Symptoms and General Pathology

All Conditions

Neoplasms

Skin and Connective Tissue Diseases

Rare Diseases

Recurrence

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

Patients undergo collection of tissue and blood samples for DNA and RNA analysis via sequencing. Based on the results of the DNA and RNA analysis, patients receive molecularly targeted therapy. Treatment continues in the absence of disease progression or unacceptable toxicity.

Undergo collection of tissue and blood samples

cytology specimen collection procedure

molecularly targeted therapy, MEK 162 therapy

MEK 162 therapy or molecularly targeted therapy

therapeutic procedure

laboratory biomarker analysis

quality-of-life assessment

Patricia LoRusso, D.O.

This phase II trial studies how well molecularly targeted therapy works in treating patients with melanoma that has spread to other parts of the body. Patients must have received or do not qualify for prior immunotherapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific types of cancer cells with less harm to normal cells. Molecularly targeted therapy works by treating patients with substances that kill cancer cells by targeting key molecules involved in cancer cell growth.

PRIMARY OBJECTIVES:

I. To determine the difference in best overall response rate (BORR) between patients treated with MEK162 following personalized molecularly guided assignment vs. a historical BORR of 7% in this patient population.

SECONDARY OBJECTIVES:

I. To evaluate the safety of performing individualized drug therapy (including novel agents and commercially-available agents) in the context of a personalized medicine clinical trial.

II. To define the difference in progression free survival (PFS) between patients treated with MEK162 following personalized molecularly guided assignment vs. a historical PFS rate of 2 months in this patient population.

III. To continually assess data in real time so as to iteratively refine and standardize a set of statistical and informatics methodologies for matching treatments to the patient's tumor, based on the molecular profile.

OUTLINE:

Patients undergo collection of tissue and blood samples for deoxyribonucleic acid (DNA) and ribonucleic acid (RNA) analysis via sequencing. Based on the results of the DNA and RNA analysis, patients receive molecularly targeted therapy. Treatment continues in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up for 30 days.

Molecularly Targeted Therapy in Treating Patients With BRAF Wild-type Melanoma That is Metastatic

Stand Up to Cancer Consortium Genomics-Enabled Medicine for Melanoma (G.E.M.M.): Using Molecularly-Guided Therapy for Patients With BRAF Wild-Type (BRAFwt) Metastatic Melanoma

The best overall response rate (BORR) was assessed up to 1 year.

A log rank test will be performed for the comparison between the two groups.

National Cancer Institute (NCI)

Yale University

Patients undergo collection of tissue and blood samples for DNA and RNA analysis via sequencing. Based on the results of the DNA and RNA analysis, patients receive molecularly targeted therapy. Treatment continues in the absence of disease progression or unacceptable toxicity.

cytology specimen collection procedure: Undergo collection of tissue and blood samples

MEK 162 therapy or molecularly targeted therapy: molecularly targeted therapy, MEK 162 therapy

therapeutic procedure

laboratory biomarker analysis: Correlative studies

quality-of-life assessment: Ancillary studies

Arm I (Molecularly Targeted Therapy)

30-39

40-49

50-59

60-69

70-79

80-89

Age, Customized

Sex: Female, Male

American Indian or Alaskan Native

Asian or Pacific Islander

Black, not of Hispanic Origin

Hispanic

White, not of Hispanic Origin

Other/

Race/Ethnicity, Customized

United States

Region of Enrollment

Because of the lack of clinical response, it was decided that enrollment should discontinue. Twenty patients who received MEK162 were evaluated for the primary outcome; secondary outcome was not evaluated.

Manuel Avedissian, M.D.

Progressive Disease

Stable Disease

Partial Response

Best Overall Response Rate (BORR)

Given the lack of response in this study and the decision to terminate it early, no data for PFS data were collected or analyzed. In addition, with the study amendment removing the second arm, this analysis would not have been possible to conduct.

Progression-Free Survival (PFS)

Overall Study

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Molecularly Targeted Therapy in Treating Patients With BRAF Wild-type Melanoma That is Metastatic	NCT02094872	Recurrent Melan... Stage IIIA Mela... Stage IIIB Mela... Stage IIIC Mela... Stage IV Melano...	cytology specim... MEK 162 therapy... therapeutic pro... laboratory biom... quality-of-life...	18 Years -	Yale University
Molecularly Targeted Therapy in Treating Patients With BRAF Wild-type Melanoma That is Metastatic	NCT02094872	Recurrent Melan... Stage IIIA Mela... Stage IIIB Mela... Stage IIIC Mela... Stage IV Melano...	cytology specim... MEK 162 therapy... therapeutic pro... laboratory biom... quality-of-life...	18 Years -	Yale University
Molecularly Targeted Therapy in Treating Patients With BRAF Wild-type Melanoma That is Metastatic	NCT02094872	Recurrent Melan... Stage IIIA Mela... Stage IIIB Mela... Stage IIIC Mela... Stage IV Melano...	cytology specim... MEK 162 therapy... therapeutic pro... laboratory biom... quality-of-life...	18 Years -	Yale University

Spots Global Cancer Trial Database for mek 162 therapy or molecularly targeted therapy