Clinical trial NCT06251934: Real-World Patient Characteristics, Treatment Patterns, and Clinical Outcomes Among Patients With BRAF-Positive Metastatic Melanoma
Official title: Real-World Patient Characteristics, Treatment Patterns, and Clinical Outcomes Among Patients With BRAF-Positive Metastatic Melanoma
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: BRAF-positive Metastatic Melanoma
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2024-02-09
Enrollment: 785
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This was a retrospective, noninterventional cohort study.

Brief summary: This was a retrospective, longitudinal, observational study conducted using the Flatiron Health electronic health record (EHR)-derived database. BRAF+ advanced or metastatic (i.e., stage III or IV) melanoma patients treated at oncology practices across the US were identified for potential inclusion. All included patients were aged ≥18 years and required to have a diagnosis of melanoma (International Classification of Diseases (ICD)-9 172.x \& ICD-10 C43 or D03x), a pathologic unresectable stage III or IV diagnosis, subsequent first-line (1L) treatment with either immunotherapy (IO) (nivolumab, pembrolizumab, ipilimumab + nivolumab) or targeted therapy (TT) dafratenib + trametinib (dab/tram) on or after 01 January 2014, and evidence of a BRAF-positive result at any point in time.


Eligibility criteria: Inclusion criteria:

* Diagnosis of melanoma (ICD-9 172x \& ICD-10 C43 or D03x)
* ≥2 documented clinical encounters on different days in the Flatiron network on or after 01 January 2011
* Pathologic unresectable stage III or IV at initial diagnosis after 01 January 2011, or earlier stage disease accompanied by development of a first locoregional recurrence after 01 January 2011
* Diagnosis of MM after 01 January 2011
* Evidence of a BRAF-positive result at any point in time
* Treatment with one of the following 1L therapies on or after 01 January 2014:

  * IO (nivolumab, pembrolizumab, or ipilimumab + nivolumab)
  * TT (dab/tram)
* At least 18 years of age at the time of initiation of 1L therapy
* At least 6-months of continuous follow-up from the time of initiation of 1L therapy

Exclusion Criteria:

* Lacking relevant unstructured documents (i.e., information such as free text from a physician note or pathology report that is captured systematically during the data abstraction process) in the Flatiron Health database
* Evidence of non-skin melanoma (ocular, subungual, mucosal, palmar, plantar)
* Documented receipt of a clinical study drug, defined as any uncancelled order, administration, or oral episode for a clinical study drug for cancer at any time prior to or during 1L treatment
* Presence of leptomeningeal disease (ICD-9 198.4 \& ICD-10 C79.32 or C79.49)

Primary outcomes:
measure: Mean age of 1L dab/tram-treated BRAF+ MM patients
timeFrame: Baseline
measure: Number and percentage of 1L dab/tram-treated BRAF+ MM patients who identify per sex
timeFrame: Baseline
measure: Number and percentage of 1L dab/tram-treated BRAF+ MM patients per race category
timeFrame: Baseline
measure: Number and percentage of 1L dab/tram-treated BRAF+ MM patients per ethnicity category
timeFrame: Baseline
measure: Number and percentage of 1L dab/tram-treated BRAF+ MM patients per geographic region
timeFrame: Baseline
measure: Number and percentage of 1L dab/tram-treated BRAF+ MM patients per type of medical practice
timeFrame: Baseline
measure: Number and percentage of 1L dab/tram-treated BRAF+ MM patients per insurance type
timeFrame: Baseline
measure: Number and percentage of 1L dab/tram-treated BRAF+ MM patients with an Eastern Cooperative Oncology Group (ECOG) score of 0, 1, 2, 3, or 4
timeFrame: Baseline
measure: Number and percentage of 1L dab/tram-treated BRAF+ MM patients per location of metastatic site
timeFrame: Baseline
measure: Alanine aminotransferase (ALT) levels in 1L dab/tram-treated BRAF+ MM patients
timeFrame: Baseline
measure: Aspartate aminotransferase (AST) levels in 1L dab/tram-treated BRAF+ MM patients
timeFrame: Baseline
measure: Lactate dehydrogenase (LDH) levels in 1L dab/tram-treated BRAF+ MM patients
timeFrame: Baseline
measure: Number and percentage of 1L dab/tram-treated BRAF+ MM patients treated with 2L therapy
timeFrame: Up to approximately 2 years
measure: Time from the start of 1L dab/tram therapy until the start of 2L therapy
timeFrame: Up to approximately 2 years
measure: Real-world progression-free survival (rwPFS) for 1L dab/tram-treated BRAF+ MM patients
timeFrame: Up to approximately 2 years
measure: Overall survival (OS) for 1L dab/tram-treated BRAF+ MM patients
timeFrame: Up to approximately 2 years
Secondary outcomes:
measure: Mean age of 1L IO-refractory patients with BRAF+ MM
timeFrame: Baseline
measure: Number and percentage of 1L IO-refractory patients with BRAF+ MM who identify per sex
timeFrame: Baseline
measure: Number and percentage of 1L IO-refractory patients with BRAF+ MM per race category
timeFrame: Baseline
measure: Number and percentage of 1L IO-refractory patients with BRAF+ MM per ethnicity category
timeFrame: Baseline
measure: Number and percentage of 1L IO-refractory patients with BRAF+ MM per geographic region
timeFrame: Baseline
measure: Number and percentage of 1L IO-refractory patients with BRAF+ MM per type of medical practice
timeFrame: Baseline
measure: Number and percentage of 1L IO-refractory patients with BRAF+ MM per insurance type
timeFrame: Baseline
measure: Number and percentage of 1L IO-refractory patients with BRAF+ MM with an ECOG score of 0, 1, 2, 3, or 4
timeFrame: Baseline
measure: Number and percentage of 1L IO-refractory patients with BRAF+ MM per location of metastatic site
timeFrame: Baseline
measure: Alanine aminotransferase (ALT) levels in 1L IO-refractory patients with BRAF+ MM
timeFrame: Baseline
measure: Aspartate aminotransferase (AST) levels in 1L IO-refractory patients with BRAF+ MM
timeFrame: Baseline
measure: Lactate dehydrogenase (LDH) levels in 1L IO-refractory patients with BRAF+ MM
timeFrame: Baseline
measure: Time from the first disease progression until next disease progression in 1L IO-refractory patients with BRAF+ MM
timeFrame: Up to approximately 2 years
measure: Number and proportion of patients remaining on 1L IO therapy following disease progression
timeFrame: Up to approximately 2 years
measure: Time patients remained on 1L IO therapy following disease progression
timeFrame: Up to approximately 2 years
measure: Number and percentage of patients treated with 2L therapy, overall and by therapy type
timeFrame: Up to approximately 2 years
measure: Time from the start of 1L IO until the start of 2L therapy
timeFrame: Up to approximately 2 years
measure: Time from the first disease progression until the start of 2L therapy
timeFrame: Up to approximately 2 years
measure: rwPFS for 1L IO-refractory patients with BRAF+ MM
timeFrame: Up to approximately 2 years
measure: OS for 1L IO-refractory patients with BRAF+ MM
timeFrame: Up to approximately 2 years
Other outcomes:
Locations:
city: East Hanover
country: United States
facility: Novartis
state: New Jersey
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: