Status:
COMPLETED
Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis
Lead Sponsor:
M.D. Anderson Cancer Center
Collaborating Sponsors:
Novartis Pharmaceuticals
Conditions:
Systemic Mastocytosis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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.
Detailed Description
RAD001 is designed to stop cancer cells from multiplying. It may also stop the growth of new blood vessels that help tumor growth, which may cause the tumor cells to die. Before you can start treatme...
Eligibility Criteria
Inclusion
- Patients with SM; including mast cell leukemia.
- Age \>/= 18 years
- Minimum of two weeks since any major surgery or completion of radiation.
- Eastern Cooperative Oncology Group (ECOG) performance status \</= 2
- Adequate liver function as shown by serum bilirubin \</= 1.5 x upper limit of normal (ULN), and serum Alanine transaminase (ALT) \</= 3 x ULN
- Prothrombin Time (PT)/Partial thromboplastin time (PTT)/International normalized ratio (INR) within normal institutional limits
- Signed informed consent
Exclusion
- Treatment with any conventional (specifically, interferon or cladribine) or investigational medicine for SM within the preceding 4 weeks
- Chronic treatment with systemic steroids or another immunosuppressive agent
- 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.
- 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)
- A known history of Human immunodeficiency virus (HIV) seropositivity
- 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)
- Patients with a bleeding diathesis or on oral anti-vitamin K medication
- 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)
- Patients who have received prior treatment with an mTOR inhibitor (e.g., sirolimus, temsirolimus)
- Patients with a known hypersensitivity to RAD001 (everolimus) or other rapamycins (sirolimus, temsirolimus) or to its excipients
- Patients unwilling to or unable to comply with the protocol
Key Trial Info
Start Date :
April 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2009
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT00449748
Start Date
April 1 2007
End Date
October 1 2009
Last Update
May 1 2025
Active Locations (1)
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1
The University of Texas M.D. Anderson Cancer Center
Houston, Texas, United States, 77030