Trial Outcomes & Findings for Sporadic Angiomyolipomas (AMLs) Growth Kinetics While on Everolimus (NCT NCT02539459)
NCT ID: NCT02539459
Last Updated: 2022-12-15
Results Overview
Measuring the efficacy and tolerability of everolimus by measuring the number of patients with tumor volume reduction greater than 25% in sporadic AMLs as measured by DCE MRI
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
12 months
Results posted on
2022-12-15
Participant Flow
Participant milestones
| Measure |
Treatment (Everolimus)
Patients will take 10 mg (1 tablet) of everolimus each day for 4 months
Everolimus: 10 mg tablets
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
18
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Sporadic Angiomyolipomas (AMLs) Growth Kinetics While on Everolimus
Baseline characteristics by cohort
| Measure |
Treatment (Everolimus)
n=20 Participants
Patients will take 10 mg (1 tablet) of everolimus each day for 4 months
Everolimus: 10 mg tablets
|
|---|---|
|
Age, Continuous
|
68 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
19 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=5 Participants
|
|
BMI
|
29.4 kg/m^2
n=5 Participants
|
|
AML volume
|
47.3 cc
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: Some participants experienced adverse events and were withdrawn from the study, and therefore tumor volume reduction was not measured in these patients.
Measuring the efficacy and tolerability of everolimus by measuring the number of patients with tumor volume reduction greater than 25% in sporadic AMLs as measured by DCE MRI
Outcome measures
| Measure |
Treatment (Everolimus)
n=10 Participants
Patients will take 10 mg (1 tablet) of everolimus each day for 4 months
Everolimus: 10 mg tablets
|
|---|---|
|
Number of Patients With Tumor Volume Reduction Greater Than 25%
|
5 Participants
|
SECONDARY outcome
Timeframe: 12 monthsNumber of participants with treatment-related adverse events requiring dose reduction or study withdrawal as assessed by Common Toxicity Criteria for Adverse Effects (CTCAE v4.0)
Outcome measures
| Measure |
Treatment (Everolimus)
n=20 Participants
Patients will take 10 mg (1 tablet) of everolimus each day for 4 months
Everolimus: 10 mg tablets
|
|---|---|
|
Safety and Tolerability of Everolimus in Patients With Sporadic AML
|
10 Participants
|
Adverse Events
Treatment (Everolimus)
Serious events: 3 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Everolimus)
n=20 participants at risk
Patients will take 10 mg (1 tablet) of everolimus each day for 4 months
Everolimus: 10 mg tablets
|
|---|---|
|
Cardiac disorders
chest pain
|
5.0%
1/20 • 12 months
|
|
Gastrointestinal disorders
flank pain
|
5.0%
1/20 • 12 months
|
|
Renal and urinary disorders
dysuria
|
5.0%
1/20 • 12 months
|
|
Infections and infestations
pneumonitis
|
5.0%
1/20 • 12 months
|
|
Cardiac disorders
heart palpitations
|
5.0%
1/20 • 12 months
|
Other adverse events
| Measure |
Treatment (Everolimus)
n=20 participants at risk
Patients will take 10 mg (1 tablet) of everolimus each day for 4 months
Everolimus: 10 mg tablets
|
|---|---|
|
Infections and infestations
oral mucositis
|
70.0%
14/20 • Number of events 20 • 12 months
|
|
Renal and urinary disorders
Alanine aminotransferase increase
|
50.0%
10/20 • 12 months
|
|
Blood and lymphatic system disorders
anemia
|
45.0%
9/20 • 12 months
|
|
Renal and urinary disorders
Aspartate aminotransferase increase
|
45.0%
9/20 • 12 months
|
|
General disorders
fatigue
|
35.0%
7/20 • 12 months
|
|
Blood and lymphatic system disorders
Hypercholesterolemia
|
35.0%
7/20 • 12 months
|
|
Skin and subcutaneous tissue disorders
Maculopapular rash
|
35.0%
7/20 • 12 months
|
|
Gastrointestinal disorders
diarrhea
|
25.0%
5/20 • 12 months
|
|
Blood and lymphatic system disorders
Leukocyte decrease
|
25.0%
5/20 • 12 months
|
|
Blood and lymphatic system disorders
Hypertriglyceridemia
|
20.0%
4/20 • 12 months
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
20.0%
4/20 • 12 months
|
|
Gastrointestinal disorders
constipation
|
15.0%
3/20 • 12 months
|
|
General disorders
Xerostomia
|
15.0%
3/20 • 12 months
|
|
Blood and lymphatic system disorders
Lymphocyte decrease
|
15.0%
3/20 • 12 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
15.0%
3/20 • 12 months
|
|
Metabolism and nutrition disorders
Metabolism + nutrional disorders - other
|
15.0%
3/20 • 12 months
|
|
General disorders
Dysgeusia
|
15.0%
3/20 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
15.0%
3/20 • 12 months
|
|
Vascular disorders
Epistaxis
|
15.0%
3/20 • 12 months
|
|
Skin and subcutaneous tissue disorders
Skin + subcutaneous tissue disorders - other
|
15.0%
3/20 • 12 months
|
|
General disorders
Chills
|
10.0%
2/20 • 12 months
|
|
General disorders
fever
|
10.0%
2/20 • 12 months
|
|
Skin and subcutaneous tissue disorders
rash
|
10.0%
2/20 • 12 months
|
|
Blood and lymphatic system disorders
limb edema
|
10.0%
2/20 • 12 months
|
|
Infections and infestations
UTI
|
10.0%
2/20 • 12 months
|
|
Renal and urinary disorders
Elevated creatinine
|
10.0%
2/20 • 12 months
|
|
Blood and lymphatic system disorders
Neutrophil decrease
|
10.0%
2/20 • 12 months
|
|
General disorders
headache
|
10.0%
2/20 • 12 months
|
|
Nervous system disorders
Nervous system disorders - other
|
10.0%
2/20 • 12 months
|
|
Infections and infestations
pneumonitis
|
10.0%
2/20 • 12 months
|
|
Skin and subcutaneous tissue disorders
acneiform rash
|
10.0%
2/20 • 12 months
|
|
Nervous system disorders
involuntary movements
|
5.0%
1/20 • 12 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place