Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis
Condition: Systemic Mastocytosis
MeSH terms: Mastocytosis; Mastocytosis, Systemic
Intervention: RAD001 (Everolimus)
Intervention description: Oral RAD001 10 mg daily for 30 days
Intervention MeSH terms: Everolimus
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 10.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Systemic Mastocytosis; RAD001

Summary:

      The goal of this clinical research study is to see if RAD001 can help to control the disease
      in patients with systemic mastocytosis (SM). The safety of this treatment will also be
      studied.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Patients with SM; including mast cell leukemia.

          2. Age >/= 18 years

          3. Minimum of two weeks since any major surgery or completion of radiation.

          4. Eastern Cooperative Oncology Group (ECOG) performance status </= 2

          5. Adequate liver function as shown by serum bilirubin </= 1.5 x upper limit of normal
             (ULN), and serum Alanine transaminase (ALT) </= 3 x ULN

          6. Prothrombin Time (PT)/Partial thromboplastin time (PTT)/International normalized ratio
             (INR) within normal institutional limits

          7. Signed informed consent

        Exclusion Criteria:

          1. Treatment with any conventional (specifically, interferon or cladribine) or
             investigational medicine for SM within the preceding 4 weeks

          2. Chronic treatment with systemic steroids or another immunosuppressive agent

          3. Other malignancies within the past 3 years except for adequately treated carcinoma of
             the cervix or basal or squamous cell carcinomas of the skin, unless patient has
             SM-associated clonal hematologic disease that does not require therapy, as judged by
             treating physician and approved by principal investigator.

          4. Other concurrent severe and/or uncontrolled medical disease which could compromise
             participation in the study as judged by the Principal Investigator (i.e., severely
             impaired lung function, uncontrolled diabetes, uncontrolled hypertension, severe
             infection, severe malnutrition, unstable angina, or congestive heart failure - New
             York Heart Association Class III or IV, ventricular arrhythmias, active ischemic heart
             disease, myocardial infarction within six months, chronic liver or renal disease,
             active upper GI tract ulceration)

          5. A known history of Human immunodeficiency virus (HIV) seropositivity

          6. Impairment of gastrointestinal function or gastrointestinal disease that may
             significantly alter the absorption of RAD001 as judged by the Principal Investigator
             (e.g., ulcerative disease; uncontrolled nausea, vomiting or diarrhea; malabsorption
             syndrome or small bowel resection)

          7. Patients with a bleeding diathesis or on oral anti-vitamin K medication

          8. Women who are pregnant or breast feeding, or women/men able to conceive and unwilling
             to practice an effective method of birth control (women of childbearing potential must
             have a negative urine or serum pregnancy test within 48 hours prior to administration
             of RAD001; protocol definition of post-menopausal women is: 12 months of natural
             (spontaneous) amenorrhea or 6 months of spontaneous amenorrhea with serum FSH levels >
             40 mIU/m or 6 weeks post-surgical bilateral oophorectomy with or without hysterectomy)

          9. Patients who have received prior treatment with an mTOR inhibitor (e.g., sirolimus,
             temsirolimus)

         10. Patients with a known hypersensitivity to RAD001 (everolimus) or other rapamycins
             (sirolimus, temsirolimus) or to its excipients

         11. Patients unwilling to or unable to comply with the protocol