NCT02539459

Brief Summary

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2015

Typical duration for phase_2

Geographic Reach
1 country

8 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 20, 2015

Completed
14 days until next milestone

First Posted

Study publicly available on registry

September 3, 2015

Completed
20 days until next milestone

Study Start

First participant enrolled

September 23, 2015

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 20, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 20, 2018

Completed
2.5 years until next milestone

Results Posted

Study results publicly available

February 17, 2021

Completed
Last Updated

December 15, 2022

Status Verified

December 1, 2022

Enrollment Period

2.9 years

First QC Date

August 20, 2015

Results QC Date

January 27, 2021

Last Update Submit

December 13, 2022

Conditions

Sporadic Angiomyolipomas (AMLs)

Outcome Measures

Primary Outcomes (1)

  • Number of Patients With Tumor Volume Reduction Greater Than 25%

    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

    12 months

Secondary Outcomes (1)

  • Safety and Tolerability of Everolimus in Patients With Sporadic AML

    12 months

Study Arms (1)

Treatment (everolimus)

EXPERIMENTAL

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

Drug: Everolimus

Interventions

EverolimusDRUG

10 mg tablets

Treatment (everolimus)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Must have a diagnosis of renal AML \> 3 cm confirmed on pre-enrollment Dynamic Contrast Enhanced MRI (DCE-MRI)
  • Must not have received any prior treatment for AML
  • Eastern Cooperative Oncology Group (ECOG) Performance Status 0 or 1
  • Absolute neutrophil count \>= 1,500/ microliter (mcL)
  • Hemoglobin \>=10 g/dL
  • Platelets \>= 100,000/ mcL
  • international normalized ratio (INR) \<= 1.2 X Upper limit Normal (ULN)
  • activated partial thromboplastin time (aPTT) \<= 1.2 X ULN
  • aspartate aminotransferase (AST) / alanine transaminase (ALT) \<= 2.5 X ULN
  • Total bilirubin \<= 2.0mg/dL
  • Renal Function epidermal growth factor receptor (eGFR) \>= 30 mL/min via calculated creatinine clearance
  • Fasting serum cholesterol \<= 300 mg/dL OR \<= 7.75 mmol/L AND fasting triglycerides \<= 2.5x ULN.

You may not qualify if:

  • History of tuberous sclerosis, LAM or any active malignancy
  • Treatment with any other investigational agents for any other disease
  • Clinically significant gastrointestinal abnormalities that may increase the risk for gastrointestinal bleeding or affect absorption of investigational product
  • Active diarrhea of any grade.
  • History of human immunodeficiency virus (HIV) infection, hepatitis B or C (screening for all three is mandatory prior to study); prior hepatitis C infection
  • Presence of any active or ongoing infection.
  • Any known uncontrolled underlying pulmonary disease by history, physical exam or if applicable pulmonary function test (PFTs)
  • History of certain cardiovascular conditions within the past 6 months
  • History of Class III or IV congestive heart failure, as defined by the New York Heart Association Classification of Congestive Heart Failure.
  • History of cerebrovascular accident including transient ischemic attack (TIA), pulmonary embolism or untreated deep venous thrombosis (DVT) within the past 6 months.
  • Corrected QT interval (QTc) \> 480 milliseconds
  • Poorly controlled hypertension, defined as systolic blood pressure (SBP) of \>= 140 millimeters of mercury(mmHg) or diastolic blood pressure (DBP) of \>= 90 mmHg.
  • Evidence of active bleeding or bleeding diathesis
  • Uncontrolled diabetes mellitus (defined by a Hgb A1c \>8) obtained within 14 days prior to registration. Optimal glucose control (Hgb A1c \<= 8) must be achieved before registration and monitored during protocol treatment
  • Any serious and/or unstable pre-existing medical, psychiatric, or other condition that could interfere with subject's safety, provision of informed consent, or compliance to study procedures.
  • +12 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Yale School of Medicine

New Haven, Connecticut, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Location

Mayo Clinic

Rochester, Minnesota, United States

Location

Memorial Sloan Kettering

New York, New York, United States

Location

Duke University Health System

Durham, North Carolina, United States

Location

Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Location

M D Anderson Cancer Center

Houston, Texas, United States

Location

Related Publications (1)

  • Geynisman DM, Kadow BT, Shuch BM, Boorjian SA, Matin SF, Rampersaud E, Milestone BN, Plimack ER, Zibelman MR, Kutikov A, Smaldone MC, Chen DY, Viterbo R, Joshi S, Greenberg RE, Malizzia L, McGowan T, Ross EA, Uzzo RG. Sporadic Angiomyolipomas Growth Kinetics While on Everolimus: Results of a Phase II Trial. J Urol. 2020 Sep;204(3):531-537. doi: 10.1097/JU.0000000000001065. Epub 2020 Apr 6.

    PMID: 32250730RESULT

MeSH Terms

Interventions

Everolimus

Intervention Hierarchy (Ancestors)

SirolimusMacrolidesLactonesOrganic Chemicals

Limitations and Caveats

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.

Results Point of Contact

Title
Robert Uzzo, M.D.
Organization
Fox Chase Cancer Center
robert.uzzo@fccc.edu
215-728-3096

Study Officials

  • Robert G Uzzo, MD

    PI

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 20, 2015

First Posted

September 3, 2015

Study Start

September 23, 2015

Primary Completion

August 20, 2018

Study Completion

August 20, 2018

Last Updated

December 15, 2022

Results First Posted

February 17, 2021

Record last verified: 2022-12

Locations

US(8)