating Efficacy and Safety of Capmatinib in Combination With Osimertinib in Adult Subjects With Non-small Cell Lung Cancers as Second Line Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Run-in Part: Capmatinib + Osimertinib
n=6 Participants
Participants received a starting dose of capmatinib 400 mg, orally, twice daily (BID) in combination with osimertinib 80 mg, orally, once daily (QD)
Age, Continuous
61.7 years
STANDARD_DEVIATION 7.76 • n=5 Participants
Sex: Female, Male
Female
4 Participants
n=5 Participants
Sex: Female, Male
Male
2 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
6 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
0 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Up to 21 Days

Population: FAS included all participants who received any component of the study treatment.

A DLT was defined as an adverse event (AE) or abnormal laboratory value assessed as unrelated to disease, progressive disease, inter-current illness, or concomitant medications, that despite optimal therapeutic intervention that occurred within the first 21 days of treatment with capmatinib in combination with osimertinib.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Number of Participants With Dose Limiting Toxicities (DLTs)
0 Participants

PRIMARY outcome

Timeframe: From randomization to first documented progression or deaths, planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not initiated. Hence the data was not collected for the outcome measures in the randomized part of the study.

PFS was defined as time from date of randomization to the date of first documented disease progression (PD) based on Blinded Independent Review Committee (BIRC) as per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), or death from any cause, whichever occurred first. Progression was defined as a ≥20% increase in sum of longest diameters (SLD) compared to smallest SLD in the study, or progression of non-target lesions or new lesions.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From the first dose until last dose of study treatment, assessed up to 39 weeks

Population: Safety set included all participants who received at least one dose of study treatment.

Number of participants with at least one dose interruption and dose reduction were reported for each study drug.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Number of Participants With at Least One Dose Interruption and Dose Reduction of Each Study Drug
Dose Interruption: Capmatinib
5 Participants
Run-in Part: Number of Participants With at Least One Dose Interruption and Dose Reduction of Each Study Drug
Dose Interruption: Osimertinib
4 Participants
Run-in Part: Number of Participants With at Least One Dose Interruption and Dose Reduction of Each Study Drug
Dose Reduction: Capmatinib
3 Participants
Run-in Part: Number of Participants With at Least One Dose Interruption and Dose Reduction of Each Study Drug
Dose Reduction: Osimertinib
0 Participants

SECONDARY outcome

Timeframe: From the first dose until last dose of study treatment, assessed up to 39 weeks

Population: Safety set included all participants who received at least one dose of study treatment.

Dose intensity was computed as the ratio of actual cumulative dose (milligrams) received and actual duration of exposure (weeks) to study drug.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Dose Intensity of Each Study Drug
Capmatinib
5042.8 milligrams per week (mg/week)
Standard Deviation 947.28
Run-in Part: Dose Intensity of Each Study Drug
Osimertinib
534.9 milligrams per week (mg/week)
Standard Deviation 41.29

SECONDARY outcome

Timeframe: From the first dose until last dose of study treatment, assessed up to 39 weeks

Population: Safety set included all participants who received at least one dose of study treatment.

Duration of exposure is defined as the time (in weeks) between the first and the last dose of study treatment.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Median Duration of Exposure to Each Study Drug
Capmatinib
23.5 weeks
Interval 1.0 to 39.0
Run-in Part: Median Duration of Exposure to Each Study Drug
Osimertinib
24.0 weeks
Interval 1.0 to 39.0

SECONDARY outcome

Timeframe: Pre-dose, 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose on Days 1 and 15 of Cycle 1 (Cycle = 21 days)

Population: PK analysis set included all participants who provided at least one evaluable PK concentration. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint.

Blood samples were collected. Cmax of capmatinib was calculated from plasma concentration-time data using non-compartmental methods and summarized using descriptive statistics. Actual recorded sampling times were considered for the calculations

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Maximum Plasma Concentration (Cmax) of Capmatinib
Day 1
5510 nanograms per milliliter (ng/mL)
Standard Deviation 2380
Run-in Part: Maximum Plasma Concentration (Cmax) of Capmatinib
Day 15
5750 nanograms per milliliter (ng/mL)
Standard Deviation 1050

SECONDARY outcome

Timeframe: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8 and 24 hours post-dose on Days 1 and 15 of Cycle 1 (Cycle = 21 days)

Population: PK analysis set included all participants who provided at least one evaluable PK concentration. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available at specified timepoint.

Blood samples were collected. Cmax of osimertinib and its metabolites (AZ5104 and AZ7550) was calculated from plasma concentration-time data using non-compartmental methods and summarized using descriptive statistics.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=5 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Maximum Plasma Concentration (Cmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
Osimertinib: Day 1
99.5 ng/mL
Standard Deviation 57.0
Run-in Part: Maximum Plasma Concentration (Cmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
Osimertinib: Day 15
265 ng/mL
Standard Deviation 113
Run-in Part: Maximum Plasma Concentration (Cmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ5104: Day 1
4.86 ng/mL
Standard Deviation 3.22
Run-in Part: Maximum Plasma Concentration (Cmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ5104: Day 15
22.0 ng/mL
Standard Deviation 9.23
Run-in Part: Maximum Plasma Concentration (Cmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ7550: Day 1
3.61 ng/mL
Standard Deviation 1.53
Run-in Part: Maximum Plasma Concentration (Cmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ7550: Day 15
33.0 ng/mL
Standard Deviation 11.9

SECONDARY outcome

Timeframe: Pre-dose, 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose on Days 1 and 15 of Cycle 1 (Cycle = 21 days)

Population: PK analysis set included all participants who provided at least one evaluable PK concentration. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint.

Blood samples were collected. Tmax of capmatinib was calculated from plasma concentration-time data using non-compartmental methods and summarized using descriptive statistics. Actual recorded sampling times were considered for the calculations

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Time to Maximum Plasma Concentration (Tmax) of Capmatinib
Day 1
1.92 hours
Interval 0.983 to 3.02
Run-in Part: Time to Maximum Plasma Concentration (Tmax) of Capmatinib
Day 15
1.00 hours
Interval 0.917 to 1.82

SECONDARY outcome

Timeframe: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8 and 24 hours post-dose on Days 1 and 15 of Cycle 1 (Cycle = 21 days)

Population: PK analysis set included all participants who provided at least one evaluable PK concentration. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available at specified timepoint.

Blood samples were collected. Tmax of osimertinib and its metabolites (AZ5104 and AZ7550) was calculated from plasma concentration-time data using non-compartmental methods and summarized using descriptive statistics. Actual recorded sampling times were considered for the calculations

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=5 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Time to Maximum Plasma Concentration (Tmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ5104: Day 1
23.1 hours
Interval 22.3 to 23.5
Run-in Part: Time to Maximum Plasma Concentration (Tmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ5104: Day 15
4.00 hours
Interval 0.0 to 7.13
Run-in Part: Time to Maximum Plasma Concentration (Tmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
Osimertinib: Day 1
6.00 hours
Interval 2.9 to 23.1
Run-in Part: Time to Maximum Plasma Concentration (Tmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
Osimertinib: Day 15
4.00 hours
Interval 0.0 to 7.33
Run-in Part: Time to Maximum Plasma Concentration (Tmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ7550: Day 1
5.68 hours
Interval 2.9 to 23.1
Run-in Part: Time to Maximum Plasma Concentration (Tmax) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ7550: Day 15
2.25 hours
Interval 0.0 to 6.0

SECONDARY outcome

Timeframe: Pre-dose, 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose on Days 1 and 15 of Cycle 1 (Cycle = 21 days)

Population: PK analysis set included all participants who provided at least one evaluable PK concentration. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint.

Blood samples were collected. AUClast of capmatinib was calculated from plasma concentration-time data using non-compartmental methods and summarized using descriptive statistics.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Area Under the Curve to the Last Measurable Concentration (AUClast) of Capmatinib
Day 1
20100 nanograms*hours/milliliter (ng*hr/mL)
Standard Deviation 6080
Run-in Part: Area Under the Curve to the Last Measurable Concentration (AUClast) of Capmatinib
Day 15
21300 nanograms*hours/milliliter (ng*hr/mL)
Standard Deviation 7050

SECONDARY outcome

Timeframe: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8 and 24 hours post-dose on Days 1 and 15 of Cycle 1 (Cycle = 21 days)

Population: PK analysis set included all participants who provided at least one evaluable PK concentration. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available at specified timepoint.

Blood samples were collected. AUClast of osimertinib and its metabolites (AZ5104 and AZ7550) was calculated from plasma concentration-time data using non-compartmental methods and summarized using descriptive statistics.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=5 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Area Under the Curve to the Last Measurable Concentration (AUClast) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
Osimertinib: Day 15
1380 ng*hr/mL
Standard Deviation 900
Run-in Part: Area Under the Curve to the Last Measurable Concentration (AUClast) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ5104: Day 1
82.9 ng*hr/mL
Standard Deviation 59.4
Run-in Part: Area Under the Curve to the Last Measurable Concentration (AUClast) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ5104: Day 15
136 ng*hr/mL
Standard Deviation 56.8
Run-in Part: Area Under the Curve to the Last Measurable Concentration (AUClast) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
Osimertinib: Day 1
1790 ng*hr/mL
Standard Deviation 1030
Run-in Part: Area Under the Curve to the Last Measurable Concentration (AUClast) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ7550: Day 1
47.1 ng*hr/mL
Standard Deviation 46.3
Run-in Part: Area Under the Curve to the Last Measurable Concentration (AUClast) of Osimertinib and Its Metabolites (AZ5104 and AZ7550)
AZ7550: Day 15
112 ng*hr/mL
Standard Deviation 111

SECONDARY outcome

Timeframe: Up to end of study, assessed up to 39 weeks

Population: FAS included all participants who received any component of the study treatment.

ORR was defined as the percentage of participants with a confirmed best overall response (BOR) of complete response (CR) or partial response (PR), as per investigator judgment and according to RECIST v1.1. Criteria for CR: Disappearance of all lesions and pathologic lymph nodes; PR: ≥30% decrease in the SLD of the target lesions or no new lesions or no progression of non-target lesions.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Overall Response Rate (ORR) as Per Investigator Assessment
50.0 percentage of participants
Interval 11.8 to 88.2

SECONDARY outcome

Timeframe: Up to disease progression or death or end of study, assessed up to 39 weeks

Population: FAS included all participants who received any component of the study treatment. 'Overall number of participants analyzed' indicates the number of participants with CR or PR.

DOR was defined as the time from first documented response of CR or PR to the date of first documented PD or death due to any cause. Criteria for CR: Disappearance of all lesions and pathologic lymph nodes; PR: ≥30% decrease in the SLD of the target lesions or no new lesions or no progression of non-target lesions; PD: ≥20% increase in SLD compared to the smallest SLD in the study, or progression of non-target lesions or new lesions.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=3 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Duration of Response (DOR) as Per Investigator Assessment
6.93 months
Interval 2.5 to
Upper limit of 95% confidence interval (CI) was not estimable due to an insufficient number of participants with events.

SECONDARY outcome

Timeframe: From first dose of treatment up to end of study, assessed up to 39 weeks

Population: FAS included all participants who received any component of the study treatment. 'Overall number of participants analyzed' indicates the number of participants with CR or PR.

TTR was defined as the duration of time between the date of fist dose of treatmwnr and the date of the first documented response of either CR or PR as per investigator judgment and according to RECIST v1.1 criteria. Criteria for CR: Disappearance of all lesions and pathologic lymph nodes; PR: ≥30% decrease in SLD of the target lesions or no new lesions or no progression of non-target lesions.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=3 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Time to Response (TTR) as Per Investigator Assessment
NA months
Interval 1.41 to
Median and the upper limit of 95% CI were not estimable due to an insufficient number of participants with events.

SECONDARY outcome

Timeframe: From randomization up to end of study, assessed up to 39 weeks

Population: FAS included all participants who received any component of the study treatment.

DCR was defined as the percentage of participants with a BOR of CR, PR, and stable disease (SD) as per investigator judgment and according to RECIST v1.1. Criteria for CR: Disappearance of all lesions and pathologic lymph nodes; PR: ≥30% decrease in SLD of the target lesions or no new lesions or no progression of non-target lesions; SD: Neither sufficient shrinkage to qualify for PR or CR nor and increase in lesions which would qualify for PD. PD: ≥20% increase in SLD compared to the smallest SLD in the study, or progression of non-target lesions or new lesions.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Disease Control Rate (DCR) as Per Investigator Assessment
66.7 percentage of participants
Interval 22.3 to 95.7

SECONDARY outcome

Timeframe: From first dose of treatment until first documented progression or death or end of study, assessed up to 39 weeks

Population: FAS included all participants who received any component of the study treatment.

PFS was defined as the time (in months) from first dose of treatment to the date of the first documented progression or death due to any cause as per investigator judgment and according to RECIST v1.1. Progression was defined as a ≥20% increase in SLD compared to smallest SLD in the study, or progression of non-target lesions or new lesions. PFS was censored if no PFS event (progression or death) was observed.

Outcome measures

Outcome measures
Measure
Randomized Part: Capmatinib + Osimertinib
n=6 Participants
In the randomized part, capmatinib in combination with osimertinib was to be administered at the recommended Phase III regimen (defined in the safety run-in part). The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated.
Randomized Part: Platinum + Pemetrexed Based Doublet Chemotherapy
In the randomized part, platinum-pemetrexed based doublet chemotherapy was to follow local guidelines as per standard of care and products labels. Participants randomized to platinum-pemetrexed based doublet chemotherapy arm were to be allowed to crossover to receive capmatinib in combination with osimertinib. The study was terminated early based on Sponsor's decision unrelated to safety concerns and the randomized part of the study was not initiated
Run-in Part: Progression-Free Survival (PFS) as Per Investigator Assessment
4.58 months
Interval 0.99 to
The upper limit of 95% CI was not estimable due to an insufficient number of participants with events.

SECONDARY outcome

Timeframe: Planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

ORR was defined as the percentage of participants with confirmed BOR of CR or PR, as per BIRC by RECIST v1.1. Criteria for CR: Disappearance of all lesions and pathologic lymph nodes; PR: ≥30% decrease in SLD of the target lesions or no new lesions or no progression of non-target lesions.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

OIRR was defined as the percentage of participants with a confirmed best overall intracranial response (BOIR) of CR or PR as per BIRC according to Response Assessment in Neuro-Oncology Brain Metastases (RANO-BM) criteria. Criteria for CR: Disappearance of all CNS target and non-target lesions sustained for at least 4 weeks, with no new lesions, and no use of corticosteroids. PR: ≥30% decrease in the SLD of CNS target lesions or no new lesions or and stable to decreased corticosteroid dose.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From first documented response to first documented progression or death, planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

DOR was defined as the time from the first documented response of CR or PR to the date of first documented progression or death as per BIRC according to RECIST v1.1 criteria. Criteria for CR: Disappearance of all lesions and pathologic lymph nodes; PR: ≥30% decrease in SLD of the target lesions or no new lesions or no progression of non-target lesions; PD: ≥20% increase in SLD compared to the smallest SLD in the study, or progression of non-target lesions or new lesions.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From randomization to first documented response, planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

TTR was defined as duration of time between the date of randomization and the date of first documented response of either CR or PR as per BIRC according to RECIST v1.1 criteria. Criteria for CR: Disappearance of all lesions and pathologic lymph nodes; PR: ≥30% decrease in SLD of the target lesions or no new lesions or no progression of non-target lesions.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

DCR was defined as the percentage of participants with CR or PR or SD as per BIRC according to RECIST v1.1 criteria. Criteria for CR: Disappearance of all lesions and pathologic lymph nodes; PR: ≥30% decrease in SLD of the target lesions or no new lesions or no progression of non-target lesions; SD: Neither sufficient shrinkage to qualify for PR or CR nor and increase in lesions which would qualify for PD. PD: ≥20% increase in SLD compared to the smallest SLD in the study, or progression of non-target lesions or new lesions.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

PFS2 was defined as the time from date of randomization to the first documented progression on the next line therapy as per investigator judgment according to RECIST v1.1 response criteria or death due to any cause.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre-dose on Day 1 of Cycles 1, 2, 3, 4 and 6 (Cycle = 21 days)

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

Cmax of capmatinib calculated from plasma concentration-time data using non-compartmental methods and summarized using descriptive statistics.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre-dose on Day 1 of Cycles 1, 2, 3, 4 and 6 (Cycle = 21 days)

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

Cmax of osimertinib and its metabolites (AZ5104 and AZ7550) calculated from plasma concentration-time data using non-compartmental methods and summarized using descriptive statistics.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From randomization to date of death, planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

OS was defined as the time from date of randomization to date of death due to any cause.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Cycle 1 Day 1, Cycle 3 Day 1, then every 6 weeks for the 18 months after Cycle 1 Day 1 and every 12 weeks thereafter, until end of treatment, end of treatment and 6, 12 and 18 weeks post-progression (Cycle = 21 days)

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

EORTC QLQ-C30 is a questionnaire developed to assess the quality of life of cancer patients. It contains 30 items and is composed of both multi-item scales and single-item measures based on the participant's experience over the past week. These include five scales (physical, role, emotional, cognitive, and social functioning), three symptom scales (fatigue, nausea/vomiting, and pain), six single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial impact) and a global health status/quality of life scale (QoL) scale. All of the scales and single-item measures range in score from 0 to 100. A high score for a functional scale represents a high /healthy level of functioning; a high score for the global health status / QoL represents a high QoL, but a high score for a symptom scale / item represents a high level of symptomatology/problems.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Cycle 1 Day 1, Cycle 3 Day 1, then every 6 weeks for the 18 months after Cycle 1 Day 1 and every 12 weeks thereafter, until end of treatment, end of treatment and 6, 12 and 18 weeks post-progression (Cycle = 21 days)

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

EORTC QLQ-LC13 is used in conjunction with the EORTC QLQ-C30 and provides information on an additional 13 items specifically related to lung cancer. The five domains of the LC13 include pain, dyspnea, coughing and hemoptysis, and are based on their presence over the past week. All but the pain domain are scored on a 4 point Likert scale ranging from "not at all" to "very much". Pain score is based on its presence, hence yes or no. Scores are averaged and transformed to 0 to 100. A higher score indicates a higher presence of symptoms.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Cycle 1 Day 1, Cycle 3 Day 1, then every 6 weeks for the 18 months after Cycle 1 Day 1 and every 12 weeks thereafter, until end of treatment, end of treatment and 6, 12 and 18 weeks post-progression (Cycle = 21 days)

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

EQ-5D-5L is a standardized measure of health status developed by the EuroQol (EQ) Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The EQ-5D-5L consists of 2 pages - the descriptive system and the EQ visual analogue scale (EQ VAS). The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of these dimensions, the participant self-assigned a score: from 1 (no problems) to 5 (extreme problems). The questionnaire also includes a Visual Analogue Scale (VAS), where the participant is asked to rate current health status on a scale of 0 to 100, with 0 being the worst imaginable health state.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Cycle 1 Day 1, Cycle 3 Day 1, then every 6 weeks for the 18 months after Cycle 1 Day 1 and every 12 weeks thereafter, until end of treatment, end of treatment and 6, 12 and 18 weeks post-progression (Cycle = 21 days)

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

The NCCN FACT-Brain Symptom Index version 2.0 (FBrSI) is a questionnaire used to explore changes in symptoms associated with potential brain metastases (BM). The symptoms module contains 12 items with a recall period of the past 7 days. The participant responds to 12 statements and indicates to what extent it applies to them on a 5-point Likert response scale from "not at all"=0 to "very much"=4. Higher scores indicates a greater impact of symptoms on the participant's quality of life.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From baseline to symptom deterioration, planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

Time to symptom deterioration from baseline from baseline for participant scores from the EORTC QLQ-C30 questionnaire. EORTC QLQ-C30 is a questionnaire developed to assess the quality of life of cancer patients. It contains 30 items and is composed of both multi-item scales and single-item measures based on the participant's experience over the past week. These include five scales (physical, role, emotional, cognitive, and social functioning), three symptom scales (fatigue, nausea/vomiting, and pain), six single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial impact) and a global health status/quality of life scale (QoL) scale. All of the scales and single-item measures range in score from 0 to 100. A high score for a functional scale represents a high /healthy level of functioning; a high score for the global health status / QoL represents a high QoL, but a high score for a symptom scale / item represents a high level of symptomatology/problems.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From baseline to symptom deterioration, planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

Time to symptom deterioration from baseline for participant scores from EORTC QLQ-LC13 questionnaire. EORTC QLQ-LC13 is used in conjunction with the EORTC QLQ-C30 and provides information on an additional 13 items specifically related to lung cancer. The five domains of the LC13 include pain, dyspnea, coughing and hemoptysis, and are based on their presence over the past week. All but the pain domain are scored on a 4 point Likert scale ranging from "not at all" to "very much". Pain score is based on its presence, hence yes or no. Scores are averaged and transformed to 0 to 100. A higher score indicates a higher presence of symptoms.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From baseline to symptom deterioration, planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

Time to symptom deterioration from baseline for participant scores from the NCCN FBrSl questionnaire. The NCCN FACT-Brain Symptom Index version 2.0 (FBrSI) is a questionnaire used to explore changes in symptoms associated with potential brain metastases (BM). The symptoms module contains 12 items with a recall period of the past 7 days. The participant responds to 12 statements and indicates to what extent it applies to them on a 5-point Likert response scale from "not at all"=0 to "very much"=4. Higher scores indicates a greater impact of symptoms on the participant's quality of life.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From the date of first documented intracranial response to first documented intracranial progression or death, planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

DOIR was defined as the time from the date of first documented intracranial response of either CR or PR to the date of the first documented intracranial progression per RANO-BM criteria as assessed by BIRC review or the date of death due to any cause. Criteria for CR: Disappearance of all CNS target and non-target lesions sustained for at least 4 weeks, with no new lesions, and no use of corticosteroids; PR: ≥30% decrease in the SLD of CNS target lesions or no new lesions, and stable to decreased corticosteroid dose.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From randomization to first documented response, planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

TTIR was defined as the time from the date of randomization to the date of the first documented intracranial response of either CR or PR as per BIRC according to RANO-BM criteria. Criteria for CR: Disappearance of all CNS target and non-target lesions sustained for at least 4 weeks, with no new lesions, and no use of corticosteroids; PR: ≥30% decrease in the SLD of CNS target lesions or no new lesions, and stable to decreased corticosteroid dose.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Planned to be assessed up to 37 months

Population: Due to early study termination, the randomized part of the study was not conducted. Hence the data was not collected for the outcome measures in the randomized part of the study.

IDCR was defined as the percentage of participants with a confirmed BOIR of CR or PR or SD as per BIRC according to RANO-BM criteria. Criteria for CR: Disappearance of all CNS target and non-target lesions sustained for at least 4 weeks, with no new lesions, and no use of corticosteroids; PR: ≥30% decrease in the SLD of CNS target lesions or no new lesions, and stable to decreased corticosteroid dose; SD: Neither sufficient shrinkage to qualify for partial response nor sufficient increase to qualify for progressive disease.

Outcome measures

Outcome data not reported

Adverse Events

Run-in Part: Capmatinib + Osimertinib

Serious events: 2 serious events
Other events: 5 other events
Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure
Run-in Part: Capmatinib + Osimertinib
n=6 participants at risk
Participants received a starting dose of capmatinib 400 mg, orally, twice daily (BID) in combination with osimertinib 80 mg, orally, once daily (QD)
Cardiac disorders
Pericardial effusion
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Gastrointestinal disorders
Nausea
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Infections and infestations
COVID-19
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Infections and infestations
Pneumonia
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Metabolism and nutrition disorders
Decreased appetite
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.

Other adverse events

Other adverse events
Measure
Run-in Part: Capmatinib + Osimertinib
n=6 participants at risk
Participants received a starting dose of capmatinib 400 mg, orally, twice daily (BID) in combination with osimertinib 80 mg, orally, once daily (QD)
Blood and lymphatic system disorders
Neutropenia
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Cardiac disorders
Pericardial effusion
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Ear and labyrinth disorders
Ear discomfort
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Eye disorders
Cataract
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Gastrointestinal disorders
Diarrhoea
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Gastrointestinal disorders
Nausea
33.3%
2/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Gastrointestinal disorders
Vomiting
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
General disorders
Chills
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
General disorders
Fatigue
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
General disorders
Oedema peripheral
66.7%
4/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
General disorders
Peripheral swelling
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
General disorders
Pyrexia
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Infections and infestations
COVID-19
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Infections and infestations
Paronychia
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Investigations
Amylase increased
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Investigations
Blood creatinine increased
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Investigations
Lipase increased
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Metabolism and nutrition disorders
Decreased appetite
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Metabolism and nutrition disorders
Hypoalbuminaemia
50.0%
3/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Musculoskeletal and connective tissue disorders
Back pain
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Nervous system disorders
Headache
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Respiratory, thoracic and mediastinal disorders
Pleural effusion
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Skin and subcutaneous tissue disorders
Dry skin
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Skin and subcutaneous tissue disorders
Pruritus
16.7%
1/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.
Skin and subcutaneous tissue disorders
Rash
50.0%
3/6 • From first dose of treatment until 30 days after last dose of study treatment, assessed up to 39 weeks.
Safety set included all participants who received at least one dose of study treatment. Due to early study termination, the randomized part of the study was not conducted. Hence safety data was not collected in the randomized part of the study.

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: +1 862 778 8300

Results disclosure agreements

  • Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
  • Publication restrictions are in place

Restriction type: OTHER