Trial Outcomes & Findings for Quality of Life in Patients With Statin-Associated Myopathy (NCT NCT00850460)
NCT ID: NCT00850460
Last Updated: 2016-11-07
Results Overview
Scores from the self-administered INQoL questionnaire will be compared at the start of the study (Week 0) and at the end (Week 8) between the statin-treated group and the placebo group. Scores range from 0-100, with 100 being a better outcome. Measures reported are the means of Week 0 and week 8, measures of dispersion is the range of the results (3 per group).
TERMINATED
PHASE4
14 participants
Week 0 to Week 8
2016-11-07
Participant Flow
Advertisements were placed (internet, radio, print) and a screening phone questionnaire was administered by study staff to determine potential inclusion. A collaboration with the Metropolitan Hospital (NY) was also established for potential referrals.
Subjects in whom statins are possibly causative, using the World Health Organization (WHO)-Causality Assessment form, were invited to the outpatient clinic for 2 screening visits. Subjects found to be at high risk for cardiovascular events and those unwilling to be shifted to placebo were not enrolled.
Participant milestones
| Measure |
Placebo
Lactose placebo pill
Placebo: Placebo pills will consist of lactose and will be given one capsule once daily
|
Statins
Statin medications
Statins: Subjects will be randomized to continue their statin dosage or placebo for 8 weeks. They will stay on the same dosage as prescribed by their physician. Usual dosage for atorvastatin 10-80 mg/tab once daily by mouth; simvastatin 20-80 mg/tab once daily by mouth; pravastatin 10-80 mg/tab once daily by mouth; rosuvastatin 5-20 mg/tab once daily by mouth.
|
|---|---|---|
|
Overall Study
STARTED
|
7
|
7
|
|
Overall Study
Started - Week 0
|
7
|
7
|
|
Overall Study
Completion - Week 8
|
3
|
3
|
|
Overall Study
COMPLETED
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
4
|
4
|
Reasons for withdrawal
| Measure |
Placebo
Lactose placebo pill
Placebo: Placebo pills will consist of lactose and will be given one capsule once daily
|
Statins
Statin medications
Statins: Subjects will be randomized to continue their statin dosage or placebo for 8 weeks. They will stay on the same dosage as prescribed by their physician. Usual dosage for atorvastatin 10-80 mg/tab once daily by mouth; simvastatin 20-80 mg/tab once daily by mouth; pravastatin 10-80 mg/tab once daily by mouth; rosuvastatin 5-20 mg/tab once daily by mouth.
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
4
|
4
|
Baseline Characteristics
Quality of Life in Patients With Statin-Associated Myopathy
Baseline characteristics by cohort
| Measure |
Placebo
n=3 Participants
Lactose placebo pill
Placebo: Placebo pills will consist of lactose and will be given one capsule once daily
|
Statins
n=3 Participants
Statin medications
Statins: Subjects will be randomized to continue their statin dosage or placebo for 8 weeks. They will stay on the same dosage as prescribed by their physician. Usual dosage for atorvastatin 10-80 mg/tab once daily by mouth; simvastatin 20-80 mg/tab once daily by mouth; pravastatin 10-80 mg/tab once daily by mouth; rosuvastatin 5-20 mg/tab once daily by mouth.
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
3 participants
n=7 Participants
|
6 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 0 to Week 8Population: Three patients from each group completed the questionnaire portion of the study at Week 0 and Week 8.
Scores from the self-administered INQoL questionnaire will be compared at the start of the study (Week 0) and at the end (Week 8) between the statin-treated group and the placebo group. Scores range from 0-100, with 100 being a better outcome. Measures reported are the means of Week 0 and week 8, measures of dispersion is the range of the results (3 per group).
Outcome measures
| Measure |
Placebo
n=3 Participants
Lactose placebo pill
Placebo: Placebo pills will consist of lactose and will be given one capsule once daily
|
Statins
n=3 Participants
Statin medications
Statins: Subjects will be randomized to continue their statin dosage or placebo for 8 weeks. They will stay on the same dosage as prescribed by their physician. Usual dosage for atorvastatin 10-80 mg/tab once daily by mouth; simvastatin 20-80 mg/tab once daily by mouth; pravastatin 10-80 mg/tab once daily by mouth; rosuvastatin 5-20 mg/tab once daily by mouth.
|
|---|---|---|
|
Individualized Neuromuscular Quality of Life (INQoL) Mean Scores From Week 0 to Week 8
Week 0 Mean Score
|
65 units on a scale
Interval 43.9 to 95.0
|
76 units on a scale
Interval 67.2 to 81.7
|
|
Individualized Neuromuscular Quality of Life (INQoL) Mean Scores From Week 0 to Week 8
Week 8 Mean Score
|
67 units on a scale
Interval 53.3 to 88.3
|
50 units on a scale
Interval 43.9 to 60.6
|
PRIMARY outcome
Timeframe: Week 0 to Week 8Population: Three patients from each group completed the questionnaire portion of the study at Week 0 and Week 8.
Scores from the self-administered SF-36 (Physical component) questionnaire were measured at the start (Week 0) of the study and at the end (Week 8) among patients in the placebo- and statin-treated group. Mean scores range from 0 (minimum) - 100 (maximum) with higher mean scores reflecting better outcomes. Measures reported are the means of Week 0 and week 8, measures of dispersion is the range of scores.
Outcome measures
| Measure |
Placebo
n=3 Participants
Lactose placebo pill
Placebo: Placebo pills will consist of lactose and will be given one capsule once daily
|
Statins
n=3 Participants
Statin medications
Statins: Subjects will be randomized to continue their statin dosage or placebo for 8 weeks. They will stay on the same dosage as prescribed by their physician. Usual dosage for atorvastatin 10-80 mg/tab once daily by mouth; simvastatin 20-80 mg/tab once daily by mouth; pravastatin 10-80 mg/tab once daily by mouth; rosuvastatin 5-20 mg/tab once daily by mouth.
|
|---|---|---|
|
Individualized Short Form-36 (SF-36) Mean Scores (Physical Component) From Week 0 to Week 8
Week 0 Mean Score
|
50 units on a scale
Interval 45.7 to 58.0
|
53 units on a scale
Interval 49.7 to 54.4
|
|
Individualized Short Form-36 (SF-36) Mean Scores (Physical Component) From Week 0 to Week 8
Week 8 Mean Score
|
56 units on a scale
Interval 53.8 to 58.6
|
48 units on a scale
Interval 47.3 to 48.2
|
Adverse Events
Placebo
Statins
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Principal Investigator
The Rockefeller University Center for Clinical and Translational
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place