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Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00449748
Recruitment Status : Completed
First Posted : March 21, 2007
Results First Posted : April 29, 2011
Last Update Posted : August 7, 2012
Novartis Pharmaceuticals
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

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.

Condition or disease Intervention/treatment Phase
Systemic Mastocytosis Drug: RAD001 (Everolimus) Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Evaluation of RAD001 as Therapy for Patients With Systemic Mastocytosis
Study Start Date : April 2007
Actual Primary Completion Date : October 2009
Actual Study Completion Date : October 2009

Arm Intervention/treatment
Experimental: RAD001
Oral 10 mg daily for 30 days
Drug: RAD001 (Everolimus)
Oral RAD001 10 mg daily for 30 days

Primary Outcome Measures :
  1. Number of Participants With Objective Response [ Time Frame: Monthly for first 3 months, then every 3 months ]
    Efficacy reported as objective response. Objective response defined as change in serum tryptase level or bone marrow mast cell percentage.

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

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

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 identifier (NCT number): NCT00449748

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United States, Texas
The University of Texas M.D. Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Novartis Pharmaceuticals
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Principal Investigator: Srdan Verstovsek, MD M.D. Anderson Cancer Center

Additional Information:
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Responsible Party: M.D. Anderson Cancer Center Identifier: NCT00449748     History of Changes
Other Study ID Numbers: 2006-0759
First Posted: March 21, 2007    Key Record Dates
Results First Posted: April 29, 2011
Last Update Posted: August 7, 2012
Last Verified: August 2012
Keywords provided by M.D. Anderson Cancer Center:
Systemic Mastocytosis
Additional relevant MeSH terms:
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Mastocytosis, Systemic
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Skin Diseases
Immune Complex Diseases
Immune System Diseases
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents