MedPath

Special Drug Use-results Surveillance of Tafinlar/Mekinist

Recruiting
Conditions
BRAF V600E Mutation-positive Unresectable Advanced or Recurrent Solid Tumor
Interventions
Drug: Tafinlar/Mekinist
Registration Number
NCT06262919
Lead Sponsor
Novartis Pharmaceuticals
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.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
110
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

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
Tafinlar/MekinistTafinlar/MekinistPatients with BRAF V600E mutation positive unresectable advanced or recurrent solid tumors treated with dabrafenib and trametinib as per Japanese Package Insert.
Primary Outcome Measures
NameTimeMethod
[Pediatric patients] Number of patients and incidence of adverse events/adverse reactions with respect to skeletal and sexual maturationUp 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 Outcome Measures
NameTimeMethod
[Pediatric patients] Incidence proportion of ORR1 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 patients] Number of patients with changes in height and body weight and sexual maturationUp to 8 years

Number of pediatric patients with changes in height and body weight and sexual maturation is going to be collected

[Pediatric and adult patients] Incidence proportion of PFS1 year

Progression Free Survival (PFS) is the percentage of participants who did not have a progression event.

Trial Locations

Locations (1)

Novartis Investigative Site

🇯🇵

Yamagata, Japan

© Copyright 2025. All Rights Reserved by MedPath