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Study Summary
No. 2006-0759:.......Leukemia......Srdan Verstovsek......Leukemia
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Study Summary Title
Study Summary
Number:
2006-0759
Study Title:Evaluation of RAD001 as Therapy for Patients with Systemic Mastocytosis
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Physician New Patient Referral
Name:Srdan VerstovsekPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:LeukemiaReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-792-7305
Contact us about clinical trials
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General Information
Disease Group:LeukemiaSupported By:N/A
Phase of Study:Phase IIReturn
Visit:
Initially monthly with review of constitutional symptoms and physical exam.
After completion of cycle 3 every three months. Then monthly phone assessment
of toxicity, review of required blood tests and assessment for the initiation
of a new cycle.
Treatment
Agents:
RAD001Home Care:Study medication will be administered by the patients themselves and will be
provided by Novartis. RAD001 should be taken by the patient in a fasting state
or with no more than a light fat-free meal.
Treatment Loc:Only at MDACC
Estimated
Length of Stay
in Houston:
N/A
Description/
Intervention:
The goal of this clinical research study is to see if RAD001 can help to
control the disease in patients with SM. The safety of this treatment will
also be studied.
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Study Objectives / Outcomes
Primary
· To assess the response rate of RAD001 in patients with Systemic Mastocytosis (SM).

Secondary
· To assess the safety of RAD001 in SM patients.
· To evaluate the time to progression and duration of response following treatment with RAD001.
  • To evaluate change in pertinent biological markers during the therapy with RAD001
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Study Status Information
Study Activation / Registration Date:04/11/2007
IRB Review and Approval Date:04/11/2007
Study Type:Phase Ii Or Phase I/Ii
Recruitment Status:Terminated
Projected Accrual:N/A
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Enrollment Eligibility
If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

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) ECOG performance status </= 2

5) Adequate liver function as shown by serum bilirubin </= 1.5 x upper limit of normal (ULN), and serum ALT </= 3 x ULN

6) PT/PTT/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 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

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Links
Registration Number: NCT00449748
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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