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Registration trial did not meet the primary end points
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This is a Phase 2 Open-Label extension study to evaluate the long term safety and tolerability of daily elamipretide injections in patients with genetically confirmed Primary Mitochondrial Disease who previously participated in the SPIMM-202 Clinical Trial
This open-label, non-comparative, extension trial will enroll subjects with genetically confirmed PMD who have completed the End-of-Study Visit in the SPIMM-202 trial. Subjects who do not discontinue or withdraw from the trial will receive treatment with 40 mg SC elamipretide for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| elamipretide | Experimental | Open-label once daily subcutaneous injection of 40mg elamipretide |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| elamipretide | Drug | 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD. |
| Measure | Description | Time Frame |
|---|---|---|
| Distance Walked on Six Minute Walk Test | Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) | EuroQol 5 dimensions 5 levels (EQ-5D-5L) by visit. Count of participants per arm, or severity level of dimension. Measures 5 dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. Score ranges from 5-25 with lower score=better outcome, and higher score means a worse outcome. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jim Carr, PharmD | Stealth BioTherapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California | San Diego | California | 92123 | United States | ||
| Massachusetts General Hospital |
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| ID | Title | Description |
|---|---|---|
| FG000 | Elamipretide | Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jan 29, 2019 |
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|
| Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| Euroquol Visual Analog Scale | Euroquol Visual Analog Scale (EQ VAS): A patient-reported measure. The visual scale is numbered from 0 to 100, participants are directed as follows: "100 means the best health you can imagine, 0 means the worst health you can imagine. Mark an X on the scale to indicate how your health is TODAY." Higher scores mean better outcome, lower scores mean worse outcome. | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| NeuroQOL Fatigue Questionnaire | Change from baseline in NeuroQOL Fatigue Questionnaire by visit. Questionnaire is comprised of 8 questions measuring participants incidence of fatigue affecting activities of daily living. Participants respond by choosing one of the following: 1=never, 2=rarely, 3=sometimes, 4=often, 5=always, with a lower score meaning a better outcome with fatigue affecting participant less, and high score meaning a worse outcome, with fatigue affecting the participant more. Total raw scores range from 8-40. Scores were converted to percentages: range0-100%. Change from baseline score: negative number means improved from baseline, positive number means worsened compared to baseline. | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| Patient Global Assessment : Categorical | Patient Global Assessment [PGA] Score by Visit, count of participants. Patient-reported current health status by visit. PGA Scale is as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means worse health status, means worse outcome. | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| Patient Global Assessment: Continuous Variable | Patient Global Assessment [PGA] Score by Visit, Patient-reported current health status by visit. PGA Scale is as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means worse health status, lower score means worse outcome. | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| Work Limitations Questionnaire | Work Limitations Questionnaire by visit. Comprised of 25 items, in 4 categories, or demands: Mental/Interpersonal, Output Tasks, Physical Tasks, and Time Management, measuring the degree to which employed individuals are experiencing limitations on-the-job due to their health problems, and health-related productivity loss. 1= All of the time (100%), 2= most of the time, 3=some of the time (about 50%), 4=slight bit of time, 5=none of the time (0%), 6=does not apply to my job. (Presenteeism). The WLQ items ask respondents to rate their level of difficulty or ability to perform specific job demands. Scale score range from 0 (limited none of the time=better outcome) to 100 (limited all of the time= worse outcome) and represent the reported amount of time in the prior two weeks respondents were limited on-the-job. | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| Physician Global Assessment Score (PhGA) by Visit | Physician Global Assessment- Mean Score by visit. PGA Scale asks physician to rate participant's current health status as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means lower health status, worse outcome; lower score means better health status, better outcome. | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| Physician Global Assessment (PhGA): Categorical | Physician Global Assessment [PhGA] Score by Visit, Physician-reported current health status by visit. PhGA Scale is as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means worse health status, means worse outcome. | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| PMMSA Individual Items | The PMMSA is a patient-reported outcome measure, assessing a participant's "worst feeling" of 10 symptoms during the last 24 hours: tiredness at rest, tiredness during activities, muscle weakness at rest, muscle weakness during activities, balance, vision, abdominal discomfort, muscle pain, numbness, and headache using a 4-point scale: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Max Score =40= worse outcome, Min score= 10= better outcome on scale. PMMSA Individual Items shows number or participants with mild, moderate, severe or no symptoms at all. | Baseline, Week 13, 26, 52, 104, 156, End of Study (Week 160) |
| Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) | Change from baseline in the Total Fatigue score Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) where participants report their "worst feeling" of 4 symptoms: Tiredness at rest, Tiredness during activities, Muscle weakness at rest, Muscle weakness during activities, at the end of each treatment period. Total fatigue score is sum of Q1 to Q4 only of PMMSA) and is scored as follows: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Total score range is 4-16; lower score means less fatigue and better outcome, higher score means more fatigue and worse outcome. Raw scores were transformed into percentages: 0-100%. Change from baseline: the lower the score, the better the outcome,=less fatigue; the higher the score, means worse outcome= more fatigue. | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) | Change from baseline in the Total Fatigue during Activities score Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) where participants report their "worst feeling" of 2 symptoms: Tiredness during activities, Muscle weakness during activities, at the end of each treatment period. Total fatigue score is sum of Q2 and Q4 only of PMMSA) and is scored as follows: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Total score range is 2-8; lower score means less fatigue and better outcome, higher score means more fatigue and worse outcome. Raw scores were transformed into percentages: 0-100%. Change from baseline: the lower the score, the better the outcome,=less fatigue; the higher the score, means worse outcome= more fatigue. | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| Boston |
| Massachusetts |
| 02114 |
| United States |
| Akron Children's Hospital | Akron | Ohio | 44308 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
|
All participants for whom Baseline measurements were taken.
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| ID | Title | Description |
|---|---|---|
| BG000 | Elamipretide | Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Baseline weight | Mean | Standard Deviation | kg |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Distance Walked on Six Minute Walk Test | Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit | All participants for whom Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit was measured. | Posted | Mean | Standard Deviation | meters | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
|
|
| |||||||||||||||||||||||||||
| Secondary | EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) | EuroQol 5 dimensions 5 levels (EQ-5D-5L) by visit. Count of participants per arm, or severity level of dimension. Measures 5 dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. Score ranges from 5-25 with lower score=better outcome, and higher score means a worse outcome. | All participants for whom EQ-5D-5L was measured. Trial was terminated early. | Posted | Count of Participants | Participants | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| ||||||||||||||||||||||||||||||
| Secondary | Euroquol Visual Analog Scale | Euroquol Visual Analog Scale (EQ VAS): A patient-reported measure. The visual scale is numbered from 0 to 100, participants are directed as follows: "100 means the best health you can imagine, 0 means the worst health you can imagine. Mark an X on the scale to indicate how your health is TODAY." Higher scores mean better outcome, lower scores mean worse outcome. | All participants for whom EQ VAS was measured. Trial was terminated early. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | NeuroQOL Fatigue Questionnaire | Change from baseline in NeuroQOL Fatigue Questionnaire by visit. Questionnaire is comprised of 8 questions measuring participants incidence of fatigue affecting activities of daily living. Participants respond by choosing one of the following: 1=never, 2=rarely, 3=sometimes, 4=often, 5=always, with a lower score meaning a better outcome with fatigue affecting participant less, and high score meaning a worse outcome, with fatigue affecting the participant more. Total raw scores range from 8-40. Scores were converted to percentages: range0-100%. Change from baseline score: negative number means improved from baseline, positive number means worsened compared to baseline. | All participants for whom Neuro-Qol Fatigue was measured. Trial was terminated early. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Patient Global Assessment : Categorical | Patient Global Assessment [PGA] Score by Visit, count of participants. Patient-reported current health status by visit. PGA Scale is as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means worse health status, means worse outcome. | All participants for whom PGA was measured. Trial was terminated early. | Posted | Count of Participants | Participants | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
|
| |||||||||||||||||||||||||||||
| Secondary | Patient Global Assessment: Continuous Variable | Patient Global Assessment [PGA] Score by Visit, Patient-reported current health status by visit. PGA Scale is as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means worse health status, lower score means worse outcome. | All participants for whom Change From Baseline in Patient Global Assessment was measured. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Work Limitations Questionnaire | Work Limitations Questionnaire by visit. Comprised of 25 items, in 4 categories, or demands: Mental/Interpersonal, Output Tasks, Physical Tasks, and Time Management, measuring the degree to which employed individuals are experiencing limitations on-the-job due to their health problems, and health-related productivity loss. 1= All of the time (100%), 2= most of the time, 3=some of the time (about 50%), 4=slight bit of time, 5=none of the time (0%), 6=does not apply to my job. (Presenteeism). The WLQ items ask respondents to rate their level of difficulty or ability to perform specific job demands. Scale score range from 0 (limited none of the time=better outcome) to 100 (limited all of the time= worse outcome) and represent the reported amount of time in the prior two weeks respondents were limited on-the-job. | All individuals for whom Work Limitations was measured. Trial was terminated early. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Physician Global Assessment Score (PhGA) by Visit | Physician Global Assessment- Mean Score by visit. PGA Scale asks physician to rate participant's current health status as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means lower health status, worse outcome; lower score means better health status, better outcome. | All participants for whom PhGA was measured. Trial was terminated early. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Physician Global Assessment (PhGA): Categorical | Physician Global Assessment [PhGA] Score by Visit, Physician-reported current health status by visit. PhGA Scale is as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means worse health status, means worse outcome. | All participants for whom PhGA was measured. Trial was terminated early. | Posted | Count of Participants | Participants | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
| ||||||||||||||||||||||||||||||
| Secondary | PMMSA Individual Items | The PMMSA is a patient-reported outcome measure, assessing a participant's "worst feeling" of 10 symptoms during the last 24 hours: tiredness at rest, tiredness during activities, muscle weakness at rest, muscle weakness during activities, balance, vision, abdominal discomfort, muscle pain, numbness, and headache using a 4-point scale: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Max Score =40= worse outcome, Min score= 10= better outcome on scale. PMMSA Individual Items shows number or participants with mild, moderate, severe or no symptoms at all. | All participants for which PMMSA was measured. Trial was terminated early. | Posted | Count of Participants | Participants | Baseline, Week 13, 26, 52, 104, 156, End of Study (Week 160) |
|
| |||||||||||||||||||||||||||||
| Secondary | Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) | Change from baseline in the Total Fatigue score Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) where participants report their "worst feeling" of 4 symptoms: Tiredness at rest, Tiredness during activities, Muscle weakness at rest, Muscle weakness during activities, at the end of each treatment period. Total fatigue score is sum of Q1 to Q4 only of PMMSA) and is scored as follows: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Total score range is 4-16; lower score means less fatigue and better outcome, higher score means more fatigue and worse outcome. Raw scores were transformed into percentages: 0-100%. Change from baseline: the lower the score, the better the outcome,=less fatigue; the higher the score, means worse outcome= more fatigue. | All participants for whom PMMSA was measured. Study terminated early. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) | Change from baseline in the Total Fatigue during Activities score Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) where participants report their "worst feeling" of 2 symptoms: Tiredness during activities, Muscle weakness during activities, at the end of each treatment period. Total fatigue score is sum of Q2 and Q4 only of PMMSA) and is scored as follows: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Total score range is 2-8; lower score means less fatigue and better outcome, higher score means more fatigue and worse outcome. Raw scores were transformed into percentages: 0-100%. Change from baseline: the lower the score, the better the outcome,=less fatigue; the higher the score, means worse outcome= more fatigue. | All participants for whom PMMSA was measured. Study terminated early. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks |
|
|
Baseline through end of study for up to 260 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Elamipretide | Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD. | 0 | 28 | 8 | 28 | 28 | 28 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pancreatitis acute | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Breast cancer stage I | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 22.1 | Systematic Assessment |
| |
| Hemiparesis | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Cerebrovascular accident | Vascular disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Hereditary optic atrophy | Congenital, familial and genetic disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Troponin increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tachycardia | Cardiac disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Impaired gastric emptying | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection site pruritus | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection site mass | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection site urticaria | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection site swelling | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection site rash | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 22.1 | Systematic Assessment |
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| Injection site haemorrhage | General disorders | MedDRA 22.1 | Systematic Assessment |
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| Injection site induration | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Fungal infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
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| Hordeolum | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
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| Postoperative wound infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Vaginal infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Concussion | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Ligament sprain | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Skin laceration | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Joint dislocation | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Blood lactic acid increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Eosinophil count increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Muscle fatigue | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Dizziness postural | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Dystonia | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Adjustment disorder with depressed mood | Psychiatric disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Apnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Dermal cyst | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Jim Carr, Pharm.D. Chief Clinical Development Officer | Stealth BioTherapeutics, Inc | 1-617-600-6888 | jim.carr@stealthbt.com |
| Feb 8, 2021 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| C506540 | arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
| Week 52 |
|
|
| Week 78 |
|
|
| Week 104 |
|
|
| Week 130 |
|
|
| Week 156 |
|
|
| End of Trial (Week 160) |
|
|
| OG004 | Extreme Problem | Participant reports symptom is an "extreme problem" |
|
|
|
|
|
|
|
| Counts |
|---|
| Participants |
|
|
|
| OG004 | Very Good | Number of participants the Physician rated current disease status as Very Good |
|
|
|
| Participants |
|
|
| Participants |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Poor |
|
| Very Good |
|
| Poor |
|
| Very Good |
|
| Poor |
|
| Very Good |
|
| Poor |
|
| Very Good |
|
| Poor |
|
| Very Good |
|
| Poor |
|
| Very Good |
|
| Poor |
|
| Very Good |
|
| Poor |
|
| Very Good |
|
|
|
|
|
|
|
|
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|
| Severe |
|