Neoplasms

All Conditions

Eye Diseases

Skin and Connective Tissue Diseases

Rare Diseases

Osteosarcoma

Sarcoma

Retinoblastoma

Chondrosarcoma

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Soft Tissue Sarcoma

Subjects will be treated with abemaciclib 200 mg twice daily by mouth.

Abemaciclib 200 mg will be taken by mouth twice daily.

Abemaciclib

Medical College of Wisconsin Cancer Center Clinical Trials Office

Mark Agulnik, MD

Steven Attia, DO

John Rieth, MD

Michele Landeau, CCRP

Brian Van Tine, MD

John Charlson, MD

This is a single-arm, phase II study that will enroll a total of 45 subjects. All subjects will have a confirmed diagnosis of metastatic or unresectable soft tissue sarcoma or bone sarcoma. All subjects must have intact Rb, identified at the time of screening, by immunohistochemistry testing of submitted tumor specimen. Subjects will receive Abemaciclib 200 mg twice daily until progression or discontinuation criteria are met.

Population to be studied:

Since abemaciclib is already being studied in dedifferentiated liposarcoma patients, patients with this sarcoma subtype will not be enrolled in the current study. This exploratory study will enroll patients with all other types of soft tissue sarcoma, in addition to patients with several bone sarcomas. We are testing the hypothesis that biomarkers of Cyclin D1 - CDK4/6 - Rb pathway activation will identify patients with a rare, heterogeneous cancer who are most likely to benefit from cyclin-dependent kinase 4/6 (CDK4/6) inhibition with abemaciclib.

There will be three cohorts of patients, intended to ensure representation of subjects with rare bone sarcomas - chondrosarcoma and osteosarcoma - that have relatively frequent occurrence of abnormalities in the Cyclin D1 - CDK4/6 - Rb pathway, in addition to soft tissue sarcoma.

Cohort 1 - Conventional chondrosarcoma, 9-12 patients;

Cohort 2 - Osteosarcoma, Dedifferentiated chondrosarcoma, 9-12 patients;

Cohort 3 - Soft tissue sarcoma (except WD/DD Liposarcoma), 22-26 patients

Abemaciclib for Bone and Soft Tissue Sarcoma With Cyclin-Dependent Kinase (CDK) Pathway Alteration

Abemaciclib for Treatment of Advanced Bone and Soft Tissue Sarcoma Identified as Having CDK Pathway Alteration

This outcome measure is the number of subjects whose disease has not progressed as defined by RECIST 1.1 criteria.

This outcome measure is the number of subjects expiring from any cause.

Medical College of Wisconsin

Associate Professor

Diseases and Abnormalities at or Before Birth

Neoplasms, Neuroepithelial

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Retinal Neoplasms

Eye Neoplasms

Eye Diseases, Hereditary

Retinal Diseases

Neuroepithelioma

Jesse L Berry, MD

Jesse Berry, MD

Retinoblastoma (RB) is a primary eye cancer that forms in the back of the eye of infants and toddlers. Traditionally, RB is diagnosed without a biopsy; tumor can only be studied once an eye has been surgically removed.

Given this limitation, we use aqueous humor (AH), the clear fluid in the front of the eye to detect specific markers, or information, that comes from the tumor itself.

Retinoblastoma (RB) is a primary intraocular malignancy that forms in the retina of infants and toddlers. Traditionally, RB is diagnosed without tissue as direct tumor biopsy is prohibited due to risk of extraocular spread; tissue can only be obtained once an eye has been surgically removed.

Given this limitation, there are no eye-specific molecular biomarkers in current clinical practice for RB. The lack of in vivo molecular data without removing the eye limits our ability to prognosticate clinical outcomes and develop personalized treatment plans. It also limits our understanding of intratumoral dynamics throughout therapy.

The aqueous humor (AH) is a high-yield source of tumor-derived nucleic acid that can be utilized as a liquid biopsy in eyes with retinoblastoma. Detection of biomarkers from the AH may be used to prognosticate the likelihood of eye salvage and in the future may facilitate targeted, patient-centered therapies based on molecular biomarkers.

OBJECTIVES:

1. Systematically and prospectively record accurate and complete data regarding the clinical presentation, treatment, and outcomes of patients diagnosed with RB.
2. Collect, bank, preserve, and analyze biomaterials including AH, blood and saliva from patients with RB.
3. Correlate phenotypic data with genotypic findings from human biomaterials in retinoblastoma patients to identify clinically relevant biomarkers

RB Liquid Biopsy Biorepository

Retinoblastoma Patient Clinical Database, Liquid Biopsy and Tissue Biorepository

De-identified data will be available to other researchers via NIH GDS/dbGAP controlled databases.

We aim to evaluate whether any identifiable molecular biomarkers (e.g. chromosomal alterations or tumor fraction) in the aqueous or blood can predict ocular salvage, or the likelihood of saving the eye with therapy

We aim to identify both somatic and germline RB1 gene mutations in the AH

We aim to evaluate whether any identifiable molecular biomarkers in the aqueous or blood can predict the presence of high risk histopathologic features that are known to increase risk of metastatic disease

We aim to evaluate whether any identifiable molecular biomarkers in the aqueous or blood are associated with the presence of type of seeding

Children's Hospital Los Angeles

Associate Professor of Ophthalmology

Breast Neoplasms

Triple Negative Breast Neoplasms

Breast Diseases

Skin Diseases

Agnieszka Witkiewicz, MD

Gordon Schwartz, MD

Matias Valsecchi, MD

A. Kathleen McClendon, PhD

Adam Berger, MD

Sunny Lee, MD

Barbara Cavanaugh, MD

Frederick Fellin, MD

Allison Zibelli, MD

Tiffany Avery, MD

Rebecca Jaslow, MD

Erik Knudsen, PhD

This is a prospective observational study aimed to validate biomarkers that predict response.

This is a prospective observational cohort study, with no control group and no direct experimental intervention, where the patient's pathological material will be used to examine the expression of molecular biomarkers believed to predict chemotherapy response. Patients with locally advanced triple negative breast cancer, who are candidates for neoadjuvant chemotherapy, will be enrolled in this clinical study. Patients will receive standard of care, FDA-approved and clinically validated chemotherapy regimens. The biopsy specimens taken during the diagnostic phase (pre-treatment specimen) will be used for specific biomarker evaluation. Data obtained from this pre-treatment phase will be compared with the surgical specimens (post-treatment phase) obtained after receiving neoadjuvant chemotherapy. The primary endpoint is to assess the ability of specific biomarkers to predict treatment response, and thus identify those patients who will achieve benefit from such treatment.

Tumor Suppressor Status as a Predictor of Chemotherapy Response in Triple Negative Breast Cancer

Tumor Suppressor Status as a Predictor of Chemotherapy Response in Triple Negative Breast Cancer.

The primary endpoint is to assess the impact of histological RB-status on pathological complete response (pCR). pCR is a well validated surrogate of chemotherapy sensitivity and allows the identification of a group of patients with excellent prognosis.

1. Correlation between RB and PTEN tumor suppressor status and other biomarkers with pCR
2. Correlation between RB-status, RB and PTEN status and other biomarkers with recurrence free survival (RFS) and overall survival (OS)

Gene expression profiling of biopsy specimens to determine utility of an RB molecular test.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Abemaciclib for Bone and Soft Tissue Sarcoma With Cyclin-Dependent Kinase (CDK) Pathway Alteration	NCT04040205	Chondrosarcoma Osteosarcoma Soft Tissue Sar...	Abemaciclib	18 Years -	Medical College of Wisconsin
RB Liquid Biopsy Biorepository	NCT04959097	Retinoblastoma Retinoblastoma ... Retinoblastoma ...		0 Years - 18 Years	Children's Hospital Los Angeles
Tumor Suppressor Status as a Predictor of Chemotherapy Response in Triple Negative Breast Cancer	NCT01514565	Breast Cancer		18 Years -	Thomas Jefferson University

Spots Global Cancer Trial Database for rb

Every month we try and update this database with for rb cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for rb

Every month we try and update this database with for rb cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for rb cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.