Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis

This study has been completed.
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
NCT00449748
First received: March 19, 2007
Last updated: August 1, 2012
Last verified: August 2012
  Purpose

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 Intervention Phase
Systemic Mastocytosis
Drug: RAD001 (Everolimus)
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evaluation of RAD001 as Therapy for Patients With Systemic Mastocytosis

Resource links provided by NLM:


Further study details as provided by M.D. Anderson Cancer Center:

Primary Outcome Measures:
  • Number of Participants With Objective Response [ Time Frame: Monthly for first 3 months, then every 3 months ] [ Designated as safety issue: No ]
    Efficacy reported as objective response. Objective response defined as change in serum tryptase level or bone marrow mast cell percentage.


Enrollment: 10
Study Start Date: April 2007
Study Completion Date: October 2009
Primary Completion Date: October 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RAD001
Oral 10 mg daily for 30 days
Drug: RAD001 (Everolimus)
Oral RAD001 10 mg daily for 30 days

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00449748

Locations
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
Investigators
Principal Investigator: Srdan Verstovsek, MD M.D. Anderson Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT00449748     History of Changes
Other Study ID Numbers: 2006-0759
Study First Received: March 19, 2007
Results First Received: April 4, 2011
Last Updated: August 1, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by M.D. Anderson Cancer Center:
Systemic Mastocytosis
RAD001

Additional relevant MeSH terms:
Mastocytosis
Mastocytosis, Systemic
Skin Diseases
Mastocytosis, Cutaneous
Pigmentation Disorders
Urticaria Pigmentosa
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on October 19, 2014