Primary outcomeLeast squares mean· Assessed at Baseline, Day 5 (end-of-treatment visit)
Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT)
| Group | Value (meters) |
|---|---|
| Low Dose | 13.5 |
| Intermediate Dose | 36.5 |
| High Dose | 64.5 |
| Placebo | 20.4 |
Trial · Interventional
CompletedPhase 1/2Results postedRegistry dataSafety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
Short name MMPOWER
NCTNCT02367014 (opens ClinicalTrials.gov in a new tab)Other idSPIMM-201
Overview
- Status
- Completed
- Phase
- Phase 1/2
- Enrollment
- 36 participants (actual)
- Sponsor
- Stealth BioTherapeutics Inc. · Industry
- Design
- Randomized · Quadruple masking
- Countries
1 country
United States
- Start
- Feb 2015
- Primary completion
- Apr 2016
- Completion
- Apr 2016
- First posted
- 20 Feb 2015 (estimated)
- Last update posted
- 27 Dec 2019
- Conditions
- Mitochondrial Myopathy
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
201520162017
- Ph 1/2
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of MTP-131 in this patient population.
Interventions
- elamipretide (low dose)Drug
- elamipretide (intermediate dose)Drug
- elamipretide (high dose)Drug
- PlaceboDrug
Arms4
Experimental
Low Dose
elamipretide 0.01 mg/kg/hr infused for 2 hours for 5 days
Experimental
Intermediate dose
elamipretide 0.10 mg/kg/hr infused for 2 hours for 5 days
Experimental
High dose
elamipretide 0.25 mg/kg/hr infused for 2 hours for 5 days
Placebo comparator
Placebo
In each cohort, subjects received either IV elamipretide given once daily for 2 hours for 5 days or matching placebo.
Primary outcomes1
Outcomes the sponsor listed as primary.
Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT)
Time frame · Assessed at Baseline, Day 5 (end-of-treatment visit)
Registry description
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 outcomes(20)
Change in Maximum Oxygen Uptake (ml/kg/Min)
Time frame · Baseline, Day 5
Registry description
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).
Change in Ventilatory Efficiency (VE/VCO2 Slope)
Time frame · Baseline, Day 5
Registry description
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).
Change in Aerobic Efficiency (ΔO2 Consumption/Δ Work Ratio)
Time frame · Baseline, Day 5
Registry description
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).
Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio)
Time frame · Baseline, Day 5
Registry description
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).
Change in Oxygen Uptake Kinetics (Mean Response Time as Measured by Seconds)
Time frame · Baseline, Day 5
Registry description
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).
Change in Pre-exercise Lactate Levels (mg/dL)
Time frame · Baseline, Day 5
Registry description
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).
Change in Post-exercise Lactate Levels (mg/dL)
Time frame · Baseline, Day 5
Registry description
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).
Change in Peak Respiratory Exchange Ratio (VCO2/VO2)
Time frame · Baseline, Day 5
Registry description
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).
Change in Peak Respiratory Rate (Breaths/Min)
Time frame · Baseline, Day 5
Registry description
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).
Change in Peak Ventilation (L/Min)
Time frame · Baseline, Day 5
Registry description
Change in peak ventilation as measured by L/min from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in Peak Heart Rate (Beats/Min)
Time frame · Baseline, Day 5
Registry description
Change in peak heart rate as measured by beats per minute from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in Peak Oxygen Saturation (% O2-saturated Hemoglobin)
Time frame · Baseline, Day 5
Registry description
Change in peak oxygen saturation as measured by percentage of O2-saturated hemoglobin from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in Peak Systolic Blood Pressure (mmHg)
Time frame · Baseline, Day 5
Registry description
Change in peak systolic blood pressure as measured by mmHg from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in Peak Diastolic Blood Pressure (mmHg)
Time frame · Baseline, Day 5
Registry description
Change in peak diastolic blood pressure as measured by mmHg from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in Peak Borg Dyspnea
Time frame · Baseline, Day 5
Registry description
Change in peak Borg dyspnea as measured by 0-10 with 0 meaning no breathlessness and 10 meaning maximal breathlessness from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in VO2 Anaerobic Threshold (mL)
Time frame · Baseline, Day 5
Registry description
Change in VO2 anaerobic threshold as measured by mL from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in Watts
Time frame · Baseline, Day 5
Registry description
Change in watts from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in Temperature (°C)
Time frame · Baseline, Day 5
Registry description
Change in temperature as measured by units of Celsius from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in ECG-PR Interval (Msec)
Time frame · Baseline, Day 5
Registry description
Change in PR interval as measured by ECG in milliseconds from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Change in ECG-QRS Complex (Msec)
Time frame · Baseline, Day 5
Registry description
Change in QRS complex as measured by ECG in msec from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(7)
Secondary outcomeMean· Baseline, Day 5
Change in Maximum Oxygen Uptake (ml/kg/Min)
| Group | Value (ml/kg/min) | Spread |
|---|---|---|
| Low Dose | 0.35 | 2.617 |
| Intermediate | 2.36 | 2.714 |
| High | 0.64 | 3.121 |
| Placebo | 2.14 | 3.137 |
Secondary outcomeMean· Baseline, Day 5
Change in Ventilatory Efficiency (VE/VCO2 Slope)
| Group | Value (VE/VCO2 slope) | Spread |
|---|---|---|
| Low Dose | 0.749 | 2.9412 |
| Intermediate Dose | -0.671 | 5.4024 |
| High Dose | -1.681 | 3.2834 |
| Placebo | -1.375 | 5.5801 |
Secondary outcomeMean· Baseline, Day 5
Change in Aerobic Efficiency (ΔO2 Consumption/Δ Work Ratio)
| Group | Value (ΔO2 consumption/Δwork ratio) | Spread |
|---|---|---|
| Low Dose | -0.234 | 4.3414 |
| Intermediate Dose | 0.643 | 2.7507 |
| High Dose | -0.214 | 1.9727 |
| Placebo | 0.738 | 3.5959 |
Secondary outcomeMean· Baseline, Day 5
Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio)
| Group | Value (ΔVO2/ΔlogVE ratio) | Spread |
|---|---|---|
| Low Dose | -118.87 | 229.105 |
| Intermediate Dose | 184.99 | 410.093 |
| High Dose | 90.13 | 116.281 |
| Placebo | 166.50 | 479.145 |
Secondary outcomeMean· Baseline, Day 5
Change in Oxygen Uptake Kinetics (Mean Response Time as Measured by Seconds)
| Group | Value (seconds) | Spread |
|---|---|---|
| Low Dose | 6.40 | 11.402 |
| Intermediate | -2.49 | 26.830 |
| High | -9.75 | 26.288 |
| Placebo | -7.47 | 14.357 |
Secondary outcomeMean· Baseline, Day 5
Change in Pre-exercise Lactate Levels (mg/dL)
| Group | Value (mg/dL) | Spread |
|---|---|---|
| Low Dose | 3.4 | 9.00 |
| Intermediate Dose | 13.8 | 18.30 |
| High Dose | 0.4 | 10.25 |
| Placebo | 4.4 | 14.06 |
Secondary outcomeMean· Baseline, Day 5
Change in Post-exercise Lactate Levels (mg/dL)
| Group | Value (mg/dL) | Spread |
|---|---|---|
| Low Dose | 6.9 | 11.87 |
| Intermediate Dose | 10.6 | 42.93 |
| High Dose | 4.3 | 22.10 |
| Placebo | 8.4 | 13.21 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Low DoseMTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days | 0 / 9 | 0 / 9 | 7 / 9 |
| Intermediate DoseMTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days | 0 / 9 | 0 / 9 | 7 / 9 |
| High DoseMTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days | 0 / 9 | 0 / 9 | 5 / 9 |
| PlaceboPlacebo: Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days | 0 / 9 | 0 / 9 | 5 / 9 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 16 Years up to 65 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
* Diagnosis of mitochondrial disease believed to impair the mitochondrial respiratory chain.
* Eligibility requires prior genetic confirmation of mitochondrial disease.
* Diagnosis of mitochondrial myopathy judged by the Investigators to be due to existing mitochondrial disease.
* Must be able to complete a Screening Visit 6MWT.
* Body mass index (BMI) score \>15.0 and \<35.0 kg/m2 at Screening Visit.
* Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the ICF until two months after the last dose of study drug.
Exclusion Criteria:
* Any prior or current medical condition that, in the judgment of the Investigator, would prevent the subject from safely participating in and/or completing all study requirements.
* Had any exclusionary Newcastle Mitochondrial Disease Adult Scale (NMDAS) scores at Screening Visit.
* Hospitalized (admitted as in-patient) within 1 month prior to the Baseline Visit.
* A history of type 1 diabetes mellitus (T1DM).
* Uncontrolled Type 1 (T1DM) or Type 2 diabetes mellitus (T2DM), in the opinion of the investigator.
* A creatinine clearance \<45 mL/min as calculated by the Cockcroft Gault equation.
* Requires pacemaker, defibrillator, or has undergone cardiac surgery within 2 years of Screening Visit.
* QTc elongation defined as a QTc \>450 msec in male subjects and \>480 msec in female subjects.
* Uncontrolled hypertension (\>160 mmHg systolic or \>100 mmHg diastolic) at Screening Visit.
* History of rhabdomyolysis defined as an acute rise in the serum creatine phosphokinase (CPK) value that, in the opinion of the investigator, caused clinically significant symptoms.
* Serum sodium more than 5 meq/L below the reference lower limit of normal at Screening Visit.
* Participated in another interventional clinical trial within 3 months of the screening visit or is currently enrolled in a non-interventional clinical trial judged by the Investigator to be incompatible with the current trial.
* Other protocol-defined inclusion/exclusion criteria may apply.
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