Trial Outcomes & Findings for Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis (NCT NCT00449748)
NCT ID: NCT00449748
Last Updated: 2025-05-01
Results Overview
Efficacy reported as objective response. Objective response defined as change in serum tryptase level or bone marrow mast cell percentage.
COMPLETED
PHASE2
10 participants
Monthly for first 3 months, then every 3 months
2025-05-01
Participant Flow
Recruitment Period 4/11/2007 to 4/15/2009. All patients registered at The University of Texas M.D. Anderson Cancer Center.
Participant milestones
| Measure |
RAD001
Oral RAD001 10 mg daily for 30 days
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis
Baseline characteristics by cohort
| Measure |
RAD001
n=10 Participants
Oral RAD001 10 mg daily for 30 days
|
|---|---|
|
Age, Continuous
|
56 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Monthly for first 3 months, then every 3 monthsPopulation: Analysis was per protocol.
Efficacy reported as objective response. Objective response defined as change in serum tryptase level or bone marrow mast cell percentage.
Outcome measures
| Measure |
RAD001
n=10 Participants
Oral RAD001 10 mg daily for 30 days
|
|---|---|
|
Number of Participants With Objective Response
|
0 Participants
|
Adverse Events
RAD001
Serious adverse events
| Measure |
RAD001
n=10 participants at risk
Oral RAD001 10 mg daily for 30 days
|
|---|---|
|
Gastrointestinal disorders
Nausea
|
10.0%
1/10 • Number of events 1 • 3 years 7 months
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
2/10 • Number of events 2 • 3 years 7 months
|
|
Gastrointestinal disorders
Diarrhea
|
10.0%
1/10 • Number of events 1 • 3 years 7 months
|
Other adverse events
| Measure |
RAD001
n=10 participants at risk
Oral RAD001 10 mg daily for 30 days
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
10.0%
1/10 • Number of events 1 • 3 years 7 months
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
10.0%
1/10 • Number of events 1 • 3 years 7 months
|
|
Blood and lymphatic system disorders
Neutropenia
|
30.0%
3/10 • Number of events 3 • 3 years 7 months
|
|
Gastrointestinal disorders
Diarrhea
|
40.0%
4/10 • Number of events 4 • 3 years 7 months
|
|
Gastrointestinal disorders
Mucositis
|
70.0%
7/10 • Number of events 7 • 3 years 7 months
|
|
General disorders
Fatigue
|
50.0%
5/10 • Number of events 5 • 3 years 7 months
|
|
General disorders
Fever
|
10.0%
1/10 • Number of events 1 • 3 years 7 months
|
|
Nervous system disorders
Headache
|
40.0%
4/10 • Number of events 4 • 3 years 7 months
|
|
Gastrointestinal disorders
Nausea
|
30.0%
3/10 • Number of events 3 • 3 years 7 months
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
1/10 • Number of events 1 • 3 years 7 months
|
|
Blood and lymphatic system disorders
Hyperglycemia
|
10.0%
1/10 • Number of events 1 • 3 years 7 months
|
|
Blood and lymphatic system disorders
Hyperlipidemia
|
30.0%
3/10 • Number of events 3 • 3 years 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
2/10 • Number of events 2 • 3 years 7 months
|
Additional Information
Srdan Verstovsek M.D./Associate Professor
The University of Texas M. D. Anderson Cancer Cent
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place