Trial Outcomes & Findings for Safety of Simvastatin in LAM and TSC (NCT NCT02061397)
NCT ID: NCT02061397
Last Updated: 2020-08-25
Results Overview
Safety is a primary outcome measure which will be assessed by any major changes or deterioration in patient health.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
10 participants
Primary outcome timeframe
5 months
Results posted on
2020-08-25
Participant Flow
Participant milestones
| Measure |
Single Simvastatin Treatment Arm
Simvastatin 20 mg oral daily for 2 months; if tolerated, followed by simvastatin 40 mg oral daily for 2 months
Simvastatin: Eligible patients will be assigned to receive 20 mg of simvastatin once daily for a period of two months. If tolerated, the dosage of simvastatin will be advanced to 40 mg once daily in months 3 and 4.
|
|---|---|
|
Simvastatin 20 mg Oral Daily for 2 Month
STARTED
|
10
|
|
Simvastatin 20 mg Oral Daily for 2 Month
COMPLETED
|
10
|
|
Simvastatin 20 mg Oral Daily for 2 Month
NOT COMPLETED
|
0
|
|
Simvastatin 40 mg Oral Daily for 2 Month
STARTED
|
10
|
|
Simvastatin 40 mg Oral Daily for 2 Month
COMPLETED
|
10
|
|
Simvastatin 40 mg Oral Daily for 2 Month
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety of Simvastatin in LAM and TSC
Baseline characteristics by cohort
| Measure |
Single Simvastatin Treatment Arm
n=10 Participants
Simvastatin 20 mg oral daily for 2 months; if tolerated, followed by simvastatin 40 mg oral daily for 2 months
Simvastatin: Eligible patients will be assigned to receive 20 mg of simvastatin once daily for a period of two months. If tolerated, the dosage of simvastatin will be advanced to 40 mg once daily in months 3 and 4.
|
|---|---|
|
Age, Continuous
|
50.4 years
STANDARD_DEVIATION 9.5 • n=99 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=99 Participants
|
|
Rate of change in FEV1
|
68.7 %
STANDARD_DEVIATION 23.8 • n=99 Participants
|
PRIMARY outcome
Timeframe: 5 monthsSafety is a primary outcome measure which will be assessed by any major changes or deterioration in patient health.
Outcome measures
| Measure |
Single Simvastatin Treatment Arm
n=10 Participants
Simvastatin 20 mg oral daily for 2 months; if tolerated, followed by simvastatin 40 mg oral daily for 2 months
Simvastatin: Eligible patients will be assigned to receive 20 mg of simvastatin once daily for a period of two months. If tolerated, the dosage of simvastatin will be advanced to 40 mg once daily in months 3 and 4.
|
|---|---|
|
Safety of Simvastatin in the Treatment of LAM-S and LAM-TS Patients
|
0 participants
|
SECONDARY outcome
Timeframe: 5 monthsLung function will be measured by FEV1: forced expiratory volume in 1s mean and calculated as % predicted +\_ SD (standard deviation).
Outcome measures
| Measure |
Single Simvastatin Treatment Arm
n=10 Participants
Simvastatin 20 mg oral daily for 2 months; if tolerated, followed by simvastatin 40 mg oral daily for 2 months
Simvastatin: Eligible patients will be assigned to receive 20 mg of simvastatin once daily for a period of two months. If tolerated, the dosage of simvastatin will be advanced to 40 mg once daily in months 3 and 4.
|
|---|---|
|
Percent Predicted FEV1
|
-2.9 percent predicted FEV1
Standard Deviation 3.7
|
Adverse Events
Single Simvastatin Treatment Arm
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Single Simvastatin Treatment Arm
n=10 participants at risk
Simvastatin 20 mg oral daily for 2 months; if tolerated, followed by simvastatin 40 mg oral daily for 2 months
Simvastatin: Eligible patients will be assigned to receive 20 mg of simvastatin once daily for a period of two months. If tolerated, the dosage of simvastatin will be advanced to 40 mg once daily in months 3 and 4.
|
|---|---|
|
Cardiac disorders
Tachycardia
|
10.0%
1/10 • Number of events 1 • 5 months
|
|
Gastrointestinal disorders
Diarrhea
|
10.0%
1/10 • Number of events 1 • 5 months
|
|
Blood and lymphatic system disorders
Peripheral adema
|
10.0%
1/10 • Number of events 4 • 5 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place