Trial · NCT02367014
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
NCT02367014, Phase 1, Phase 2, Completed, Stealth BioTherapeutics Inc.; results first posted 2019-12-27
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy. Trials, record NCT02367014. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02367014
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
- NCT number
- NCT02367014
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 36 participants, actual
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Conditions
- Mitochondrial Myopathy
- Interventions
- elamipretide (low dose), elamipretide (intermediate dose), elamipretide (high dose), Placebo
- First posted
- 2015-02-20
- Last updated
- 2019-12-27
- Results first posted
- 2019-12-27
- Limitations and caveats
CPET was not performed on all subjects.Sensitivity analysis on change from Baseline in pre-and post-exercise lactate levels was not completed.Data from study sites were combined for efficacy and safety analyses.No site- effect analysis was performed.
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) | Assessed at Baseline, Day 5 (end-of-treatment visit) | Change in distance walked as measured by meters on the 6-minute walk test from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
| Secondary | Change in Maximum Oxygen Uptake (ml/kg/Min) | Baseline, Day 5 | Change in maximum oxygen uptake as measured by mL/kg/min from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
| Secondary | Change in Ventilatory Efficiency (VE/VCO2 Slope) | Baseline, Day 5 | Change in ventilatory efficiency as measured by the VE/VCO2 slope from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
| Secondary | Change in Aerobic Efficiency (ΔO2 Consumption/Δ Work Ratio) | Baseline, Day 5 | Change in aerobic efficiency as measured by ΔO2 consumption/Δ work ratio from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
| Secondary | Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio) | Baseline, Day 5 | Change in oxygen utilization as measured by ΔVO2/ΔlogVE ratio from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
| Secondary | Change in Oxygen Uptake Kinetics (Mean Response Time as Measured by Seconds) | Baseline, Day 5 | Change in oxygen uptake kinetics (mean response time) as measured by seconds from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
| Secondary | Change in Pre-exercise Lactate Levels (mg/dL) | Baseline, Day 5 | Change in pre-exercise lactate levels as measured by mg/dL from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
| Secondary | Change in Post-exercise Lactate Levels (mg/dL) | Baseline, Day 5 | Change in post-exercise lactate levels as measured by mg/dL from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
| Secondary | Change in Peak Respiratory Exchange Ratio (VCO2/VO2) | Baseline, Day 5 | Change in peak respiratory exchange ratio as measured by VCO2/VO2 from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
| Secondary | Change in Peak Respiratory Rate (Breaths/Min) | Baseline, Day 5 | Change in peak respiratory rate as measured by breaths/min from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit). |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) | Assessed at Baseline, Day 5 (end-of-treatment visit) | meters | LEAST_SQUARES_MEAN | |
| SECONDARY | Change in Maximum Oxygen Uptake (ml/kg/Min) | Baseline, Day 5 | All participants for whom maximum oxygen uptake was measured at baseline and Day 5 | ml/kg/min | MEAN |
| SECONDARY | Change in Ventilatory Efficiency (VE/VCO2 Slope) | Baseline, Day 5 | All participants for whom VE/VCO2 slope measurements were recorded at Baseline and Day 5 | VE/VCO2 slope | MEAN |
| SECONDARY | Change in Aerobic Efficiency (ΔO2 Consumption/Δ Work Ratio) | Baseline, Day 5 | All participants for whom aerobic efficiency measurements were recorded at Baseline and Day 5 | ΔO2 consumption/Δwork ratio | MEAN |
| SECONDARY | Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio) | Baseline, Day 5 | All participants for whom oxygen utilization measurements were recorded at Baseline and Day 5 | ΔVO2/ΔlogVE ratio | MEAN |
| SECONDARY | Change in Oxygen Uptake Kinetics (Mean Response Time as Measured by Seconds) | Baseline, Day 5 | Participants for whom oxygen uptake kinetics was measured | seconds | MEAN |
| SECONDARY | Change in Pre-exercise Lactate Levels (mg/dL) | Baseline, Day 5 | Participants for whom pre-exercise lactate levels were measured | mg/dL | MEAN |
| SECONDARY | Change in Post-exercise Lactate Levels (mg/dL) | Baseline, Day 5 | Participants for whom post- exercise lactate levels were measured | mg/dL | MEAN |
| SECONDARY | Change in Peak Respiratory Exchange Ratio (VCO2/VO2) | Baseline, Day 5 | Participants for whom peak respiratory exchange ratio was measured. | VCO2/VO2 ratio | MEAN |
| SECONDARY | Change in Peak Respiratory Rate (Breaths/Min) | Baseline, Day 5 | Participants for whom peak respiratory rate was measured | breaths/min | MEAN |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group112
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) | Low Dose | 13.5 | Full Range -72 to 86 | meters | |
| Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) | Intermediate Dose | 36.5 | Full Range -20 to 107 | meters | |
| Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) | High Dose | 64.5 | Full Range 4 to 133 | meters | |
| Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) | Placebo | 20.4 | Full Range -37 to 90 | meters | |
| Change in Maximum Oxygen Uptake (ml/kg/Min) | Low Dose | 0.35 | Standard Deviation 2.617 | ml/kg/min | |
| Change in Maximum Oxygen Uptake (ml/kg/Min) | Intermediate | 2.36 | Standard Deviation 2.714 | ml/kg/min | |
| Change in Maximum Oxygen Uptake (ml/kg/Min) | High | 0.64 | Standard Deviation 3.121 | ml/kg/min | |
| Change in Maximum Oxygen Uptake (ml/kg/Min) | Placebo | 2.14 | Standard Deviation 3.137 | ml/kg/min | |
| Change in Ventilatory Efficiency (VE/VCO2 Slope) | Low Dose | 0.749 | Standard Deviation 2.9412 | VE/VCO2 slope | |
| Change in Ventilatory Efficiency (VE/VCO2 Slope) | Intermediate Dose | -0.671 | Standard Deviation 5.4024 | VE/VCO2 slope |
1 to 10 of 112Source ClinicalTrials.gov
See all 112 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Low Dose | 9 | |
| Overall Study | STARTED | Intermediate Dose | 9 | |
| Overall Study | STARTED | High Dose | 9 | |
| Overall Study | STARTED | Placebo | 9 | |
| Overall Study | COMPLETED | Low Dose | 9 | |
| Overall Study | COMPLETED | Intermediate Dose | 9 | |
| Overall Study | COMPLETED | High Dose | 9 | |
| Overall Study | COMPLETED | Placebo | 9 | |
| Overall Study | NOT COMPLETED | Low Dose | 0 | |
| Overall Study | NOT COMPLETED | Intermediate Dose | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events144
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Headache | Nervous system disorders | Low Dose | 1 | 9 | |
| other | Headache | Nervous system disorders | Intermediate Dose | 1 | 9 | |
| other | Headache | Nervous system disorders | High Dose | 2 | 9 | |
| other | Headache | Nervous system disorders | Placebo | 2 | 9 | |
| other | Dizziness | Nervous system disorders | Low Dose | 2 | 9 | |
| other | Dizziness | Nervous system disorders | Intermediate Dose | 1 | 9 | |
| other | Dizziness | Nervous system disorders | High Dose | 0 | 9 | |
| other | Dizziness | Nervous system disorders | Placebo | 0 | 9 | |
| other | Abdominal pain | Gastrointestinal disorders | Low Dose | 0 | 9 | |
| other | Abdominal pain | Gastrointestinal disorders | Intermediate Dose | 2 | 9 |
1 to 10 of 144Source ClinicalTrials.gov
See all 144 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Low Dose | 0 | 9 |
| Total, all-cause mortality | Intermediate Dose | 0 | 9 |
| Total, all-cause mortality | High Dose | 0 | 9 |
| Total, all-cause mortality | Placebo | 0 | 9 |
| Total, other adverse events | Low Dose | 7 | 9 |
| Total, other adverse events | Intermediate Dose | 7 | 9 |
| Total, other adverse events | High Dose | 5 | 9 |
| Total, other adverse events | Placebo | 5 | 9 |
| Total, serious adverse events | Low Dose | 0 | 9 |
| Total, serious adverse events | Intermediate Dose | 0 | 9 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02367014
GET https://conductscience.org/api/trials/v1/NCT02367014/registered?page=2