Neoplasms

Skin and Connective Tissue Diseases

All Conditions

Rare Diseases

Melanoma

Neuroendocrine Tumors

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Nerve Tissue

Nevus, Pigmented

Nevus

Nevi and Melanomas

Skin Neoplasms

Skin Diseases

Neuroendocrine Tumor

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Pembrolizumab

Immunomodulating Agents

Nivolumab

Ipilimumab

Trametinib

Dabrafenib

Vemurafenib

Antineoplastic Agents, Immunological

Immune Checkpoint Inhibitors

Protein Kinase Inhibitors

Enzyme Inhibitors

Included patients were aged more than or equal to 18 years, were required to have a diagnosis of melanoma (ICD-9 172.x \& ICD-10 C43.x or D03.x), pathologic stage III disease, evidence of resection, adjuvant treatment with IO (e.g., nivo or pembro) or TT (e.g., dab+tram) on or after January 1, 2014, and prior to August 30,2020 (data cut-off), and any evidence of a BRAF+ result.

Included patients were aged more than or equal to 18 years, and were required to have a diagnosis of melanoma (ICD-9 172.x \& ICD-10 C43 or D03x), a pathologic stage IV diagnosis, treatment with IO (e.g. ipi, nivo, pembro, ipi+nivo) or TT (dab+tram, vem+cobi, enco+bini) on or after January 1, 2014 and prior to May 31, 2020 (data cut-off), and evidence of a BRAF+ result after therapy initiation. Patients were required to be classified as LTB at the time of stage IV diagnosis. LTB was defined as having normal LDH and \<3 metastatic sites at the time of stage IV diagnosis.

Dabrafenib+Trametinib

Ipilimumab+Nivolumab

Vemurafenib+Cobimetinib

Encorafenib+Binimetinib

Novartis Pharmaceuticals

This was an observational study utilizing electronic health record (EHR)-derived data collected retrospectively during routine care of real-world patients with advanced melanoma from NOBLE (Novartis Braf+ meLanoma patients ObsErvational) dataset.

The NOBLE database was built from the harmonization of two customized oncology specific EHR databases: Flatiron Health Spotlight and Concerto Custom Patient360. BRAF v600 mutated advanced (i.e., stage III or IV) patients treated at oncology practices across the US were identified in these two databases for potential inclusion. Both the Flatiron Health EHR-derived database and the Concerto Patient360 database contain clinical, demographic, treatment, and mortality information for melanoma patients from the time of initial diagnosis until death or the most recent data cut-off, which is August 31, 2020 (for population 1), and May 31, 2020 (for population 2).

For population 1 (patients treated in the adjuvant setting): included patients were aged more than or equal to 18 years, were required to have a diagnosis of melanoma (ICD-9 172.x \& ICD-10 C43.x or D03.x), pathologic stage III disease, evidence of resection, adjuvant treatment with Immunotherapy (IO) (e.g., Nivolumab (nivo) or Pembrolizumab (pembro)) or Targeted Therapy (TT) (e.g., Dabrafenib+ Trametinib (dab+tram)) on or after January 1, 2014 and prior to August 30,2020 (data cut-off), and any evidence of a BRAF+ result. Patients were required to have at least 6 months of follow-up after initiation of adjuvant treatment. Patients were followed until the earlier of death, data cut-off, loss of follow-up, or received MM diagnosis. While the first systemic therapy approved for use in the adjuvant melanoma setting occurred in 2015, the study period of interest begins on January 1, 2014, to include any potential off-label use of these therapies as adjuvant therapies.

For population 2 (patients with Low tumor burden (LTB) treated in the metastatic setting): included patients were aged more than or equal to 18 years, and were required to have a diagnosis of melanoma (ICD-9 172.x \& ICD-10 C43 or D03x), a pathologic stage IV diagnosis, treatment with IO (e.g. Ipilimumab (ipi), nivo, pembro, ipi+nivo) or TT (dab+tram, Vemurafenib+Cobimetinib, Encorafenib+Binimetinib (vem+cobi, enco+bini) on or after January 1, 2014 and prior to May 31, 2020 (data cut-off), and evidence of a BRAF+ result after therapy initiation. Patients were required to be classified as LTB at the time of stage IV diagnosis. LTB was defined as having normal LDH and \<3 metastatic sites at the time of stage IV diagnosis. To align with recent FDA approvals for combination therapies use in the MM setting, the study period of interest began on January 1, 2014. Furthermore, this sampling interval allowed for a maximum of 6 years of follow-up from the start of study period.

Treatment Patterns and Outcomes of Targeted Therapy and Immunotherapy Among BRAF-Positive Melanoma Patients Treated in the Adjuvant Setting and Among BRAF-Positive Metastatic Melanoma Patients With Low Tumor Burden

Real-World Treatment Patterns and Outcomes of Targeted Therapy and Immunotherapy Among BRAF-Positive Melanoma Patients Treated in the Adjuvant Setting and Among BRAF-Positive Metastatic Melanoma Patients With Low Tumor Burden: An Observational Study

To describe treatment patterns among patients prescribed with TT versus IO in both the populations.

To evaluate discontinuation of 1L treatment among patients receiving TT or IO.

To estimate OS from initiation of 1L treatment among patients receiving TT or IO.

To estimate Recurrence Free Survival (RFS) (for population 1) from initiation of 1L treatment among patients receiving TT or IO.

To estimate Progression Free Survival (PFS) (for population 2) from initiation of 1L treatment among patients receiving TT or IO.

Blood and Lymph Conditions

Hematologic Neoplasms

Hematologic Diseases

Amino Acids

Trastuzumab

Pertuzumab

Atezolizumab

Alectinib

Pralsetinib

Entrectinib

Tyrosine Kinase Inhibitors

Tyrosine

For patients with a molecular tumor profile that can potentially be targeted by alectinib.

For patients with a molecular tumor profile that can potentially be targeted by cobimetinib.

For patients with a molecular tumor profile that can potentially be targeted by vismodegib.

For patients with a molecular tumor profile that can potentially be targeted by trastuzumab+pertuzuma combination.

For patients with a molecular tumor profile that can potentially be targeted by entrectinib.

For patients with a molecular tumor profile that can potentially be targeted by atezolizumab.

For patients with a molecular tumor profile that can potentially be targeted by vemurafenib.

For patients with a molecular tumor profile that can potentially be targeted by regorafenib.

For patients with a molecular tumor profile that can potentially be targeted by apalutamide.

For patients with a molecular tumor profile that can potentially be targeted by abemaciclib.

For patients with a molecular tumor profile that can potentially be targeted by selpercatinib.

For patients with a molecular tumor profile that can potentially be targeted by dabrafenib.

For patients with a molecular tumor profile that can potentially be targeted by trametinib.

For patients with a molecular tumor profile that can potentially be targeted by dabrafenib+trametinib combination.

For patients with a molecular tumor profile that can potentially be targeted by pralsetinib.

Cobimetinib

Vismodegib

Trastuzumab+Pertuzumab

Regorafenib

Apalutamide

Abemaciclib

Selpercatinib

Tanja Juslin

Katriina Jalkanen, MD, PhD

Erika Alanne, MD, PhD

Okko Kääriäinen, MD

Minna Tanner, MD, PhD

Katriina Peltola, MD, PhD

This is a prospective non-randomized national clinical phase 2 trial that aims to determine the efficacy and toxicity of targeted anticancer drugs or combinations that are approved or under review by EMA, FDA or PMDA and are used for treatment of patients with advanced cancer with a potentially actionable variant as revealed by a genomic, RNA-molecular or protein expression test.

This is a prospective non-randomized clinical trial that aims to determine the efficacy and toxicity of targeted anticancer drugs or combinations that are approved or under review by EMA, FDA or PMDA and are used for treatment of patients with advanced cancer with a potentially actionable variant as revealed by a genomic, RNA-molecular or protein expression test. The study also aims to facilitate patient access to approved targeted therapies that are contributed to the program by collaborating pharmaceutical companies and to perform next generation sequencing and further deeper analysis on tumor biopsies and/or liquid biopsies for biomarker analyses and resistance mechanisms. Eligible patients have an advanced cancer for which standard treatment options no longer exist and acceptable performance status and organ function. A tumour DNA, RNA or protein expression analysis is required and the results must identify at least one potentially actionable molecular variant as defined in the protocol. Molecular profiling will be utilized to determine an appropriate drug(s) from among those available in the protocol. Drug selection will be guided by a list of potential profiles, the molecular tumor board and databases of identified targets for review and approval of the recommended treatment. The protocol-specified treatment will be administered to the patient once any drug- and disease specific eligibility criteria and overall study criteria are met. Data for standard efficacy outcomes including tumor response, progression-free and overall survival as well as duration of treatment will be collected for all patients receiving treatment within the trial. In addition, treatment related toxicity will be collected according to CTCAE 5.0.

The Finnish National Study to Facilitate Patient Access to Targeted Anti-cancer Drugs

The Finnish National Study to Facilitate Patient Access to Targeted Anti-cancer Drugs to Determine the Efficacy in Treatment of Advanced Cancers With a Known Molecular Profile

Investigators plan to share the clinical study report.

Disease control rate at 16 weeks after treatment initiation (defined as patients by CR, PR, SD)

Treatment-related grade ≥3 and serious adverse events

Best overall response (defined as patients by CR, PR, SD)

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Treatment Patterns and Outcomes of Targeted Therapy and Immunotherapy Among BRAF-Positive Melanoma Patients Treated in the Adjuvant Setting and Among BRAF-Positive Metastatic Melanoma Patients With Low Tumor Burden	NCT05611229	Metastatic Mela...	Nivolumab Pembrolizumab Dabrafenib+Tram... Ipilimumab+Nivo... Vemurafenib+Cob... Encorafenib+Bin...	18 Years -	Novartis
The Finnish National Study to Facilitate Patient Access to Targeted Anti-cancer Drugs	NCT05159245	Advanced Cancer Solid Tumor Haematological ...	Alectinib Cobimetinib Vismodegib Trastuzumab+Per... Entrectinib Atezolizumab Vemurafenib Regorafenib Apalutamide Abemaciclib Selpercatinib Dabrafenib Trametinib Dabrafenib+Tram... Pralsetinib	18 Years - 100 Years	Helsinki University Central Hospital
Treatment Patterns and Outcomes of Targeted Therapy and Immunotherapy Among BRAF-Positive Melanoma Patients Treated in the Adjuvant Setting and Among BRAF-Positive Metastatic Melanoma Patients With Low Tumor Burden	NCT05611229	Metastatic Mela...	Nivolumab Pembrolizumab Dabrafenib+Tram... Ipilimumab+Nivo... Vemurafenib+Cob... Encorafenib+Bin...	18 Years -	Novartis

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