Neoplasms

All Conditions

Angiomyolipoma

Neoplasms, Adipose Tissue

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Perivascular Epithelioid Cell Neoplasms

Antineoplastic Agents

All Drugs and Chemicals

Anti-Infective Agents

Everolimus

Sirolimus

Temsirolimus

MTOR Inhibitors

Protein Kinase Inhibitors

Enzyme Inhibitors

Immunosuppressive Agents

Immunologic Factors

Patients will take 10 mg (1 tablet) of everolimus each day for 4 months

Robert G Uzzo, MD

The purpose of this research study is to see if oral everolimus is tolerable and effective in the treatment of sporadic Angiomyolipomas (AMLs). AMLs are the most common non-cancerous tumor of the kidney. They are composed of blood vessels, muscle cells and fat cells.Everolimus is already an approved drug for several other diseases like kidney cancer, but is being studied now specifically to see if it is helpful for people with AML.

Primary Objective

1. To evaluate the efficacy and tolerability of everolimus in reducing tumor volume in sporadic AMLs as measured by dynamic contrast enhanced magnetic resonance imaging (DCE MRI), in patients who might otherwise be considered for active surgical or percutaneous intervention.

Secondary Objectives

1. To evaluate health-related quality of life (HRQoL) in subjects treated with everolimus for sporadic AMLs.
2. To assess the growth kinetics of sporadic AMLs in patients who have been treated with everolimus as part of the study and demonstrate an objective response as well as those who have been treated with everolimus during the study with a suboptimal or no response.
3. To measure the rate of surgical or percutaneous (embolization) intervention at 1 year from day 1 of study.
4. To assess the safety and tolerability of everolimus in patients with sporadic AML.

Sporadic Angiomyolipomas (AMLs) Growth Kinetics While on Everolimus

A Phase II Study of Sporadic Angiomyolipomas (AMLs) Growth Kinetics While on Everolimus Therapy

Measuring the efficacy and tolerability of everolimus by measuring the number of patients with tumor volume reduction greater than 25% in sporadic AMLs as measured by DCE MRI

Number of participants with treatment-related adverse events requiring dose reduction or study withdrawal as assessed by Common Toxicity Criteria for Adverse Effects (CTCAE v4.0)

Novartis Pharmaceuticals

Fox Chase Cancer Center

Patients will take 10 mg (1 tablet) of everolimus each day for 4 months

Everolimus: 10 mg tablets

Treatment (Everolimus)

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

AML volume

Lack of tissue to correlate response to molecular markers of aberrant mTOR pathways. Lack for serum everolimus levels. Clinical significance of 25% volume reduction unknown. Patient withdrawals and incomplete protocol imaging.

Robert Uzzo, M.D.

Some participants experienced adverse events and were withdrawn from the study, and therefore tumor volume reduction was not measured in these patients.

Number of Patients With Tumor Volume Reduction Greater Than 25%

Safety and Tolerability of Everolimus in Patients With Sporadic AML

Overall Study

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Sporadic Angiomyolipomas (AMLs) Growth Kinetics While on Everolimus	NCT02539459	Sporadic Angiom...	Everolimus	18 Years -	Fox Chase Cancer Center
Sporadic Angiomyolipomas (AMLs) Growth Kinetics While on Everolimus	NCT02539459	Sporadic Angiom...	Everolimus	18 Years -	Fox Chase Cancer Center
Sporadic Angiomyolipomas (AMLs) Growth Kinetics While on Everolimus	NCT02539459	Sporadic Angiom...	Everolimus	18 Years -	Fox Chase Cancer Center

Spots Global Cancer Trial Database for sporadic angiomyolipomas (amls)