Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis

NCT00449748 | Completed Phase 2 Results DMC

• 1 other identifier: 2006-0759
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Systemic Mastocytosis

Keywords

Systemic Mastocytosis; RAD001

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Objective Response

  Efficacy reported as objective response. Objective response defined as change in serum tryptase level or bone marrow mast cell percentage.

  Monthly for first 3 months, then every 3 months

Study Arms (1)

RAD001

EXPERIMENTAL

Oral 10 mg daily for 30 days

Drug: RAD001 (Everolimus)

Interventions

RAD001 (Everolimus) DRUG

Oral RAD001 10 mg daily for 30 days

RAD001

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

The University of Texas M.D. Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• M.D. Anderson's website

MeSH Terms

Conditions

Mastocytosis, Systemic

Interventions

Everolimus

Condition Hierarchy (Ancestors)

Mastocytosis; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Mast Cell Activation Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Sirolimus; Macrolides; Lactones; Organic Chemicals

Results Point of Contact

• Title: Srdan Verstovsek M.D./Associate Professor
• Organization: The University of Texas M. D. Anderson Cancer Cent

eharriso@mdanderson.org

713-792-7305

Study Officials

• Srdan Verstovsek, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2007
• First Posted: March 21, 2007
• Study Start: April 1, 2007
• Primary Completion: October 1, 2009
• Study Completion: October 1, 2009
• Last Updated: May 1, 2025
• Results First Posted: April 29, 2011

Record last verified: 2025-04

Locations

US (1)