BRAF V600E Mutation-positive Unresectable Advanced or Recurrent Solid Tumor
Conditions
Keywords
Tafinlar, dabrafenib, Mekinist, trametinib, BRAF V600E mutation-positive unresectable advanced, solid tumor, Japan, recurrent
Brief summary
This is a prospective, multicenter, single-arm, non-interventional and observational J-PMS conducted by the central registration system and operated in Electronic data capture.
Detailed description
In the Post-Marketing Surveillance (PMS), dabrafenib and trametinib are used as the marketed drugs. Registration of the corresponding patients is to be conducted by the central registered system under current medical practice. Target number of adult patient is 65 (as the number of patients in the effectiveness analysis set). Target number of pediatric patient is not determined. Estimated number of enrolled patients is approximately 20 (as the number of patients in the enrolled set) The observation period for pediatric patients will last after the start of treatment until 8 years (planned, November 2031) after the approval of additional indications, regardless of discontinuation of the product, in order to collect long-term information from as many patients as possible during the reexamination period. The duration of observation for adult patients will be 1 year after the start of treatment with the product.
Interventions
DRUGTafinlar/Mekinist
There is no treatment allocation. Patients administered Tafinlar/Mekinist by prescription that have started before inclusion of the patient into the study will be enrolled.
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
1. Patients who have given written consent to cooperate in this surveillance 2. For patients aged \< 18 years at the start of treatment with the product, their legally authorized representative must have given written informed consent for cooperation in this surveillance prior to patient enrollment. 3. Patients who start treatment with the product for BRAF-mutation-positive advanced/recurrent solid tumors (excluding colorectal cancer) after the approval of additional indications
Exclusion criteria
1. Patients who have received or are receiving a product containing the same ingredient as the product in any other study or research than this surveillance 2. Patients with BRAF-mutation-positive malignant melanoma 3. Patients with BRAF-mutation-positive non-small cell lung cancer 4. Patients with BRAF-mutation-positive hairy cell leukemia
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| [Pediatric patients] Number of patients and incidence of adverse events/adverse reactions with respect to skeletal and sexual maturation | Up to 8 years | To evaluate impairment in growth and development in pediatric patients (skeletal and sexual maturation) of dabrafenib and/or trametinib in pediatric patients under 18 years old in the post marketing phase. |
| [Adult patients] overall response rate (ORR) | 1 year | The Overall Response Rate (ORR) is defined as the proportion of patients with a best OR of confirmed Complete Response (CR) or Partial Response (PR) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| [Pediatric patients] Incidence proportion of ORR | 1 year | The Overall Response Rate (ORR) is defined as the proportion of patients with a best OR of confirmed Complete Response (CR) or Partial Response (PR) |
| [Pediatric and adult patients] Incidence proportion of PFS | 1 year | Progression Free Survival (PFS) is the percentage of participants who did not have a progression event. |
| [Pediatric patients] Number of patients with changes in height and body weight and sexual maturation | Up to 8 years | Number of pediatric patients with changes in height and body weight and sexual maturation is going to be collected |
Countries
Japan
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed