Trial Outcomes & Findings for Comparative Effectiveness of Targeted Therapies in BRAF Positive Metastatic Melanoma in the US (NCT NCT05260684)

NCT ID: NCT05260684

Last Updated: 2025-03-06

Results Overview

As per COLUMBUS trial, OS was defined as the time from the date of randomization to the date of death due to any cause; if death was not observed, participants were censored at the date of last contact or the data analysis cut-off date, whichever occurred first. As per Flatiron EHR, OS was defined as the time from the index date to the date of death; participants without a date of death were censored at their last known activity date or the end of the follow-up period, whichever occurred first. Index date in each treatment group was defined as the date of treatment initiation. Kaplan-Meier analyses was used for analysis.

Recruitment status

COMPLETED

Target enrollment

716 participants

Primary outcome timeframe

COLUMBUS: From date of randomization until death or censoring (approx 80.5 months); Flatiron: From index date until death due or censoring or end of follow-up period (92.7 months)

Results posted on

2025-03-06

Participant Flow

Data of eligible participants with v-Raf murine sarcoma viral oncogene homolog B protein (BRAF)V-600 mutant melanoma who aged greater than or equal to 18 years at the time of initiating treatment with encorafenib plus binimetinib (ENCO+BINI) or dabrafenib plus trametinib (DAB+TRAM) or vemurafenib plus cobimetinib (VEM+COBI) was collected retrospectively.

Data sources: COLUMBUS trial from 30-Dec-2013-15-Sept-2020 \[approximately 80.5 months\] and Flatiron Health electronic health Records (EHR) real-world database (RWD) from duration of 09-Jan-2014-30-Sep-2021 \[92.7 months\]. Planned cohorts in this study: 1) ENCO+BINI, pooled across COLUMBUS and Flatiron EHR; 2) DAB+TRAM: from Flatiron EHR; 3) VEM+COBI: from Flatiron EHR. Available retrospective data was evaluated in this observational study from 17-Jan-2022-31-Dec-2023 (approximately 23.5 months).

Participant milestones

Participant milestones
Measure
Encorafenib+Binimetinib
Participants with BRAFV600-mutant metastatic melanoma, who were enrolled between 30-December 2013 to 15-September-2020 in phase 3 COLUMBUS trial and eligible participants from RWD cohort identified from Flatiron Health Database between 27-June-2018 to 30-September- 2021 and treated with Encorafenib 450 mg once daily (QD) and Binimetinib 45 mg twice daily (BID)(ENCO+BINI) were included.
Dabrafenib+Trametinib
Eligible participants from RWD cohort with BRAFV600-mutant metastatic melanoma identified from Flatiron Health Database between 09-January-2014 to 30-September-2021 and treated with Dabrafenib 150 mg BID+ Trametinib 2 mg QD (DAB+TRAM) were included.
Vemurafenib + Cobimetinib
Eligible participants from RWD cohort with BRAFV600-mutant metastatic melanoma identified from Flatiron Health Database between 10-November-2015 to 30-September-2021 and treated with Vemurafenib 960 mg BID+ Cobimetinib 60 mg QD (VEM+COBI) were included.
Overall Study
STARTED
275
387
54
Overall Study
COMPLETED
275
387
54
Overall Study
NOT COMPLETED
0
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Comparative Effectiveness of Targeted Therapies in BRAF Positive Metastatic Melanoma in the US

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Encorafenib+Binimetinib
n=275 Participants
Participants with BRAFV600-mutant metastatic melanoma, who were enrolled between 30-December 2013 to 15-September-2020 in phase 3 COLUMBUS trial and eligible participants from RWD cohort identified from Flatiron Health Database between 27-June-2018 to 30-September- 2021 and treated with Encorafenib 450 mg QD and Binimetinib 45 mg BID (ENCO+BINI) were included.
Dabrafenib+Trametinib
n=387 Participants
Eligible participants from RWD cohort with BRAFV600-mutant metastatic melanoma identified from Flatiron Health Database between 09-January-2014 to 30-September-2021 and treated with Dabrafenib 150 mg BID+ Trametinib 2 mg QD (DAB+TRAM) were included.
Vemurafenib + Cobimetinib
n=54 Participants
Eligible participants from RWD cohort with BRAFV600-mutant metastatic melanoma identified from Flatiron Health Database between 10-November-2015 to 30-September-2021 and treated with Vemurafenib 960 mg BID+ Cobimetinib 60 mg QD (VEM+COBI) were included.
Total
n=716 Participants
Total of all reporting groups
Age, Continuous
57.5 Years
STANDARD_DEVIATION 13.9 • n=5 Participants
59.3 Years
STANDARD_DEVIATION 13.5 • n=7 Participants
56.4 Years
STANDARD_DEVIATION 14.6 • n=5 Participants
58.4 Years
STANDARD_DEVIATION 13.7 • n=4 Participants
Sex: Female, Male
Female
113 Participants
n=5 Participants
151 Participants
n=7 Participants
17 Participants
n=5 Participants
281 Participants
n=4 Participants
Sex: Female, Male
Male
162 Participants
n=5 Participants
236 Participants
n=7 Participants
37 Participants
n=5 Participants
435 Participants
n=4 Participants
Race/Ethnicity, Customized
White
253 Participants
n=5 Participants
353 Participants
n=7 Participants
48 Participants
n=5 Participants
654 Participants
n=4 Participants
Race/Ethnicity, Customized
Non-white
22 Participants
n=5 Participants
34 Participants
n=7 Participants
6 Participants
n=5 Participants
62 Participants
n=4 Participants

PRIMARY outcome

Timeframe: COLUMBUS: From date of randomization until death or censoring (approx 80.5 months); Flatiron: From index date until death due or censoring or end of follow-up period (92.7 months)

Population: Analysis population included all eligible participants whose data were retrieved and observed in this study.

As per COLUMBUS trial, OS was defined as the time from the date of randomization to the date of death due to any cause; if death was not observed, participants were censored at the date of last contact or the data analysis cut-off date, whichever occurred first. As per Flatiron EHR, OS was defined as the time from the index date to the date of death; participants without a date of death were censored at their last known activity date or the end of the follow-up period, whichever occurred first. Index date in each treatment group was defined as the date of treatment initiation. Kaplan-Meier analyses was used for analysis.

Outcome measures

Outcome measures
Measure
Dabrafenib+Trametinib
n=387 Participants
Eligible participants from RWD cohort with BRAFV600-mutant metastatic melanoma identified from Flatiron Health Database between 09-January-2014 to 30-September-2021 and treated with Dabrafenib 150 mg BID+ Trametinib 2 mg QD (DAB+TRAM) were included.
Vemurafenib + Cobimetinib
n=54 Participants
Eligible participants from RWD cohort with BRAFV600-mutant metastatic melanoma identified from Flatiron Health Database between 10-November-2015 to 30-September-2021 and treated with Vemurafenib 960 mg BID+ Cobimetinib 60 mg QD (VEM+COBI) were included.
Encorafenib+Binimetinib
n=275 Participants
Participants with BRAFV600-mutant metastatic melanoma, who were enrolled between 30-December 2013 to 15-September-2020 in phase 3 COLUMBUS trial and eligible participants from RWD cohort identified from Flatiron Health Database between 27-June-2018 to 30-September- 2021 and treated with Encorafenib 450 mg QD and Binimetinib 45 mg BID (ENCO+BINI) were included.
Overall Survival (OS)
16.1 Months
Interval 13.7 to 18.7
25.0 Months
Interval 17.0 to
To estimate an upper limit of 95% confidence limit (CI), enough follow-up and events were required after the median OS reached. But beyond median OS, participants at risk and events were insufficient. Hence, upper limit of 95% CI could not be estimated.
30.0 Months
Interval 24.4 to 37.6

OTHER_PRE_SPECIFIED outcome

Timeframe: COLUMBUS: From date of randomization until death or censoring (approx 80.5 months); Flatiron: From index date until death due or censoring or end of follow-up period (92.7 months)

Population: Analysis population included all eligible participants whose data were retrieved and observed in this study.

COLUMBUS:PFS=time from the date of randomization to the date of the first documented disease progression (PD) or death due to any cause, whichever occurred first; PD:at least 20 percentage (%) increase in the sum of diameter of all measured target lesions, reference to the smallest sum of diameter of all target lesions recorded at or after baseline. In addition, increase of 20%, the sum must also demonstrate an absolute increase of at least 5 millimiter square (mm2) or the appearance of 1 or more new lesion, if a participant did not have an event at the analysis cut-off date, PFS was censored at the date of the last adequate tumour assessment. Flatiron:PFS=time from the index date to either the date of first PD event or death in the absence of progression; participants without PD or death were censored at the last date the participant could have been assessed for progression or the data analysis cut-off date, whichever occurred first. Index date was =the date of treatment initiation.

Outcome measures

Outcome measures
Measure
Dabrafenib+Trametinib
n=387 Participants
Eligible participants from RWD cohort with BRAFV600-mutant metastatic melanoma identified from Flatiron Health Database between 09-January-2014 to 30-September-2021 and treated with Dabrafenib 150 mg BID+ Trametinib 2 mg QD (DAB+TRAM) were included.
Vemurafenib + Cobimetinib
n=54 Participants
Eligible participants from RWD cohort with BRAFV600-mutant metastatic melanoma identified from Flatiron Health Database between 10-November-2015 to 30-September-2021 and treated with Vemurafenib 960 mg BID+ Cobimetinib 60 mg QD (VEM+COBI) were included.
Encorafenib+Binimetinib
n=275 Participants
Participants with BRAFV600-mutant metastatic melanoma, who were enrolled between 30-December 2013 to 15-September-2020 in phase 3 COLUMBUS trial and eligible participants from RWD cohort identified from Flatiron Health Database between 27-June-2018 to 30-September- 2021 and treated with Encorafenib 450 mg QD and Binimetinib 45 mg BID (ENCO+BINI) were included.
Progression Free Survival (PFS)
7.4 Months
Interval 6.3 to 8.4
7.9 Months
Interval 5.5 to 24.4
14.1 Months
Interval 11.4 to 17.7

Adverse Events

Encorafenib+Binimetinib

Serious events: 0 serious events
Other events: 0 other events
Deaths: 159 deaths

Dabrafenib+Trametinib

Serious events: 0 serious events
Other events: 0 other events
Deaths: 259 deaths

Vemurafenib + Cobimetinib

Serious events: 0 serious events
Other events: 0 other events
Deaths: 30 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer Inc.

Phone: 1-800-718-1021

Results disclosure agreements

  • Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publication until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
  • Publication restrictions are in place

Restriction type: OTHER