Clinical Study To Further Evaluate The Efficacy Of Dabrafenib Plus Trametinib In Patients With Rare BRAF V600E Mutation-Positive Unresectable or Metastatic Solid Tumors

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05868629

Collaborator

(none)

Enrollment

48.8

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

This study is a phase IV, pragmatic single-arm prospective, open label study in pediatric (6 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.

Condition or Disease	Intervention/Treatment	Phase
Rare Unresectable or Metastatic BRAF V600E Mutation-positive Solid Tumors	Other: Non-investigational

Study Design

Study Type:

Observational

Anticipated Enrollment :

40 participants

Observational Model:

Other

Time Perspective:

Prospective

Official Title:

Clinical Study To Further Evaluate The Efficacy Of Dabrafenib Plus Trametinib In Patients With Rare BRAF V600E Mutation-Positive Unresectable or Metastatic Solid Tumors

Anticipated Study Start Date :

Aug 4, 2023

Anticipated Primary Completion Date :

Aug 29, 2027

Anticipated Study Completion Date :

Aug 29, 2027

Arms and Interventions

Arm	Intervention/Treatment
dabrafenib plus trametinib patients treated with dabrafenib and trametinib	Other: Non-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

Arm

Intervention/Treatment

dabrafenib plus trametinib

patients treated with dabrafenib and trametinib

Other: Non-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

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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;