Neoplasms

All Conditions

Symptoms and General Pathology

Digestive System Diseases

Rare Diseases

Adenocarcinoma

Neoplasm Metastasis

Carcinoid Tumor

Appendiceal Neoplasms

Adenocarcinoma, Mucinous

Cystadenocarcinoma

Neoplasms, Cystic, Mucinous, and Serous

Carcinoma

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Neuroendocrine Tumors

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms, Nerve Tissue

Cecal Neoplasms

Intestinal Neoplasms

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Cecal Diseases

Intestinal Diseases

Adenocarcinoma of the Appendix

Neuroendocrine Tumor

Neuroepithelioma

Anti-Infective Agents

All Drugs and Chemicals

Formaldehyde

Individuals ages 18+ years with an appendix cancer diagnosis in the United States.

Individuals ages 18+ years with an appendix cancer diagnosis seen at Vanderbilt.

Biological parents of active GAP (Social and Vanderbilt) Study participants.

Genetic profiling

Collection of archived fixed-formalin, paraffin-embedded (FFPE) primary appendix tumor tissues that were previously removed

Retrospective tissue procurement

GAP Study Coordinator (Rebecca B.)

Andreana N Holowatyj, PhD, MSCI

The GAP Study is a prospective cohort study designed to comprehensively investigate genetic variations that may contribute to cancer development among individuals diagnosed with appendix/appendiceal cancer who are ages 18+ years.

The Genetics of Appendix Cancer (GAP) Study aims to analyze hereditary factors, tumor characteristics and clinical features/outcomes among adults diagnosed with appendix cancer and their biological parents. Patients are recruited at any time after a diagnosis of appendix cancer and followed for up to 6 years after study enrollment. This cohort is enriched by robust biospecimens and data collections.

Genetics of Appendix Cancer Study

Genetics of Appendix Cancer: The GAP Study

Andreana Holowatyj, PhD, MSCI

Sponsor Investigator

Carcinoma, Squamous Cell

Syndrome

Neoplasms, Squamous Cell

Small Intestinal Adenocarcinoma

Antineoplastic Agents

Atezolizumab

Antibodies

Antibodies, Monoclonal

Immunoglobulins

Immune Checkpoint Inhibitors

Antineoplastic Agents, Immunological

Immunologic Factors

Participants receive cobimetinib PO QD on days 1-21 and atezolizumab IV over 60 minutes on days 1 and 15. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Cobimetinib

Kanwal P Raghav

This phase II trial studies how well cobimetinib and atezolizumab work in treating participants with rare tumors that have spread to other places in the body (advanced) or that does not respond to treatment (refractory). Cobimetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving cobimetinib and atezolizumab may work better in treating participants with advanced or refractory rare tumors.

PRIMARY OBJECTIVE:

I. To evaluate the efficacy of cobimetinib plus atezolizumab (COTEZO) in cohorts of advanced rare tumors using objective response rate (ORR) per Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1.

SECONDARY OBJECTIVES:

I. To determine progression-free survival (PFS) on COTEZO in cohorts of advanced rare tumors per RECIST v1.1 and immune-related (ir)RECIST.

II. To determine overall survival (OS) on COTEZO in cohorts of advanced rare tumors.

III. To determine disease control rate (DCR) and duration of response (DOR) on COTEZO in cohorts of advanced rare tumors per RECIST v1.1 and irRECIST.

IV. To determine objective response rate (ORR) per immune-related RECIST criteria.

V. To determine safety profile and adverse events encountered by patients with advanced rare tumors treated with COTEZO.

VI. To collect and bank tumor tissue and peripheral blood for future correlative analyses from patients with advanced rare tumors treated with COTEZO.

OUTLINE:

Participants receive cobimetinib orally (PO) once daily (QD) on days 1-21 and atezolizumab intravenously (IV) over 60 minutes on days 1 and 15. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, participants followed up every 3 months thereafter.

Cobimetinib and Atezolizumab in Treating Participants With Advanced or Refractory Rare Tumors

A Phase II, Open-Label, Single-Arm, Multi-Cohort, Proof-of-Principle Study to Investigate the Efficacy of Cobimetinib and Atezolizumab in Advanced Rare Tumors

ORR (partial response (PR) or complete response (CR) measured as per RECIST 1.1.

National Cancer Institute (NCI)

M.D. Anderson Cancer Center

Mouth and Tooth Diseases

Ear, Nose, and Throat Diseases

Respiratory Tract (Lung and Bronchial) Diseases

Infections

Nasopharyngeal Carcinoma

Recurrence

Carcinoma, Neuroendocrine

Mesothelioma

Mesothelioma, Malignant

Carcinoma, Merkel Cell

Lung Neoplasms

Disease Attributes

Nasopharyngeal Neoplasms

Pharyngeal Neoplasms

Otorhinolaryngologic Neoplasms

Head and Neck Neoplasms

Nasopharyngeal Diseases

Pharyngeal Diseases

Stomatognathic Diseases

Otorhinolaryngologic Diseases

Adenoma

Neoplasms, Mesothelial

Respiratory Tract Neoplasms

Thoracic Neoplasms

Pleural Neoplasms

Lung Diseases

Respiratory Tract Diseases

Communicable Diseases

Polyomavirus Infections

Virus Diseases

DNA Virus Infections

Tumor Virus Infections

Malignant Mesothelioma

Penile Cancer

Pancreatic Neuroendocrine Tumor

Merkel Cell Carcinoma

Bevacizumab

Immunoglobulin G

Endothelial Growth Factors

Mitogens

Immunoglobulins, Intravenous

Angiogenesis Inhibitors

Growth Inhibitors

Patients receive atezolizumab and bevacizumab IV over 60 minutes on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Laboratory Biomarker Analysis

Pharmacological Study

Daniel M Halperin, MD

This phase II trial studies how well atezolizumab and bevacizumab work in treating patients with rare solid tumors. Immunotherapy with monoclonal antibodies, such as atezolizumab and bevacizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

PRIMARY OBJECTIVES:

I. To evaluate the efficacy of atezolizumab when given in combination with bevacizumab (atezo bev).

SECONDARY OBJECTIVES:

I. To evaluate the efficacy of atezo bev. II. To evaluate the safety of atezo + bev.

EXPLORATORY BIOMARKER OBJECTIVES:

I. To identify biomarkers that are predictive of response to atezo bev (i.e., predictive biomarkers), are associated with progression to a more severe disease state (i.e., prognostic biomarkers), are associated with resistance to atezo bev, are associated with susceptibility to developing adverse events, can provide evidence of study treatment activity, or can increase the knowledge and understanding of disease biology.

OUTLINE:

Patients receive atezolizumab and bevacizumab intravenously (IV) over 60 minutes on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months.

Atezolizumab and Bevacizumab in Treating Patients With Rare Solid Tumors

A Phase II, Single-Arm Open-Label Study of the Combination of Atezolizumab and Bevacizumab in Rare Solid Tumors

The relationship between biomarkers in blood and tumor tissue and efficacy, safety, pharmacokinetics, immunogenicity, or other biomarker endpoints will be assessed. Summary statistics for biomarkers and their corresponding changes (or percent changes) from baseline will be tabulated by planned study day and dose in each tumor group. The time-course of biomarker measures will be investigated graphically. If there is indication of meaningful pattern over time, further analysis (eg, by linear mixed model) may be performed to characterize the relationship. Methods such as, but not limited to, logistic regression will be used to explore possible associations between biomarker measures and clinical outcomes.

Will be assessed by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (modified RECIST for pleural mesothelioma). Will be defined as a complete response or partial response on two consecutive occasions 4 weeks apart as determined by an independent radiologist. For each tumor group, the best response rate and its 95% exact confidence interval will be estimated using the Clopper and Pearson method. The combination treatment will be assessed by performing the independent binomial test comparing the best response rate versus the historical control for each tumor group. The Bayesian classification and information sharing method proposed by Lee and Chen may be applied.

Will be assessed by immune-modified RECIST

Will be assessed by RECIST 1.1 (modified RECIST for pleural mesothelioma). Will be estimated using Kaplan-Meier method. The log-rank test will be performed to test the difference in time-to-event distributions between patient groups. Cox proportional hazards model may be utilized to include multiple covariates in the time-to-event analysis.

Will be assessed by RECIST 1.1 (modified RECIST for pleural mesothelioma). For each tumor group the median duration of response and corresponding 2-sided 95% confidence interval will be reported.

Will be estimated using Kaplan-Meier method. The log-rank test will be performed to test the difference in time-to-event distributions between patient groups. Cox proportional hazards model may be utilized to include multiple covariates in the time-to-event analysis.

Will be graded according to National Cancer Institute Common Terminology Criteria for Adverse Events, version 4.0. Verbatim adverse events and severity will be graded according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0. The safety analyses will include all patients who received at least one dose of study treatment. Toxicity data will be summarized by frequency tables for each tumor type group. The association between the types and severity of toxicity and the tumor group will be evaluated. No formal statistical testing will be performed on these summary.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Genetics of Appendix Cancer Study	NCT05734430	Appendix Cancer Appendiceal Can... Appendiceal Neo... Appendiceal Muc... Appendiceal Ade... Appendiceal Car... Appendiceal Neo... Appendix Adenoc... Appendix Mucino... Appendix Tumor Appendix Cancer... Appendix NET Low-Grade Appen... High Grade Appe... High Grade Appe...	Genetic profili... Retrospective t...	18 Years -	Vanderbilt-Ingram Cancer Center
Cobimetinib and Atezolizumab in Treating Participants With Advanced or Refractory Rare Tumors	NCT03108131	Skin Squamous C... Appendix Adenoc... Rare Lesion Locally Advance... Locally Advance... Metastatic Mali... Metastatic Skin... Metastatic Smal... Rare Neoplastic... Refractory Mali... Stage IV Small ... Unresectable Ma...	Atezolizumab Cobimetinib	18 Years -	M.D. Anderson Cancer Center
Atezolizumab and Bevacizumab in Treating Patients With Rare Solid Tumors	NCT03074513	Appendix Adenoc... Human Papilloma... Human Papilloma... Human Papilloma... Human Papilloma... Neuroendocrine ... Pancreatic Neur... Recurrent Merke... Recurrent Nasop... Recurrent Perit... Recurrent Pleur... Stage III Merke... Stage III Nasop... Stage III Pleur... Stage IV Merkel... Stage IV Nasoph... Stage IV Pleura... Stage IVA Nasop... Stage IVB Nasop... Stage IVC Nasop... Vaginal Squamou...	Atezolizumab Bevacizumab Laboratory Biom... Pharmacological...	18 Years -	M.D. Anderson Cancer Center
Genetics of Appendix Cancer Study	NCT05734430	Appendix Cancer Appendiceal Can... Appendiceal Neo... Appendiceal Muc... Appendiceal Ade... Appendiceal Car... Appendiceal Neo... Appendix Adenoc... Appendix Mucino... Appendix Tumor Appendix Cancer... Appendix NET Low-Grade Appen... High Grade Appe... High Grade Appe...	Genetic profili... Retrospective t...	18 Years -	Vanderbilt-Ingram Cancer Center
Atezolizumab and Bevacizumab in Treating Patients With Rare Solid Tumors	NCT03074513	Appendix Adenoc... Human Papilloma... Human Papilloma... Human Papilloma... Human Papilloma... Neuroendocrine ... Pancreatic Neur... Recurrent Merke... Recurrent Nasop... Recurrent Perit... Recurrent Pleur... Stage III Merke... Stage III Nasop... Stage III Pleur... Stage IV Merkel... Stage IV Nasoph... Stage IV Pleura... Stage IVA Nasop... Stage IVB Nasop... Stage IVC Nasop... Vaginal Squamou...	Atezolizumab Bevacizumab Laboratory Biom... Pharmacological...	18 Years -	M.D. Anderson Cancer Center

Spots Global Cancer Trial Database for appendix adenocarcinoma