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Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis

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ClinicalTrials.gov Identifier: NCT00449748
Recruitment Status : Completed
First Posted : March 21, 2007
Results First Posted : April 29, 2011
Last Update Posted : August 7, 2012
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Systemic Mastocytosis
Intervention Drug: RAD001 (Everolimus)
Enrollment 10
Recruitment Details Recruitment Period 4/11/2007 to 4/15/2009. All patients registered at The University of Texas M.D. Anderson Cancer Center.
Pre-assignment Details  
Arm/Group Title RAD001
Hide Arm/Group Description Oral RAD001 10 mg daily for 30 days
Period Title: Overall Study
Started 10
Completed 10
Not Completed 0
Arm/Group Title RAD001
Hide Arm/Group Description Oral RAD001 10 mg daily for 30 days
Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 10 participants
56
(43 to 75)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
6
  60.0%
Male
4
  40.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants
10
1.Primary Outcome
Title Number of Participants With Objective Response
Hide Description Efficacy reported as objective response. Objective response defined as change in serum tryptase level or bone marrow mast cell percentage.
Time Frame Monthly for first 3 months, then every 3 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was per protocol.
Arm/Group Title RAD001
Hide Arm/Group Description:
Oral RAD001 10 mg daily for 30 days
Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: Participants
0
Time Frame 3 years 7 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title RAD001
Hide Arm/Group Description Oral RAD001 10 mg daily for 30 days
All-Cause Mortality
RAD001
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
RAD001
Affected / at Risk (%) # Events
Total   2/10 (20.00%)    
Gastrointestinal disorders   
Nausea  1  1/10 (10.00%)  1
Vomiting  1  2/10 (20.00%)  2
Diarrhea  1  1/10 (10.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
RAD001
Affected / at Risk (%) # Events
Total   10/10 (100.00%)    
Blood and lymphatic system disorders   
Anemia  1  1/10 (10.00%)  1
Thrombocytopenia  1  1/10 (10.00%)  1
Neutropenia  1  3/10 (30.00%)  3
Hyperglycemia  1  1/10 (10.00%)  1
Hyperlipidemia  1  3/10 (30.00%)  3
Gastrointestinal disorders   
Diarrhea  1  4/10 (40.00%)  4
Mucositis  1  7/10 (70.00%)  7
Nausea  1  3/10 (30.00%)  3
Vomiting  1  1/10 (10.00%)  1
General disorders   
Fatigue  1  5/10 (50.00%)  5
Fever  1  1/10 (10.00%)  1
Nervous system disorders   
Headache  1  4/10 (40.00%)  4
Respiratory, thoracic and mediastinal disorders   
Cough  1  2/10 (20.00%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Srdan Verstovsek M.D./Associate Professor
Organization: The University of Texas M. D. Anderson Cancer Cent
Phone: 713-792-7305
Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT00449748     History of Changes
Other Study ID Numbers: 2006-0759
First Submitted: March 19, 2007
First Posted: March 21, 2007
Results First Submitted: April 4, 2011
Results First Posted: April 29, 2011
Last Update Posted: August 7, 2012