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A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension (MMPOWER-3)

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ClinicalTrials.gov Identifier: NCT03323749
Recruitment Status : Terminated (Part1,double blind portion of the trial did not meet the primary end points)
First Posted : October 27, 2017
Results First Posted : April 2, 2021
Last Update Posted : April 2, 2021
Sponsor:
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Primary Mitochondrial Myopathy
Interventions Combination Product: elamipretide
Combination Product: placebo comparator
Combination Product: elamipretide open label treatment
Enrollment 218
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Double-Blind Elamipretide, Then Open Label Elamepretide Double-Blind Placebo Then Open-Label Elamepretide
Hide Arm/Group Description

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

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.

Period Title: Double-blind Period
Started 109 109
Completed 102 103
Not Completed 7 6
Reason Not Completed
Lost to Follow-up             4             5
Adverse Event             3             1
Period Title: Open-Label Period
Started [1] 93 103
Completed 0 0
Not Completed 93 103
Reason Not Completed
Termination of the Clinical Development of Elamipretide in Subjects with PMM             83             88
Adverse Event             3             10
Withdrawal by Subject             7             5
[1]
A total of 196 subjects enrolled in Part 2 of the trial: 93 from the elamipretide group in Part 1, and 103 from the placebo group in Part 1. No subjects completed the trial: it was terminated early due to the Sponsor's decision.
Arm/Group Title Elamipretide Placebo Total
Hide Arm/Group Description 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 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 of all reporting groups
Overall Number of Baseline Participants 109 109 218
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Years, Double-blind Number Analyzed 109 participants 109 participants 218 participants
45.5  (15.72) 44.3  (14.34) 44.9  (15.02)
Years, Open-label Number Analyzed 93 participants 103 participants 196 participants
45.7  (15.97) 45.0  (14.25) 45.3  (15.05)
[1]
Measure Analysis Population Description: 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   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Double-Blind Sex Number Analyzed 109 participants 109 participants 218 participants
Female
67
  61.5%
73
  67.0%
140
  64.2%
Male
42
  38.5%
36
  33.0%
78
  35.8%
Open-Enrollment Sex Number Analyzed 93 participants 103 participants 196 participants
Female
56
  60.2%
67
  65.0%
123
  62.8%
Male
37
  39.8%
36
  35.0%
73
  37.2%
[1]
Measure Analysis Population Description: 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)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Ethnicity Double-Blind Number Analyzed 109 participants 109 participants 218 participants
Hispanic or Latino
11
  10.1%
10
   9.2%
21
   9.6%
Not Hispanic or Latino
95
  87.2%
96
  88.1%
191
  87.6%
Unknown or Not Reported
3
   2.8%
3
   2.8%
6
   2.8%
Ethnicity Open-Label Number Analyzed 93 participants 103 participants 196 participants
Hispanic or Latino
8
   8.6%
10
   9.7%
18
   9.2%
Not Hispanic or Latino
82
  88.2%
91
  88.3%
173
  88.3%
Unknown or Not Reported
3
   3.2%
2
   1.9%
5
   2.6%
[1]
Measure Analysis Population Description: 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)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Double-blind Period Number Analyzed 109 participants 109 participants 218 participants
American Indian or Alaska Native
1
   0.9%
0
   0.0%
1
   0.5%
Asian
2
   1.8%
5
   4.6%
7
   3.2%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   0.9%
0
   0.0%
1
   0.5%
White
103
  94.5%
100
  91.7%
203
  93.1%
More than one race
2
   1.8%
1
   0.9%
3
   1.4%
Unknown or Not Reported
0
   0.0%
3
   2.8%
3
   1.4%
Open-label period Number Analyzed 93 participants 103 participants 196 participants
American Indian or Alaska Native
1
   1.1%
0
   0.0%
1
   0.5%
Asian
0
   0.0%
4
   3.9%
4
   2.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   1.1%
0
   0.0%
1
   0.5%
White
89
  95.7%
95
  92.2%
184
  93.9%
More than one race
2
   2.2%
1
   1.0%
3
   1.5%
Unknown or Not Reported
0
   0.0%
3
   2.9%
3
   1.5%
[1]
Measure Analysis Population Description: 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)   [1] 
Mean (Standard Deviation)
Unit of measure:  Kg
Double-blind Number Analyzed 109 participants 109 participants 218 participants
64.81  (20.287) 67.24  (17.336) 66.02  (18.865)
Open-label Number Analyzed 93 participants 103 participants 196 participants
65.66  (20.867) 68.01  (17.237) 66.89  (19.033)
[1]
Measure Analysis Population Description: 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.Primary Outcome
Title Six-minute Walk Test (6MWT)
Hide Description Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit
Time Frame Baseline to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All participants for whom 6MWT was measured
Arm/Group Title Elamipretide Placebo
Hide Arm/Group Description:
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
Overall Number of Participants Analyzed 109 109
Mean (Standard Deviation)
Unit of Measure: meters
Week 4 Number Analyzed 108 participants 105 participants
16.243  (42.0071) 7.811  (39.5737)
Week 12 Number Analyzed 106 participants 102 participants
18.286  (47.7766) 8.801  (52.1093)
Week 24 Number Analyzed 100 participants 102 participants
15.330  (61.4860) 17.386  (51.6956)
2.Primary Outcome
Title Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Hide Description 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.
Time Frame Baseline to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All participants for whom Total Fatigue Score (Q1 to Q4) Based on PMMSA by Visit was measured
Arm/Group Title Elamipretide Placebo
Hide Arm/Group Description:
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
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
Overall Number of Participants Analyzed 109 109
Mean (Standard Deviation)
Unit of Measure: score on a scale
Week 4 Number Analyzed 106 participants 105 participants
-1.01  (1.714) -1.08  (1.865)
Week 12 Number Analyzed 103 participants 103 participants
-1.08  (1.775) -1.26  (2.259)
Week 24 Number Analyzed 98 participants 101 participants
-1.18  (2.132) -1.09  (2.443)
3.Secondary Outcome
Title Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA).
Hide Description 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.
Time Frame Baseline to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All participants for whom Fatigue During Activities was measured.
Arm/Group Title Elamipretide Placebo
Hide Arm/Group Description:
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
Overall Number of Participants Analyzed 109 109
Mean (Standard Deviation)
Unit of Measure: score on a scale
Week 4 Number Analyzed 106 participants 105 participants
-0.48  (0.890) -0.60  (0.998)
Week 12 Number Analyzed 103 participants 103 participants
-0.57  (0.923) -0.67  (1.199)
Week 24 Number Analyzed 98 participants 101 participants
-0.64  (1.151) -0.59  (1.360)
4.Secondary Outcome
Title Neuro-QoL Fatigue Activities of Daily Living
Hide Description 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.
Time Frame Baseline to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All participants for whom Change From Baseline in Neuro-QoL Fatigue Activities of Daily Living by Visit was measured.
Arm/Group Title Elamipretide Placebo
Hide Arm/Group Description:
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
Overall Number of Participants Analyzed 109 109
Mean (Standard Deviation)
Unit of Measure: score on a scale
Week 4 Number Analyzed 109 participants 106 participants
-2.5  (5.33) -2.7  (5.31)
Week 12 Number Analyzed 106 participants 104 participants
-2.9  (6.00) -2.9  (5.13)
Week 24 Number Analyzed 101 participants 103 participants
-2.7  (6.06) -2.1  (5.39)
5.Secondary Outcome
Title Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment
Hide Description 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.
Time Frame Baseline to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All participants for whom Most Bothersome Symptom Score (PMMSA) by Visit was measured.
Arm/Group Title Elamipretide Placebo
Hide Arm/Group Description:
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
Overall Number of Participants Analyzed 109 109
Mean (Standard Deviation)
Unit of Measure: score on a scale
Week 4 Number Analyzed 106 participants 105 participants
-0.21  (0.492) -0.24  (0.535)
Week 12 Number Analyzed 103 participants 103 participants
-0.24  (0.535) -0.34  (0.656)
Week 24 Number Analyzed 98 participants 101 participants
-0.25  (0.618) -0.30  (0.713)
6.Secondary Outcome
Title Neuro-QoL Fatigue Short Form Score
Hide Description Change From Baseline in Neuro-QoL Fatigue - Short Form: 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. The lowest possible raw score is 8 (8 x 1); the highest possible raw score is 40 (8 x 5). Lower scores means less fatigue and better outcome; higher means more fatigue, worse outcome. The Neuro-QoL Fatigue Short Form T-scores are calculated from the short form scoring table provided by the instrument authors (Neuro-QoL User Manual, 2015). This table converts summed scores to an Item Response Theory metric.
Time Frame 24 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All participants for whom Neuro-QoL Fatigue Short Form Score was measured.
Arm/Group Title Elamipretide Placebo
Hide Arm/Group Description:
Double-blind Period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
Double-Blind Period: Placebo comparator: 40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks
Overall Number of Participants Analyzed 109 109
Mean (Standard Deviation)
Unit of Measure: score on a scale
Week 4 Number Analyzed 109 participants 106 participants
-3.37  (5.077) -2.89  (5.915)
Week 12 Number Analyzed 106 participants 104 participants
-3.43  (5.681) -3.60  (5.614)
Week 24 Number Analyzed 101 participants 103 participants
-2.68  (5.769) -2.61  (5.773)
Time Frame Double-blind period: 24 weeks and continued with Open-label period: up to 144 weeks.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Double-Blind Elamipretide Double-Blind Placebo Open-Label Elamipretide Open-Label Placebo
Hide Arm/Group Description 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

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: 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 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.
All-Cause Mortality
Double-Blind Elamipretide Double-Blind Placebo Open-Label Elamipretide Open-Label Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/109 (0.00%)   0/109 (0.00%)   0/93 (0.00%)   0/103 (0.00%) 
Hide Serious Adverse Events
Double-Blind Elamipretide Double-Blind Placebo Open-Label Elamipretide Open-Label Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   5/109 (4.59%)   3/109 (2.75%)   12/93 (12.90%)   9/103 (8.74%) 
Cardiac disorders         
Acute myocardial infarction  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Coronary artery dissection  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Atrioventricular block complete  2  0/109 (0.00%)  0/109 (0.00%)  0/93 (0.00%)  1/103 (0.97%) 
Congenital, familial and genetic disorders         
MELAS syndrome  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Ear and labyrinth disorders         
Vertigo  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Gastrointestinal disorders         
Gastroenteritis  2  0/109 (0.00%)  1/109 (0.92%)  0/93 (0.00%)  0/103 (0.00%) 
Intestinal pseudo-obstruction  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Duodenal ulcer haemorrhage  2  0/109 (0.00%)  0/109 (0.00%)  0/93 (0.00%)  1/103 (0.97%) 
General disorders         
Chest pain  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Hepatobiliary disorders         
Cholelithiasis  2  0/109 (0.00%)  0/109 (0.00%)  0/93 (0.00%)  1/103 (0.97%) 
Infections and infestations         
Cellulitis  2  1/109 (0.92%)  0/109 (0.00%)  0/93 (0.00%)  0/103 (0.00%) 
Diverticulitis  2  1/109 (0.92%)  0/109 (0.00%)  0/93 (0.00%)  0/103 (0.00%) 
Viral sinusitis  2  1/109 (0.92%)  0/109 (0.00%)  0/93 (0.00%)  0/103 (0.00%) 
Sinusitis  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Injury, poisoning and procedural complications         
Humerus fracture  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Vaginal laceration  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Metabolism and nutrition disorders         
Dehydration  2  1/109 (0.92%)  0/109 (0.00%)  0/93 (0.00%)  0/103 (0.00%) 
Lactic Acidosis  2  1/109 (0.92%)  0/109 (0.00%)  0/93 (0.00%)  0/103 (0.00%) 
Musculoskeletal and connective tissue disorders         
Rheumatoid arthritis  2  0/109 (0.00%)  1/109 (0.92%)  0/93 (0.00%)  1/103 (0.97%) 
Intervertebral disc disorder  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Breast cancer  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Papillary thyroid cancer  2  0/109 (0.00%)  0/109 (0.00%)  0/93 (0.00%)  1/103 (0.97%) 
Nervous system disorders         
Hypoaesthesia  2  0/109 (0.00%)  1/109 (0.92%)  0/93 (0.00%)  0/103 (0.00%) 
Psychogenic seizure  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Cerebrovascular accident  2  0/109 (0.00%)  0/109 (0.00%)  0/93 (0.00%)  2/103 (1.94%) 
Spinal cord compression  2  0/109 (0.00%)  0/109 (0.00%)  0/93 (0.00%)  1/103 (0.97%) 
Hemiplegia  2  0/109 (0.00%)  0/109 (0.00%)  0/93 (0.00%)  1/103 (0.97%) 
Psychiatric disorders         
Suicidal Ideation  1  1/109 (0.92%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Renal and urinary disorders         
Urinary retention  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Chronic obstructive pulmonary disease  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Asthma  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Skin and subcutaneous tissue disorders         
Urticaria  2  0/109 (0.00%)  0/109 (0.00%)  0/93 (0.00%)  1/103 (0.97%) 
Vascular disorders         
Hypertension  2  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  0/103 (0.00%) 
Labile blood pressure  2  0/109 (0.00%)  0/109 (0.00%)  0/93 (0.00%)  1/103 (0.97%) 
1
Term from vocabulary, MedDRA 22.0
2
Term from vocabulary, MedDRA (22.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
Double-Blind Elamipretide Double-Blind Placebo Open-Label Elamipretide Open-Label Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   107/109 (98.17%)   83/109 (76.15%)   88/93 (94.62%)   102/103 (99.03%) 
Blood and lymphatic system disorders         
Eosinophilia  1  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  9/103 (8.74%) 
Gastrointestinal disorders         
Nausea  1  5/109 (4.59%)  8/109 (7.34%)  4/93 (4.30%)  2/103 (1.94%) 
Constipation  1  0/109 (0.00%)  0/109 (0.00%)  3/93 (3.23%)  2/103 (1.94%) 
Abdominal Pain  1  0/109 (0.00%)  0/109 (0.00%)  3/93 (3.23%)  1/103 (0.97%) 
General disorders         
Injection site erythema  1  94/109 (86.24%)  31/109 (28.44%)  66/93 (70.97%)  87/103 (84.47%) 
Injection site pruritus  1  82/109 (75.23%)  10/109 (9.17%)  57/93 (61.29%)  81/103 (78.64%) 
Injection site pain  1  43/109 (39.45%)  20/109 (18.35%)  32/93 (34.41%)  37/103 (35.92%) 
Injection site swelling  1  42/109 (38.53%)  7/109 (6.42%)  29/93 (31.18%)  20/103 (19.42%) 
Injection site induration  1  31/109 (28.44%)  6/109 (5.50%)  25/93 (26.88%)  41/103 (39.81%) 
injection site bruising  1  9/109 (8.26%)  18/109 (16.51%)  5/93 (5.38%)  15/103 (14.56%) 
injection site haemorrhage  1  7/109 (6.42%)  10/109 (9.17%)  2/93 (2.15%)  8/103 (7.77%) 
injection site urticaria  1  14/109 (12.84%)  0/109 (0.00%)  12/93 (12.90%)  8/103 (7.77%) 
injection site nodule  1  11/109 (10.09%)  2/109 (1.83%)  8/93 (8.60%)  8/103 (7.77%) 
injection site mass  1  9/109 (8.26%)  2/109 (1.83%)  4/93 (4.30%)  4/103 (3.88%) 
Fatigue  1  4/109 (3.67%)  4/109 (3.67%)  4/93 (4.30%)  4/103 (3.88%) 
injection site haematoma  1  0/109 (0.00%)  7/109 (6.42%)  0/93 (0.00%)  0/103 (0.00%) 
Pyrexia  1  2/109 (1.83%)  3/109 (2.75%)  0/93 (0.00%)  0/103 (0.00%) 
Asthenia  1  0/109 (0.00%)  0/109 (0.00%)  3/93 (3.23%)  2/103 (1.94%) 
Injection site injury  1  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  4/103 (3.88%) 
Infections and infestations         
Upper respiratory tract Infection  1  7/109 (6.42%)  7/109 (6.42%)  7/93 (7.53%)  7/103 (6.80%) 
Nasopharyngitis  1  8/109 (7.34%)  2/109 (1.83%)  6/93 (6.45%)  2/103 (1.94%) 
Sinusitis  1  2/109 (1.83%)  3/109 (2.75%)  2/93 (2.15%)  4/103 (3.88%) 
Influenza  1  0/109 (0.00%)  0/109 (0.00%)  2/93 (2.15%)  2/103 (1.94%) 
Pneumonia  1  0/109 (0.00%)  0/109 (0.00%)  2/93 (2.15%)  2/103 (1.94%) 
Urinary Tract Infection  1  0/109 (0.00%)  0/109 (0.00%)  4/93 (4.30%)  0/103 (0.00%) 
Injury, poisoning and procedural complications         
Fall  1  6/109 (5.50%)  3/109 (2.75%)  8/93 (8.60%)  7/103 (6.80%) 
Skin Abrasion  1  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  3/103 (2.91%) 
Investigations         
Eosinophil count increased  1  7/109 (6.42%)  0/109 (0.00%)  7/93 (7.53%)  7/103 (6.80%) 
Blood creatine phosphokinase increased  1  0/109 (0.00%)  0/109 (0.00%)  4/93 (4.30%)  1/103 (0.97%) 
Weight Decreased  1  0/109 (0.00%)  0/109 (0.00%)  2/93 (2.15%)  3/103 (2.91%) 
Blood glucose increased  1  0/109 (0.00%)  0/109 (0.00%)  2/93 (2.15%)  2/103 (1.94%) 
Blood lactic acid increased  1  0/109 (0.00%)  0/109 (0.00%)  2/93 (2.15%)  2/103 (1.94%) 
Musculoskeletal and connective tissue disorders         
Pain in extremity  1  1/109 (0.92%)  5/109 (4.59%)  2/93 (2.15%)  5/103 (4.85%) 
Back Pain  1  0/109 (0.00%)  0/109 (0.00%)  4/93 (4.30%)  2/103 (1.94%) 
Arthralgia  1  0/109 (0.00%)  0/109 (0.00%)  3/93 (3.23%)  2/103 (1.94%) 
Muscle Spasms  1  0/109 (0.00%)  0/109 (0.00%)  4/93 (4.30%)  0/103 (0.00%) 
Muscular Weakness  1  0/109 (0.00%)  0/109 (0.00%)  3/93 (3.23%)  1/103 (0.97%) 
Nervous system disorders         
Headache  1  8/109 (7.34%)  4/109 (3.67%)  5/93 (5.38%)  6/103 (5.83%) 
Dizziness  1  6/109 (5.50%)  3/109 (2.75%)  1/93 (1.08%)  3/103 (2.91%) 
Diarrhoea  1  3/109 (2.75%)  9/109 (8.26%)  3/93 (3.23%)  6/103 (5.83%) 
Hypoaesthesia  1  0/109 (0.00%)  0/109 (0.00%)  1/93 (1.08%)  3/103 (2.91%) 
Migraine  1  0/109 (0.00%)  0/109 (0.00%)  2/93 (2.15%)  2/103 (1.94%) 
Respiratory, thoracic and mediastinal disorders         
Cough  1  4/109 (3.67%)  1/109 (0.92%)  3/93 (3.23%)  3/103 (2.91%) 
Skin and subcutaneous tissue disorders         
Rash  1  0/109 (0.00%)  0/109 (0.00%)  2/93 (2.15%)  2/103 (1.94%) 
Vascular disorders         
Hypertension  1  0/109 (0.00%)  0/109 (0.00%)  2/93 (2.15%)  3/103 (2.91%) 
1
Term from vocabulary, MedDRA (22.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jim Carr, Pharm.D. Chief Clinical Development Officer
Organization: Stealth BioTherapeutics, Inc
Phone: 1-617-600-6888
EMail: jim.carr@stealthbt.com
Layout table for additonal information
Responsible Party: Stealth BioTherapeutics Inc.
ClinicalTrials.gov Identifier: NCT03323749    
Other Study ID Numbers: SPIMM-301
First Submitted: October 12, 2017
First Posted: October 27, 2017
Results First Submitted: February 8, 2021
Results First Posted: April 2, 2021
Last Update Posted: April 2, 2021