Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia
Condition: Waldenstrom's Macroglobulinemia
MeSH terms: Waldenstrom Macroglobulinemia
Intervention: RAD001
Intervention description: Taken orally once a day
Intervention MeSH terms: Everolimus
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 33.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: everolimus; RAD001; WM

Summary:

      The purpose of this research study is to determine the safety of RAD001(Everolimus) and the
      highest dose of this drug that can be given to people safely. RAD001(Everolimus) is a drug
      that works by preventing cells in the body from growing and dividing. Information from basic
      and Phase I clinical research studies suggests that RAD001 also may help to prevent tumor
      growth in people with relapsed or refractory lymphoma.
    

Eligibility criteria:

        Inclusion Criteria:

          -  18 years of age or older

          -  Adequate liver and renal function as outlined in the protocol

          -  Fasting serum cholesterol 300mg/dl or less OR 7.75mmol/L or less AND fasting
             triglycerides 2.5 x institutional ULN or less.

          -  Clinicopathological diagnosis of Waldenstrom's macroglobulinemia as defined by
             consensus panel of the Second International Workshop on Waldenstrom's
             macroglobulinemia

          -  No previous therapy for WM

          -  Measurable disease, defined as presence of immunoglobulin M (IgM) paraprotein with a
             minimum IgM level of 2 times the upper limit of each institution's normal value or
             greater is required

          -  ECOG Performance status of 0-2

          -  Patients must have a life expectancy of at least 3 months

          -  Baseline platelet and absolute neutrophil as outlined in the protocol

          -  INR and PTT 1.5 x normalized ratio or less

          -  A male subject agrees to use an acceptable method for contraception for the duration
             of study and for 8 weeks after the last dose of the study drug

          -  Female subject either post-menopausal or surgically sterilized or willing to use
             acceptable methods of birth control for the duration of the study and for 8 weeks
             after the last dose of study drug

        Exclusion Criteria:

          -  Patients experiencing symptomatic hyperviscosity and requiring plasmapheresis. This
             includes any patient who, in the judgement of the investigator requires urgent
             response and will not be eligible. These patients have hyperviscosity which includes
             serum IgM levels of 5000 mg/dL or greater. Symptoms may include nosebleeds, visual
             complications, fatigue, headaches, confusion, etc.

          -  Patients, who have had a major surgery or significant traumatic injury within 4 weeks
             of start of study drug, patients who have not recovered from the side effects of any
             major surgery or patients that may require major surgery during the course of the
             study.

          -  Patients receiving chronic, systemic treatment with corticosteroids or another
             immunosuppressive agent. Topical or inhaled corticosteroids are allowed.

          -  Patients should not receive any immunization with attenuated live vaccines within one
             week of study entry or during study period.

          -  Patients who have had any severe and/or uncontrolled medical conditions or other
             conditions that would affect their participation in the study.

          -  Impairment of gastrointestinal function or gastrointestinal disease that may
             significantly alter the absorption of RAD001.

          -  Female patients that are pregnant or breast feeding, or adults of reproductive
             potential who are not using effective birth control methods.

          -  Patients with known hypersensitivity to RAD001 or other rapamycins or to its
             excipients

          -  Patients with other malignancies within the past 3 years except for adequately treated
             carcinoma of the cervix or basal or squamous cell of the skin

          -  Patients with known history of HIV seropositivity

          -  History of noncompliance to medical regimens

          -  Patients unwilling to or unable to comply with the protocol