Rare Unresectable or Metastatic BRAF V600E Mutation-positive Solid Tumors
Conditions
Keywords
BRAF, v-raf murine sarcoma viral oncogene homolog B1, dabrafenib, trametinib, tumor agnostic
Brief summary
This study is a phase IV, pragmatic single-arm prospective, open label study in pediatric (1 years or older) and adult study participants with rare BRAF V600E mutation-positive unresectable or metastatic solid tumors for whom a decision has already been made to be treated with dabrafenib and trametinib, irrespective of the trial participation.
Interventions
OTHERNon-investigational
Participants obtaining commercial (non-investigational) dabrafenib plus trametinib (i.e. solid formulation or liquid formulation, if approved and commercially available locally) per local guidance or patient access program
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
1 Years to 100 Years
Healthy volunteers
No
Inclusion criteria
* Study participant with a BRAF V600E mutation-positive solid tumor as confirmed by a local laboratory test; * At least 1 measurable lesion as defined by RECIST v1.1 per local review; * Study participant previously not treated with dabrafenib and/or trametinib. Study participants who received dabrafenib and trametinib in the past for the treatment of other malignancies are eligible if treatment has been discontinued for greater than 1 year; * Ability to provide scans for central imaging review
Exclusion criteria
* Those with the following tumor types: melanoma, NSCLC, ATC, BTC, glioma and CRC; * Study participants who have contraindication to receive dabrafenib and/ or trametinib according to the local label;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Response Rate (ORR) | Approximately 4 years | Proportion of study participants with a best overall confirmed Complete Response (CR) or Partial Response (PR) by independent central review according to RECIST v1.1 or other relevant response criteria. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Duration of response (DOR) | Approximately 4 years | Defined as time from the onset of tumor response until the date of the first documented progressive disease (PD) or death due to any cause. |
| Clinical benefit rate (CBR) | Approximately 4 years | Defined as the proportion of study participants with a best overall response of CR, PR or stable disease (SD), lasting as per independent central review, for a duration of at least 24 weeks. CR, PR and SD are defined according to RECIST v1.1. or other relevant response criteria. |
Countries
United States
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed