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Sporadic Angiomyolipomas (AMLs) Growth Kinetics While on Everolimus (SAGE)

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ClinicalTrials.gov Identifier: NCT02539459
Recruitment Status : Terminated (Extreme toxicity, met toxicity stopping rules)
First Posted : September 3, 2015
Last Update Posted : September 17, 2018
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Fox Chase Cancer Center

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.

Condition or disease Intervention/treatment Phase
Sporadic Angiomyolipomas (AMLs) Drug: Everolimus Phase 2

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Sporadic Angiomyolipomas (AMLs) Growth Kinetics While on Everolimus Therapy
Actual Study Start Date : September 23, 2015
Actual Primary Completion Date : August 20, 2018
Actual Study Completion Date : August 20, 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Treatment (everolimus)
Patients will take 10 mg (1 tablet) of everolimus each day for 4 months
Drug: Everolimus
10 mg tablets




Primary Outcome Measures :
  1. The efficacy and tolerability of everolimus in reducing tumor volume in sporadic AMLs as measured by DCE MRI [ Time Frame: 1 year from start of treatment ]

Secondary Outcome Measures :
  1. Health-related quality of life (HRQoL) [ Time Frame: 4-6 months ]
  2. Rate of surgical or percutaneous (embolization) intervention [ Time Frame: 1 year from Day 1 ]
  3. Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE v4.0) [ Time Frame: 1 year from start of treatment ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
  • Unable or unwilling to discontinue use of prohibited medications
  • Concurrent therapy given to treat cancer including treatment with an investigational agent or concurrent participation in another clinical trial involving anti-cancer investigational drug.
  • Administration of any investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
  • Known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to everolimus
  • Prior or current use of systemic anti-vascular endothelial growth factor (VEGF) inhibitors, cytokines or mechanistic target of rapamycin (mTOR) inhibitors (e.g. interferon, interleukin 2).
  • Pregnant or nursing (lactating) women
  • Women of child-bearing potential (WOCBP) must use highly effective methods of contraception during the study and 8 weeks after.
  • Unable to obtain a contrast (gadolinium) based DCE MRI, including include patients with pacemakers, automatic implantable cardioverter/defibrillator (AICDs), non MRI compatible metallic implants or eGFR <30.
  • Must not have received immunization with an attenuated live vaccine within seven days prior to registration nor have plans to receive such vaccination while on protocol treatment
  • Must not be taking, nor plan to take while on protocol treatment, strong cytochrome P450 3A4 (CYP3A4) inhibitors, (e.g. ketoconazole, itraconazole, voriconazole, posaconazole, fluvoxamine, nefazodone, nelfinavir, ritonavir) and/or strong CYP3A4 inducers (e.g. phenytoin, rifampin, rifabutin) within 14 days prior to randomization.
  • History of another primary malignancy, with the exceptions of: non-melanoma skin cancer, and carcinoma in situ of the cervix, uteri, or breast from which the patient has been disease free for >= 3 years
  • Childs-Pugh A-C liver disease (Appendix II)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02539459


Locations
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United States, Connecticut
Yale School of Medicine
New Haven, Connecticut, United States
United States, Massachusetts
Dana Farber Cancer Institute
Boston, Massachusetts, United States
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States
United States, New York
Memorial Sloan Kettering
New York, New York, United States
United States, North Carolina
Duke University Health System
Durham, North Carolina, United States
United States, Pennsylvania
Fox Chase Cancer Center
Philadelphia, Pennsylvania, United States, 19111
University of Pennsylvania
Philadelphia, Pennsylvania, United States
United States, Texas
M D Anderson Cancer Center
Houston, Texas, United States
Sponsors and Collaborators
Fox Chase Cancer Center
Novartis Pharmaceuticals

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Responsible Party: Fox Chase Cancer Center
ClinicalTrials.gov Identifier: NCT02539459     History of Changes
Other Study ID Numbers: GU-070
First Posted: September 3, 2015    Key Record Dates
Last Update Posted: September 17, 2018
Last Verified: September 2018
Additional relevant MeSH terms:
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Angiomyolipoma
Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Perivascular Epithelioid Cell Neoplasms
Sirolimus
Everolimus
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents