Trial Outcomes & Findings for A Phase 2 Study to Evaluate the Impact of MTP-131 (Bendavia™) on Skeletal Muscle Function in Elderly (NCT NCT02245620)
NCT ID: NCT02245620
Last Updated: 2020-06-23
Results Overview
Maximal ATP synthetic rate (phosphorylation capacity per unit muscle volume) as determined by a muscle fatigue test.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
41 participants
Primary outcome timeframe
From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7
Results posted on
2020-06-23
Participant Flow
2 subjects discontinued prematurely as per Investigator's discretion. These 2 subjects (Subject 001019 \[Elamipretide group\] and Subject 001063 \[Placebo group\]) were discontinued after randomization but prior to receiving study treatment; both were not treated due to their pre-infusion sodium levels meeting Exclusion Criteria 6.
Participant milestones
| Measure |
Elamipretide
Elamipretide administered as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours.
|
Placebo
Placebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours
|
|---|---|---|
|
Overall Study
STARTED
|
20
|
21
|
|
Overall Study
COMPLETED
|
19
|
20
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Elamipretide
Elamipretide administered as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours.
|
Placebo
Placebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours
|
|---|---|---|
|
Overall Study
Physician Decision
|
1
|
1
|
Baseline Characteristics
A Phase 2 Study to Evaluate the Impact of MTP-131 (Bendavia™) on Skeletal Muscle Function in Elderly
Baseline characteristics by cohort
| Measure |
MTP-131 (Bendavia™)
n=19 Participants
MTP-131 (Bendavia™) administered as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours.
|
Placebo
n=20 Participants
Placebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours
|
Total
n=39 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
67.9 years
STANDARD_DEVIATION 3.35 • n=99 Participants
|
69.0 years
STANDARD_DEVIATION 3.96 • n=107 Participants
|
68.5 years
STANDARD_DEVIATION 3.67 • n=206 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
18 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
21 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
18 Participants
n=99 Participants
|
19 Participants
n=107 Participants
|
37 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
18 Participants
n=99 Participants
|
20 Participants
n=107 Participants
|
38 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
19 participants
n=99 Participants
|
20 participants
n=107 Participants
|
39 participants
n=206 Participants
|
|
Body Mass index
|
26.5 kg/m²
STANDARD_DEVIATION 4.27 • n=99 Participants
|
26.1 kg/m²
STANDARD_DEVIATION 2.82 • n=107 Participants
|
26.3 kg/m²
STANDARD_DEVIATION 3.56 • n=206 Participants
|
PRIMARY outcome
Timeframe: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7Population: All participants for whom Maximal ATP synthetic rate was measured at baseline and Day 1 Hour 2, and Day 7
Maximal ATP synthetic rate (phosphorylation capacity per unit muscle volume) as determined by a muscle fatigue test.
Outcome measures
| Measure |
MTP-131 (Bendavia™)
n=19 Participants
MTP-131 (Bendavia™) administered as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours.
|
Placebo
n=20 Participants
Placebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours
|
|---|---|---|
|
Change From Baseline in ATPmax (Maximal ATP Synthetic Rate)
Day 1 Hour 2
|
0.17 mM/sec
Standard Deviation 0.196
|
0.12 mM/sec
Standard Deviation 0.244
|
|
Change From Baseline in ATPmax (Maximal ATP Synthetic Rate)
Day 7
|
0.09 mM/sec
Standard Deviation 0.142
|
0.06 mM/sec
Standard Deviation 0.172
|
SECONDARY outcome
Timeframe: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7Population: All participants for whom Phosphate/Oxygen (P/O) ratio was measured at baseline and Day 1 Hour 2, and Day 7
As a measure of mitochondrial hand skeletal muscle energetics, mitochondrial coupling, or Phosphate/Oxygen ratio (P/O) was assessed at Baseline, Day 1 Hour 2, and Day 7.
Outcome measures
| Measure |
MTP-131 (Bendavia™)
n=19 Participants
MTP-131 (Bendavia™) administered as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours.
|
Placebo
n=20 Participants
Placebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours
|
|---|---|---|
|
Mean Change From Baseline in Phosphate/Oxygen (P/O) Ratio
Day 1 Hour 2
|
0.25 ratio of phosphate to oxygen
Standard Deviation 0.679
|
0.26 ratio of phosphate to oxygen
Standard Deviation 0.693
|
|
Mean Change From Baseline in Phosphate/Oxygen (P/O) Ratio
Day 7
|
0.32 ratio of phosphate to oxygen
Standard Deviation 0.473
|
0.51 ratio of phosphate to oxygen
Standard Deviation 0.788
|
SECONDARY outcome
Timeframe: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7Population: All participants for whom Nicotine Adenine Dinucleotide (NAD) was measured.
Outcome measures
| Measure |
MTP-131 (Bendavia™)
n=19 Participants
MTP-131 (Bendavia™) administered as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours.
|
Placebo
n=20 Participants
Placebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours
|
|---|---|---|
|
Mean Change From Baseline in Nicotine Adenine Dinucleotide (NAD)
Day 1 Hour 2
|
0.04 millimolar
Standard Deviation 0.169
|
0.01 millimolar
Standard Deviation 0.213
|
|
Mean Change From Baseline in Nicotine Adenine Dinucleotide (NAD)
Day 7
|
0.11 millimolar
Standard Deviation 0.215
|
0.09 millimolar
Standard Deviation 0.263
|
SECONDARY outcome
Timeframe: From Baseline to Day 1 Hour 2, Day 3, and Day 7Population: All participants for whom Muscle Force-Time-Integral (FTI) was measured.
Muscle Force-Time-Integral was measured as mean change from baseline at Day 1 Hour 2, Day 3, and Day 7.
Outcome measures
| Measure |
MTP-131 (Bendavia™)
n=19 Participants
MTP-131 (Bendavia™) administered as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours.
|
Placebo
n=20 Participants
Placebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours
|
|---|---|---|
|
Mean Change From Baseline in Muscle Force-Time-Integral (FTI)
Day 1 Hour 2
|
0.43 Newton/second
Standard Deviation 0.882
|
0.10 Newton/second
Standard Deviation 0.728
|
|
Mean Change From Baseline in Muscle Force-Time-Integral (FTI)
Day 3
|
0.46 Newton/second
Standard Deviation 1.036
|
0.24 Newton/second
Standard Deviation 0.948
|
|
Mean Change From Baseline in Muscle Force-Time-Integral (FTI)
Day 7
|
0.83 Newton/second
Standard Deviation 1.485
|
0.44 Newton/second
Standard Deviation 1.114
|
Adverse Events
Elamipretide
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Elamipretide
n=19 participants at risk
Elamipretide given as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours.
Elamipretide: Elamipretide 0.25 mg/kg/hour administered as an intravenous infusion at the rate of 60 mL/hour for 2 hours
|
Placebo
n=20 participants at risk
Placebo (lyophilized excipients without elamipretide) given as an intravenous infusion at a rate of 60 mL/hr for 2 hours.
Placebo: Placebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours
|
|---|---|---|
|
Nervous system disorders
Dizziness
|
5.3%
1/19 • 36 days
|
0.00%
0/20 • 36 days
|
|
Nervous system disorders
Headache
|
0.00%
0/19 • 36 days
|
5.0%
1/20 • 36 days
|
|
Cardiac disorders
Palpitations
|
5.3%
1/19 • 36 days
|
0.00%
0/20 • 36 days
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/19 • 36 days
|
5.0%
1/20 • 36 days
|
|
General disorders
Vessel puncture site bruise
|
0.00%
0/19 • 36 days
|
5.0%
1/20 • 36 days
|
|
Infections and infestations
Herpes zoster
|
5.3%
1/19 • 36 days
|
0.00%
0/20 • 36 days
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
5.3%
1/19 • 36 days
|
0.00%
0/20 • 36 days
|
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: LTE60