Trial Outcomes & Findings for Effect of Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Reduced Ejection Fraction (NCT NCT02788747)

NCT ID: NCT02788747

Last Updated: 2020-05-14

Results Overview

Change in left ventricular end systolic volume as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 71 participants
• Primary outcome timeframe: Baseline to Week 4
• Results posted on: 2020-05-14

Participant Flow

Participant milestones

Measure	4 mg Elamipretide Subcutaneous injection of 4 mg elamipretide administered once daily for 28 consecutive days	40 mg Elamipretide Subcutaneous injection of 40 mg elamipretide administered once daily for 28 consecutive days	Placebo Subcutaneous injection of placebo administered once daily for 28 consecutive days
Overall Study STARTED	23	24	24
Overall Study COMPLETED	22	24	24
Overall Study NOT COMPLETED	1	0	0

Reasons for withdrawal

Measure	4 mg Elamipretide Subcutaneous injection of 4 mg elamipretide administered once daily for 28 consecutive days	40 mg Elamipretide Subcutaneous injection of 40 mg elamipretide administered once daily for 28 consecutive days	Placebo Subcutaneous injection of placebo administered once daily for 28 consecutive days
Overall Study Adverse Event	1	0	0

Baseline Characteristics

Effect of Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Reduced Ejection Fraction

Baseline characteristics by cohort

Measure	4 mg Elamipretide n=22 Participants Subcutaneous injection of 4 mg elamipretide administered once daily for 28 consecutive days	40 mg Elamipretide n=25 Participants Subcutaneous injection of 40 mg elamipretide administered once daily for 28 consecutive days	Placebo n=24 Participants Subcutaneous injection of placebo administered once daily for 28 consecutive days	Total n=71 Participants Total of all reporting groups
Age, Continuous	65.3 years STANDARD_DEVIATION 10.76 • n=93 Participants	62.6 years STANDARD_DEVIATION 9.63 • n=4 Participants	66.8 years STANDARD_DEVIATION 9.07 • n=27 Participants	64.8 years STANDARD_DEVIATION 9.83 • n=483 Participants
Sex: Female, Male Female	3 Participants n=93 Participants	5 Participants n=4 Participants	9 Participants n=27 Participants	17 Participants n=483 Participants
Sex: Female, Male Male	19 Participants n=93 Participants	20 Participants n=4 Participants	15 Participants n=27 Participants	54 Participants n=483 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=93 Participants	1 Participants n=4 Participants	0 Participants n=27 Participants	1 Participants n=483 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	22 Participants n=93 Participants	24 Participants n=4 Participants	24 Participants n=27 Participants	70 Participants n=483 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) White	22 Participants n=93 Participants	25 Participants n=4 Participants	24 Participants n=27 Participants	71 Participants n=483 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Left ventricular end systolic volume	87.0 mL STANDARD_DEVIATION 38.45 • n=93 Participants	79.6 mL STANDARD_DEVIATION 21.54 • n=4 Participants	77.7 mL STANDARD_DEVIATION 38.58 • n=27 Participants	81.3 mL STANDARD_DEVIATION 33.28 • n=483 Participants

PRIMARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom left ventricular end systolic volume was measured at baseline and Week 4

Change in left ventricular end systolic volume as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

Outcome measures

Measure	4 mg Elamipretide n=22 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=24 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=23 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Ventricular End Systolic Volume (ml)	-4.4 mL Standard Deviation 6.50	-1.2 mL Standard Deviation 9.04	-3.8 mL Standard Deviation 5.85

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for Left Ventricular Ejection Fraction was measured at baseline and Week 4

Change in Left Ventricular Ejection Fraction as measured by percentage of blood volume from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

Outcome measures

Measure	4 mg Elamipretide n=22 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=24 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=23 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Ventricular Ejection Fraction (% of Blood Volume)	2.2 percentage of blood volume Standard Deviation 2.43	1.5 percentage of blood volume Standard Deviation 2.82	2.3 percentage of blood volume Standard Deviation 3.69

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Left Ventricular End Diastolic Volume was measured at baseline and Week 4

Change from baseline in Left Ventricular End Diastolic Volume as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

Outcome measures

Measure	4 mg Elamipretide n=22 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=24 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=23 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Ventricular End Diastolic Volume (ml) as Measured by MRI	-2.8 mL Standard Deviation 9.50	0.5 mL Standard Deviation 11.96	-2.2 mL Standard Deviation 9.77

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Left Ventricular Stroke Volume was measured at baseline and Week 4

Change in Left Ventricular Stroke Volume as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

Outcome measures

Measure	4 mg Elamipretide n=22 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=24 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=23 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Ventricular Stroke Volume (ml)	3.1 mL Standard Deviation 9.23	3.7 mL Standard Deviation 9.36	3.0 mL Standard Deviation 13.14

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Left Ventricular Cardiac Output was measured at baseline and Week 4

Change in Left Ventricular Cardiac Output as measured by L/min from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

Outcome measures

Measure	4 mg Elamipretide n=22 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=24 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=23 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Ventricular Cardiac Output (L/Min)	0.21 L/min Standard Deviation 1.264	-0.01 L/min Standard Deviation 1.004	0.27 L/min Standard Deviation 0.860

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Left Ventricular Myocardial Mass was measured at baseline and Week 4

Change in Left Ventricular Myocardial Mass as measured by grams from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

Outcome measures

Measure	4 mg Elamipretide n=22 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=24 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=23 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Ventricular Myocardial Mass (g)	0.0 grams Standard Deviation 2.82	-0.4 grams Standard Deviation 2.78	-0.0 grams Standard Deviation 2.26

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Right Ventricular End Systolic Volume was measured at baseline and Week 4

Change in Right Ventricular End Systolic Volume as measured by mL from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

Outcome measures

Measure	4 mg Elamipretide n=22 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=24 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=23 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Right Ventricular End Systolic Volume (mL)	-0.9 mL Standard Deviation 4.21	-0.8 mL Standard Deviation 4.94	-0.6 mL Standard Deviation 3.72

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Right Ventricular End Diastolic Volume was measured at baseline and Week 4

Change in Right Ventricular End Diastolic Volume as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

Outcome measures

Measure	4 mg Elamipretide n=22 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=24 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=23 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Right Ventricular End Diastolic Volume (mL)	0.5 mL Standard Deviation 9.70	-1.2 mL Standard Deviation 10.72	-0.0 mL Standard Deviation 7.61

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Right Ventricular Ejection Fraction was measured at baseline and Week 4

Change in Right Ventricular Ejection Fraction as measured by percentage of blood volume from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by MRI.

Outcome measures

Measure	4 mg Elamipretide n=22 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=24 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=23 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Right Ventricular Ejection Fraction (% Blood Volume)	1.4 percentage of blood volume Standard Deviation 3.05	0.4 percentage of blood volume Standard Deviation 2.10	1.2 percentage of blood volume Standard Deviation 3.33

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Early and Late Mitral Inflow Velocity Ratio was measured at baseline and Week 4

Change in Early and Late Mitral Inflow Velocity Ratio from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=20 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=23 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=22 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Early and Late Mitral Inflow Velocity Ratio	-0.04 ratio Standard Deviation 0.185	-0.03 ratio Standard Deviation 0.329	-0.07 ratio Standard Deviation 0.528

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Early Mitral Inflow Velocity and Mitral Annular Early Diastolic Velocity Ratio was measured at baseline and Week 4

Change in Early Mitral Inflow Velocity and Mitral Annular Early Diastolic Velocity Ratio as measured by E/e' from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=21 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=23 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=22 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Early Mitral Inflow Velocity and Mitral Annular Early Diastolic Velocity Ratio (E/e')	-0.94 ratio Standard Deviation 3.244	0.47 ratio Standard Deviation 4.359	-0.20 ratio Standard Deviation 4.287

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Left Atrial Volume was measured at baseline and Week 4

Change in Left Atrial Volume as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=20 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=20 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=22 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Atrial Volume (mL)	-3.4 mL Standard Deviation 6.85	-0.2 mL Standard Deviation 4.41	1.6 mL Standard Deviation 4.02

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Left Ventricular Global Longitudinal Strain Assessment was measured at baseline and Week 4

Change in Left Ventricular Global Longitudinal Strain Assessment as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=4 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=3 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=9 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Ventricular Global Longitudinal Strain Assessment (%)	-1.76 percentage of myocardial length change Standard Deviation 1.993	-1.50 percentage of myocardial length change Standard Deviation 2.721	-1.46 percentage of myocardial length change Standard Deviation 2.362

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Left Ventricular End Diastolic Volume was measured at baseline and Week 4

Change in Left Ventricular End Diastolic Volume as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=15 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=16 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=20 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Ventricular End Diastolic Volume (mL) as Measured by Echocardiography	-1.1 mL Standard Deviation 16.29	1.6 mL Standard Deviation 13.68	2.0 mL Standard Deviation 15.29

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Left Ventricular End Systolic Volume was measured at baseline and Week 4

Change in Left Ventricular End Systolic Volume as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=15 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=16 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=20 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Left Ventricular End Systolic Volume (mL) as Measured by Echocardiography	-1.9 mL Standard Deviation 11.59	-0.5 mL Standard Deviation 7.91	0.6 mL Standard Deviation 10.31

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Biplane Ejection Fraction was measured at baseline and Week 4

Change in Biplane Ejection Fraction as measured by ml from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=14 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=16 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=20 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Biplane Ejection Fraction (mL)	1.8 mL Standard Deviation 2.94	2.0 mL Standard Deviation 2.82	1.5 mL Standard Deviation 4.22

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Left Ventricular Mass was measured at baseline and Week 4

Change in Left Ventricular Mass Assessment as measured by grams from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=15 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=15 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=20 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Left Ventricular Mass Assessment (g)	3.0 grams Standard Deviation 18.26	-4.2 grams Standard Deviation 18.55	-0.7 grams Standard Deviation 21.99

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Tricuspid Regurgitation Severity Assessment was measured at baseline and Week 4

Change in Tricuspid Regurgitation Severity Assessment as measured by cm from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=2 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=2 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=1 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Tricuspid Regurgitation Severity Assessment (cm²)	-1.03 cm² Standard Deviation 1.334	0.25 cm² Standard Deviation 1.336	-0.30 cm²

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Right Ventricular Fractional Area was measured at baseline and Week 4

Change Right Ventricular Fractional Area in as measured by percentage from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=13 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=14 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=15 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Right Ventricular Fractional Area (%)	0.1 percentage of area Standard Deviation 10.82	0.1 percentage of area Standard Deviation 8.79	0.9 percentage of area Standard Deviation 8.93

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Right Ventricular Systolic Pressure was measured at baseline and Week 4

Change in Change in Right Ventricular Systolic Pressure as measured by mmHg from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=8 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=9 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=8 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Right Ventricular Systolic Pressure (mmHg)	5.7 mmHg Standard Deviation 16.19	-1.5 mmHg Standard Deviation 6.63	-7.0 mmHg Standard Deviation 25.61

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: All participants for whom Mitral Regurgitation Severity was measured at baseline and Week 4

Change in Mitral Regurgitation Severity as measured by cm² from baseline (last assessment prior to start of study) to Week 4 (end of treatment visit) as assessed by echocardiography.

Outcome measures

Measure	4 mg Elamipretide n=5 Participants 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=5 Participants 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=6 Participants Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Change in Mitral Regurgitation Severity (cm²)	0.70 cm² Standard Deviation 2.668	-0.71 cm² Standard Deviation 2.200	-0.73 cm² Standard Deviation 1.834

Adverse Events

4 mg Elamipretide

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

40 mg Elamipretide

Serious events: 1 serious events

Other events: 16 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Measure	4 mg Elamipretide n=22 participants at risk 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=25 participants at risk 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=24 participants at risk Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Nervous system disorders Vestibular	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)

Other adverse events

Measure	4 mg Elamipretide n=22 participants at risk 4 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	40 mg Elamipretide n=25 participants at risk 40 mg elamipretide: subcutaneous injection administered once daily for 28 consecutive days	Placebo n=24 participants at risk Placebo: subcutaneous injection of placebo administered once daily for 28 consecutive days
Vascular disorders Deep vein thrombosis	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Vascular disorders Hypertension	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Vascular disorders Hypotension	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Investigations Increased Blood bilirubin	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Investigations Eosinphil count increased	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Investigations Troponin I increased	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Respiratory, thoracic and mediastinal disorders Productive cough	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Nervous system disorders Dizziness	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Nervous system disorders Headache	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Nervous system disorders Sciatica	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
General disorders Fatigue	9.1% 2/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Nervous system disorders Somnolence	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
General disorders Injection site erythema	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
General disorders Injection site haemorrhage	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
General disorders Injection site induration	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
General disorders Injection site mass	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
General disorders Injection site pain	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
General disorders Oedema peripheral	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
General disorders Pyrexia	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Psychiatric disorders Insomia	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Psychiatric disorders Anxiety	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Gastrointestinal disorders Constipation	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Gastrointestinal disorders Diarrhoea	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Gastrointestinal disorders Nausea	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Renal and urinary disorders Albuminiuria	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Renal and urinary disorders Haematuria	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Renal and urinary disorders Renal impairment	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Musculoskeletal and connective tissue disorders Musculoskeletal pain	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Musculoskeletal and connective tissue disorders Pain in extremity	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Metabolism and nutrition disorders Diabetes mellitus	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Metabolism and nutrition disorders Gout	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Metabolism and nutrition disorders Hyperkalemia	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Metabolism and nutrition disorders Hypokalemia	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Infections and infestations Nasopharyngitis	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	8.0% 2/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.2% 1/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Infections and infestations Gastroenteritis viral	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Infections and infestations Respiratory tract infection	4.5% 1/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)
Infections and infestations Upper respiratory tract infection	0.00% 0/22 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	4.0% 1/25 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)	0.00% 0/24 • 10 weeks (The AE reporting period began when the subject signed the ICF and continued through the clinical study's post treatment follow-up period, defined as 14 days after last administration of study medication.)

Additional Information

Jim Carr PharmD Chief Clinical Development Officer

Stealth BioTherapeutics Inc.

Phone: 617-600-6888

Email: jim.carr@stealthbt.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60