Trial Outcomes & Findings for A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension (NCT NCT03323749)
NCT ID: NCT03323749
Last Updated: 2022-01-24
Results Overview
Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
218 participants
Primary outcome timeframe
Baseline to 24 weeks
Results posted on
2022-01-24
Participant Flow
Participant milestones
| Measure |
Double-Blind Elamipretide, Then Open Label Elamepretide
40 mg (0.5mL) elamipretide subcutaneous (SC) daily
elamipretide: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then elamipretide open-label treatment: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
|
Double-Blind Placebo Then Open-Label Elamepretide
Placebo SC daily
Double-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system.
|
|---|---|---|
|
Double-blind Period
STARTED
|
109
|
109
|
|
Double-blind Period
COMPLETED
|
102
|
103
|
|
Double-blind Period
NOT COMPLETED
|
7
|
6
|
|
Open-Label Period
STARTED
|
93
|
103
|
|
Open-Label Period
COMPLETED
|
0
|
0
|
|
Open-Label Period
NOT COMPLETED
|
93
|
103
|
Reasons for withdrawal
| Measure |
Double-Blind Elamipretide, Then Open Label Elamepretide
40 mg (0.5mL) elamipretide subcutaneous (SC) daily
elamipretide: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then elamipretide open-label treatment: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
|
Double-Blind Placebo Then Open-Label Elamepretide
Placebo SC daily
Double-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system.
|
|---|---|---|
|
Double-blind Period
Lost to Follow-up
|
4
|
5
|
|
Double-blind Period
Adverse Event
|
3
|
1
|
|
Open-Label Period
Termination of the Clinical Development of Elamipretide in Subjects with PMM
|
83
|
88
|
|
Open-Label Period
Adverse Event
|
3
|
10
|
|
Open-Label Period
Withdrawal by Subject
|
7
|
5
|
Baseline Characteristics
Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
Baseline characteristics by cohort
| Measure |
Elamipretide
n=109 Participants
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then Open-label Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
|
Placebo
n=109 Participants
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks, then Open-label Period: Elamipretide: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
|
Total
n=218 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
Years, Double-blind
|
45.5 years
STANDARD_DEVIATION 15.72 • n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
44.3 years
STANDARD_DEVIATION 14.34 • n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
44.9 years
STANDARD_DEVIATION 15.02 • n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Age, Customized
Years, Open-label
|
45.7 years
STANDARD_DEVIATION 15.97 • n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
45.0 years
STANDARD_DEVIATION 14.25 • n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
45.3 years
STANDARD_DEVIATION 15.05 • n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Sex: Female, Male
Double-Blind Sex · Female
|
67 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
73 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
140 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Sex: Female, Male
Double-Blind Sex · Male
|
42 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
36 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
78 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Sex: Female, Male
Open-Enrollment Sex · Female
|
56 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
67 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
123 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Sex: Female, Male
Open-Enrollment Sex · Male
|
37 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
36 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
73 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Ethnicity (NIH/OMB)
Ethnicity Double-Blind · Hispanic or Latino
|
11 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
10 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
21 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Ethnicity (NIH/OMB)
Ethnicity Double-Blind · Not Hispanic or Latino
|
95 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
96 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
191 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Ethnicity (NIH/OMB)
Ethnicity Double-Blind · Unknown or Not Reported
|
3 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
3 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
6 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Ethnicity (NIH/OMB)
Ethnicity Open-Label · Hispanic or Latino
|
8 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
10 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
18 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Ethnicity (NIH/OMB)
Ethnicity Open-Label · Not Hispanic or Latino
|
82 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
91 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
173 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Ethnicity (NIH/OMB)
Ethnicity Open-Label · Unknown or Not Reported
|
3 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
2 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
5 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Double-blind Period · American Indian or Alaska Native
|
1 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
0 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
1 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Double-blind Period · Asian
|
2 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
5 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
7 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Double-blind Period · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
0 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
0 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Double-blind Period · Black or African American
|
1 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
0 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
1 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Double-blind Period · White
|
103 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
100 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
203 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Double-blind Period · More than one race
|
2 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
1 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
3 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Double-blind Period · Unknown or Not Reported
|
0 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
3 Participants
n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
3 Participants
n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Open-label period · American Indian or Alaska Native
|
1 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
0 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
1 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Open-label period · Asian
|
0 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
4 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
4 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Open-label period · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
0 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
0 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Open-label period · Black or African American
|
1 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
0 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
1 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Open-label period · White
|
89 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
95 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
184 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Open-label period · More than one race
|
2 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
1 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
3 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Race (NIH/OMB)
Open-label period · Unknown or Not Reported
|
0 Participants
n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
3 Participants
n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
3 Participants
n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Weight (kg)
Double-blind
|
64.81 kg
STANDARD_DEVIATION 20.287 • n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
67.24 kg
STANDARD_DEVIATION 17.336 • n=109 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
66.02 kg
STANDARD_DEVIATION 18.865 • n=218 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
|
Weight (kg)
Open-label
|
65.66 kg
STANDARD_DEVIATION 20.867 • n=93 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
68.01 kg
STANDARD_DEVIATION 17.237 • n=103 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
66.89 kg
STANDARD_DEVIATION 19.033 • n=196 Participants • Open-label Period: A total of 196 Subjects enrolled in the Open-Label Extension: 93 from the original Double-Blind Period Elamipretide group and 103 from the original Double-Blind Placebo group.
|
PRIMARY outcome
Timeframe: Baseline to 24 weeksPopulation: All participants for whom 6MWT was measured
Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit
Outcome measures
| Measure |
Elamipretide
n=109 Participants
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
|
Placebo
n=109 Participants
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
|
|---|---|---|
|
Six-minute Walk Test (6MWT)
Week 4
|
16.243 meters
Standard Deviation 42.0071
|
7.811 meters
Standard Deviation 39.5737
|
|
Six-minute Walk Test (6MWT)
Week 12
|
18.286 meters
Standard Deviation 47.7766
|
8.801 meters
Standard Deviation 52.1093
|
|
Six-minute Walk Test (6MWT)
Week 24
|
15.330 meters
Standard Deviation 61.4860
|
17.386 meters
Standard Deviation 51.6956
|
PRIMARY outcome
Timeframe: Baseline to 24 weeksPopulation: All participants for whom Total Fatigue Score (Q1 to Q4) Based on PMMSA by Visit was measured
Change from Baseline in Total fatigue score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) by visit. Each individual item score ranges from 1 (none) to 4 (severe). The total fatigue score ranges from 4-16. Lower values represent a better outcome. The total fatigue score is the sum of question 1 through question 4 on the Primary Mitochondrial Myopathy Symptom Assessment.
Outcome measures
| Measure |
Elamipretide
n=109 Participants
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
|
Placebo
n=109 Participants
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
|
|---|---|---|
|
Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 4
|
-1.01 score on a scale
Standard Deviation 1.714
|
-1.08 score on a scale
Standard Deviation 1.865
|
|
Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 12
|
-1.08 score on a scale
Standard Deviation 1.775
|
-1.26 score on a scale
Standard Deviation 2.259
|
|
Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 24
|
-1.18 score on a scale
Standard Deviation 2.132
|
-1.09 score on a scale
Standard Deviation 2.443
|
SECONDARY outcome
Timeframe: Baseline to 24 weeksPopulation: All participants for whom Fatigue During Activities was measured.
Change from baseline in Fatigue During Activities. Fatigue During Activities is the sum of question 2 (tiredness during activities) and question 4 (muscle weakness during activities.) The four response options are: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Raw scores for each subject range from 2-8. A lower score means a better outcome, with less fatigue. A higher score means a worse outcome, with more fatigue.
Outcome measures
| Measure |
Elamipretide
n=109 Participants
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
|
Placebo
n=109 Participants
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
|
|---|---|---|
|
Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA).
Week 4
|
-0.48 score on a scale
Standard Deviation 0.890
|
-0.60 score on a scale
Standard Deviation 0.998
|
|
Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA).
Week 12
|
-0.57 score on a scale
Standard Deviation 0.923
|
-0.67 score on a scale
Standard Deviation 1.199
|
|
Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA).
Week 24
|
-0.64 score on a scale
Standard Deviation 1.151
|
-0.59 score on a scale
Standard Deviation 1.360
|
SECONDARY outcome
Timeframe: Baseline to 24 weeksPopulation: All participants for whom Change From Baseline in Neuro-QoL Fatigue Activities of Daily Living by Visit was measured.
Change From Baseline in Neuro-QoL Fatigue Activities of Daily Living by Visit. Each individual item score ranges from 1-5. Total raw score for the entire item bank ranges from 19-95. Raw scores will be calibrated using Item Response Theory Model. Lower values represent a better outcome. Individual items will be summed to calculate total scores.
Outcome measures
| Measure |
Elamipretide
n=109 Participants
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
|
Placebo
n=109 Participants
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
|
|---|---|---|
|
Neuro-QoL Fatigue Activities of Daily Living
Week 4
|
-2.5 score on a scale
Standard Deviation 5.33
|
-2.7 score on a scale
Standard Deviation 5.31
|
|
Neuro-QoL Fatigue Activities of Daily Living
Week 12
|
-2.9 score on a scale
Standard Deviation 6.00
|
-2.9 score on a scale
Standard Deviation 5.13
|
|
Neuro-QoL Fatigue Activities of Daily Living
Week 24
|
-2.7 score on a scale
Standard Deviation 6.06
|
-2.1 score on a scale
Standard Deviation 5.39
|
SECONDARY outcome
Timeframe: Baseline to 24 weeksPopulation: All participants for whom Most Bothersome Symptom Score (PMMSA) by Visit was measured.
The item score rangers from 1 (none) to 4 (severe). Lower values represent a better outcome. The most bothersome score is the average of the identified most bothersome symptom of the Primary Mitochondrial Myopathy Symptom Assessment by each subject.
Outcome measures
| Measure |
Elamipretide
n=109 Participants
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
|
Placebo
n=109 Participants
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
|
|---|---|---|
|
Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment
Week 4
|
-0.21 score on a scale
Standard Deviation 0.492
|
-0.24 score on a scale
Standard Deviation 0.535
|
|
Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment
Week 12
|
-0.24 score on a scale
Standard Deviation 0.535
|
-0.34 score on a scale
Standard Deviation 0.656
|
|
Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment
Week 24
|
-0.25 score on a scale
Standard Deviation 0.618
|
-0.30 score on a scale
Standard Deviation 0.713
|
SECONDARY outcome
Timeframe: 24 WeeksPopulation: All participants for whom Neuro-QoL Fatigue Short Form Score T-scores were measured.
Change From Baseline in Neuro-QoL Fatigue - Short Form: Total T-Scores by Visit. The Neuro-QoL Fatigue Short Form is comprised of the sum of the first 8 questions of the Neuro-QoL Item Bank v1.0 - Fatigue. Each question is scored as following: 1=Never, 2=Rarely, 3=Sometimes, 4=Often, and 5=Always. The questions include: I felt exhausted, I felt that I had no energy, I felt fatigued, I was too tired to do my household chores, I was too tired to leave the house, I was frustrated by being too tired to do the things I wanted to do, I felt tired, and I had to limit my social activity because I was tired. T-scores are calculated from the short form scoring table provided by the instrument authors (Neuro-QoL User Manual, 2015). T-score distributions rescale raw scores into standardized scores with a mean of 50 and a standard deviation (SD) of 10. Change from baseline: Negative numbers mean less fatigue, better outcome, positive score means more fatigue, worse outcome.
Outcome measures
| Measure |
Elamipretide
n=109 Participants
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
|
Placebo
n=109 Participants
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
|
|---|---|---|
|
Neuro-QoL Fatigue Short Form Score
Week 4
|
-3.37 score on a scale
Standard Deviation 5.077
|
-2.89 score on a scale
Standard Deviation 5.915
|
|
Neuro-QoL Fatigue Short Form Score
Week 12
|
-3.43 score on a scale
Standard Deviation 5.681
|
-3.60 score on a scale
Standard Deviation 5.614
|
|
Neuro-QoL Fatigue Short Form Score
Week 24
|
-2.68 score on a scale
Standard Deviation 5.769
|
-2.61 score on a scale
Standard Deviation 5.773
|
Adverse Events
Double-Blind Elamipretide
Serious events: 5 serious events
Other events: 107 other events
Deaths: 0 deaths
Double-Blind Placebo
Serious events: 3 serious events
Other events: 83 other events
Deaths: 0 deaths
Open-Label Elamipretide
Serious events: 12 serious events
Other events: 88 other events
Deaths: 0 deaths
Open-Label Placebo
Serious events: 9 serious events
Other events: 102 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Double-Blind Elamipretide
n=109 participants at risk
Double-blind period: elamipretide: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then elamipretide open-label treatment: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
|
Double-Blind Placebo
n=109 participants at risk
Placebo SC daily
Double-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system.
|
Open-Label Elamipretide
n=93 participants at risk
Double blind period: elamipretide: 40mg (0.5mL) of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then elamipretide open-label treatment: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
|
Open-Label Placebo
n=103 participants at risk
Double-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Duodenal ulcer haemorrhage
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Nervous system disorders
Hemiplegia
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Vascular disorders
Labile blood pressure
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Psychiatric disorders
Suicidal Ideation
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Cellulitis
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Diverticulitis
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Viral sinusitis
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Metabolism and nutrition disorders
Lactic Acidosis
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Nervous system disorders
Hypoaesthesia
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Gastrointestinal disorders
Gastroenteritis
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Musculoskeletal and connective tissue disorders
Rheumatoid arthritis
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Nervous system disorders
Psychogenic seizure
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc disorder
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Cardiac disorders
Acute myocardial infarction
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Cardiac disorders
Coronary artery dissection
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
Chest pain
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Gastrointestinal disorders
Intestinal pseudo-obstruction
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Renal and urinary disorders
Urinary retention
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Vascular disorders
Hypertension
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Congenital, familial and genetic disorders
MELAS syndrome
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Sinusitis
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Injury, poisoning and procedural complications
Vaginal laceration
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Nervous system disorders
Cerebrovascular accident
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Cardiac disorders
Atrioventricular block complete
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Nervous system disorders
Spinal cord compression
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Papillary thyroid cancer
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
Other adverse events
| Measure |
Double-Blind Elamipretide
n=109 participants at risk
Double-blind period: elamipretide: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then elamipretide open-label treatment: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
|
Double-Blind Placebo
n=109 participants at risk
Placebo SC daily
Double-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system.
|
Open-Label Elamipretide
n=93 participants at risk
Double blind period: elamipretide: 40mg (0.5mL) of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then elamipretide open-label treatment: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
|
Open-Label Placebo
n=103 participants at risk
Double-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system.
|
|---|---|---|---|---|
|
General disorders
Injection site erythema
|
86.2%
94/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
28.4%
31/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
71.0%
66/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
84.5%
87/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
Injection site pruritus
|
75.2%
82/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
9.2%
10/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
61.3%
57/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
78.6%
81/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
Injection site pain
|
39.4%
43/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
18.3%
20/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
34.4%
32/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
35.9%
37/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
Injection site swelling
|
38.5%
42/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
6.4%
7/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
31.2%
29/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
19.4%
20/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
Injection site induration
|
28.4%
31/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
5.5%
6/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
26.9%
25/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
39.8%
41/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
injection site bruising
|
8.3%
9/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
16.5%
18/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
5.4%
5/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
14.6%
15/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
injection site haemorrhage
|
6.4%
7/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
9.2%
10/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
7.8%
8/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
injection site urticaria
|
12.8%
14/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
12.9%
12/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
7.8%
8/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
injection site nodule
|
10.1%
11/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.8%
2/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
8.6%
8/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
7.8%
8/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
injection site mass
|
8.3%
9/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.8%
2/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
4.3%
4/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.9%
4/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
Fatigue
|
3.7%
4/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.7%
4/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
4.3%
4/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.9%
4/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
injection site haematoma
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
6.4%
7/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
Pyrexia
|
1.8%
2/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.8%
3/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
Asthenia
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.2%
3/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
General disorders
Injection site injury
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.9%
4/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Upper respiratory tract Infection
|
6.4%
7/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
6.4%
7/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
7.5%
7/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
6.8%
7/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Nasopharyngitis
|
7.3%
8/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.8%
2/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
6.5%
6/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Sinusitis
|
1.8%
2/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.8%
3/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.9%
4/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Nervous system disorders
Headache
|
7.3%
8/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.7%
4/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
5.4%
5/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
5.8%
6/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Nervous system disorders
Dizziness
|
5.5%
6/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.8%
3/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.9%
3/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Gastrointestinal disorders
Nausea
|
4.6%
5/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
7.3%
8/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
4.3%
4/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Gastrointestinal disorders
Diarrhoea
|
2.8%
3/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
8.3%
9/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.2%
3/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
5.8%
6/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Investigations
Eosinophil count increased
|
6.4%
7/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
7.5%
7/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
6.8%
7/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
4.6%
5/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
4.9%
5/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Injury, poisoning and procedural complications
Fall
|
5.5%
6/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.8%
3/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
8.6%
8/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
6.8%
7/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
3.7%
4/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.2%
3/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.9%
3/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Influenza
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Pneumonia
|
0.92%
1/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Infections and infestations
Urinary Tract Infection
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
4.3%
4/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
4.3%
4/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Investigations
Weight Decreased
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.9%
3/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Investigations
Blood glucose increased
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Investigations
Blood lactic acid increased
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Nervous system disorders
Hypoaesthesia
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.9%
3/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Nervous system disorders
Migraine
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.2%
3/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.2%
3/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
4.3%
4/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.2%
3/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
4.3%
4/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Musculoskeletal and connective tissue disorders
Muscular Weakness
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
3.2%
3/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.97%
1/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Injury, poisoning and procedural complications
Skin Abrasion
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.9%
3/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.9%
2/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Blood and lymphatic system disorders
Eosinophilia
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
1.1%
1/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
8.7%
9/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
|
Vascular disorders
Hypertension
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
0.00%
0/109 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.2%
2/93 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
2.9%
3/103 • Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
|
Additional Information
Jim Carr, Pharm.D. Chief Clinical Development Officer
Stealth BioTherapeutics, Inc
Phone: 1-617-600-6888
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60