Special Drug Use-results Surveillance of Tafinlar/Mekinist

Observational Study to Assess Safety and Effectiveness of Dabrafenib and Trametinib in Patients With BRAF V600E Mutation-positive Unresectable Advanced or Recurrent Solid Tumor

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.

Study Overview

• Status: Recruiting
• Conditions: BRAF V600E Mutation-positive Unresectable Advanced or Recurrent Solid Tumor
• Intervention / Treatment: Drug: Tafinlar/Mekinist

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.

• Study Type: Observational
• Enrollment (Estimated): 90

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals
• Study Contact Backup: Name: Novartis Pharmaceuticals; Phone Number: +81337978748; Email: novartis.email@novartis.com

Study Locations

• Japan
  • Akita, Japan, 010-8543
    • Recruiting
    • Novartis Investigative Site
  • Osaka, Japan, 534-0021
    • Recruiting
    • Novartis Investigative Site
  • Osaka, Japan, 550-0015
    • Recruiting
    • Novartis Investigative Site
  • Hyogo
    • Kobe, Hyogo, Japan, 650-0011
      • Recruiting
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

patients who used the product for the treatment of BRAF-mutation-positive advanced/recurrent solid tumors (excluding colorectal cancer) as per the package insert.

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Tafinlar/Mekinist; Patients with BRAF V600E mutation positive unresectable advanced or recurrent solid tumors treated with dabrafenib and trametinib as per Japanese Package Insert.	Drug: Tafinlar/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.; Other Names: Dabrafenib/trametinib

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
[Pediatric patients] Number of patients and incidence of adverse events/adverse reactions with respect to skeletal and sexual maturation	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.	Up to 8 years
[Adult patients] overall response rate (ORR)	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)	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
[Pediatric patients] Incidence proportion of ORR	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)	1 year
[Pediatric and adult patients] Incidence proportion of PFS	Progression Free Survival (PFS) is the percentage of participants who did not have a progression event.	1 year
[Pediatric patients] Number of patients with changes in height and body weight and sexual maturation	Number of pediatric patients with changes in height and body weight and sexual maturation is going to be collected	Up to 8 years

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): February 9, 2024
• Primary Completion (Estimated): December 31, 2031
• Study Completion (Estimated): December 31, 2031

Study Registration Dates

• First Submitted: February 8, 2024
• First Submitted That Met QC Criteria: February 8, 2024
• First Posted (Actual): February 16, 2024

Study Record Updates

• Last Update Posted (Actual): March 22, 2024
• Last Update Submitted That Met QC Criteria: March 21, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: solid tumor; trametinib; dabrafenib; Japan; recurrent; Mekinist; Tafinlar; BRAF V600E mutation-positive unresectable advanced
• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Recurrence; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Protein Kinase Inhibitors; Trametinib; Dabrafenib
• Other Study ID Numbers: CDRB436I1401

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO