Clinical trial NCT06262919: Special Drug Use-results Surveillance of Tafinlar/Mekinist
Official title: Observational Study to Assess Safety and Effectiveness of Dabrafenib and Trametinib in Patients With BRAF V600E Mutation-positive Unresectable Advanced or Recurrent Solid Tumor
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: BRAF V600E Mutation-positive Unresectable Advanced or Recurrent Solid Tumor
Keywords: Tafinlar
dabrafenib
Mekinist
trametinib
BRAF V600E mutation-positive unresectable advanced
solid tumor
Japan
recurrent
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2024-02-16
Enrollment: 110
Sex: ALL
Minimum age (years): 6
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: [Pediatric patients] Number of patients and incidence of adverse events/adverse reactions with respect to skeletal and sexual maturation
timeFrame: Up to 8 years
measure: [Adult patients] overall response rate (ORR)
timeFrame: 1 year
Secondary outcomes:
measure: [Pediatric patients] Incidence proportion of ORR
timeFrame: 1 year
measure: [Pediatric and adult patients] Incidence proportion of PFS
timeFrame: 1 year
measure: [Pediatric patients] Number of patients with changes in height and body weight and sexual maturation
timeFrame: Up to 8 years
Other outcomes:
Locations:
city: Nagakute-city
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Nagoya
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Nagoya
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Toyota
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Daisen
country: Japan
facility: Novartis Investigative Site
state: Akita
city: Funabashi
country: Japan
facility: Novartis Investigative Site
state: Chiba
city: Kashiwa
country: Japan
facility: Novartis Investigative Site
state: Chiba
city: Fukuoka city
country: Japan
facility: Novartis Investigative Site
state: Fukuoka
city: Kurume city
country: Japan
facility: Novartis Investigative Site
state: Fukuoka
city: Fukuyama
country: Japan
facility: Novartis Investigative Site
state: Hiroshima
city: Sapporo
country: Japan
facility: Novartis Investigative Site
state: Hokkaido
city: Amagasaki
country: Japan
facility: Novartis Investigative Site
state: Hyogo
city: Kobe
country: Japan
facility: Novartis Investigative Site
state: Hyogo
city: Kobe
country: Japan
facility: Novartis Investigative Site
state: Hyogo
city: Mito
country: Japan
facility: Novartis Investigative Site
state: Ibaraki
city: Morioka
country: Japan
facility: Novartis Investigative Site
state: Iwate
city: Kagoshima city
country: Japan
facility: Novartis Investigative Site
state: Kagoshima
city: Sagamihara
country: Japan
facility: Novartis Investigative Site
state: Kanagawa
city: Yokohama-city
country: Japan
facility: Novartis Investigative Site
state: Kanagawa
city: Natori
country: Japan
facility: Novartis Investigative Site
state: Miyagi
city: Iida
country: Japan
facility: Novartis Investigative Site
state: Nagano
city: Matsumoto
country: Japan
facility: Novartis Investigative Site
state: Nagano
city: Okayama city
country: Japan
facility: Novartis Investigative Site
state: Okayama
city: Izumisano-city
country: Japan
facility: Novartis Investigative Site
state: Osaka
city: Takatsuki
country: Japan
facility: Novartis Investigative Site
state: Osaka
city: Kawagoe
country: Japan
facility: Novartis Investigative Site
state: Saitama
city: Bunkyo ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Bunkyo-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Bunkyo-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Chuo ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Minato-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Minato-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Setagaya-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Shibuya
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Shinjuku Ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Shinjuku-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Akita
country: Japan
facility: Novartis Investigative Site
city: Kochi
country: Japan
facility: Novartis Investigative Site
city: Kyoto
country: Japan
facility: Novartis Investigative Site
city: Osaka
country: Japan
facility: Novartis Investigative Site
city: Osaka
country: Japan
facility: Novartis Investigative Site
city: Osaka
country: Japan
facility: Novartis Investigative Site
city: Saitama
country: Japan
facility: Novartis Investigative Site
city: Wakayama
country: Japan
facility: Novartis Investigative Site
city: Yamagata
country: Japan
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: