Phase 2
N=36
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
Mitochondrial Myopathy
Bottom Line
View on ClinicalTrials.gov: NCT02367014 ↗Enrolled (actual)
36
Serious AEs
0.0%
Results posted
Dec 2019
Primary outcome: Primary: Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) — 13.5; 36.5; 64.5; 20.4 meters
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- elamipretide (low dose) (Drug); elamipretide (intermediate dose) (Drug); elamipretide (high dose) (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 16+ yrs
- Sex
- All
- Sponsor
- Stealth BioTherapeutics Inc.
- Primary completion
- Apr 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) |
13.5; 36.5; 64.5; 20.4 | — |
| SECONDARY Change in Maximum Oxygen Uptake (ml/kg/Min) |
0.35; 2.36; 0.64; 2.14 | — |
| SECONDARY Change in Ventilatory Efficiency (VE/VCO2 Slope) |
0.749; -0.671; -1.681; -1.375 | — |
| SECONDARY Change in Aerobic Efficiency (ΔO2 Consumption/Δ Work Ratio) |
-0.234; 0.643; -0.214; 0.738 | — |
| SECONDARY Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio) |
-118.87; 184.99; 90.13; 166.50 | — |
| SECONDARY Change in Oxygen Uptake Kinetics (Mean Response Time as Measured by Seconds) |
6.40; -2.49; -9.75; -7.47 | — |
| SECONDARY Change in Pre-exercise Lactate Levels (mg/dL) |
3.4; 13.8; 0.4; 4.4 | — |
| SECONDARY Change in Post-exercise Lactate Levels (mg/dL) |
6.9; 10.6; 4.3; 8.4 | — |
| SECONDARY Change in Peak Respiratory Exchange Ratio (VCO2/VO2) |
0.109; 0.114; 0.010; 0.133 | — |
| SECONDARY Change in Peak Respiratory Rate (Breaths/Min) |
1.4; -0.7; 0.9; 8.2 | — |
| SECONDARY Change in Peak Ventilation (L/Min) |
2.70; 8.87; 2.49; 8.47 | — |
| SECONDARY Change in Peak Heart Rate (Beats/Min) |
10.1; 16.7; 4.5; 3.8 | — |
| SECONDARY Change in Peak Oxygen Saturation (% O2-saturated Hemoglobin) |
0.0; 0.1; 0.6; 0.3 | — |
| SECONDARY Change in Peak Systolic Blood Pressure (mmHg) |
16.7; -1.5; 16.0; -2.7 | — |
| SECONDARY Change in Peak Diastolic Blood Pressure (mmHg) |
0.6; -1.4; -2.5; -6.6 | — |
| SECONDARY Change in Peak Borg Dyspnea |
-2.00; -2.2; -1.4; -0.4 | — |
| SECONDARY Change in VO2 Anaerobic Threshold (mL) |
30.00; 10.8; 1.6; 42.0 | — |
| SECONDARY Change in Watts |
9.6; 13.9; 5.4; 4.4 | — |
| SECONDARY Change in Temperature (°C) |
-0.3; 0.2; 0.1; 0.0 | — |
| SECONDARY Change in ECG-PR Interval (Msec) |
-3.2; 8.3; 9.1; -1.6 | — |
| SECONDARY Change in ECG-QRS Complex (Msec) |
1.2; 2.3; -2.2; 0.8 | — |
| SECONDARY Change in ECG-QT Interval (Msec) |
-6.0; -4.8; -16.7; -1.3 | — |
| SECONDARY Change in ECG-QTc Interval (Msec) |
3.4; 1.3; -23.2; 6.7 | — |
| SECONDARY Number of Participants Who Had Suicide Ideation, Suicidal Behavior, or Non-suicidal Self-injurious Behavior Post-screening. |
0; 0; 0; 0 | — |
| SECONDARY Change in Creatine Phosphokinase (IU/L) |
-24.8; -161.7; -101.2; -154.9 | — |
| SECONDARY Change in Alanine Aminotransferase (ALT) (U/L) |
19.3; -3.0; -5.3; 1.1 | — |
| SECONDARY Change in Aspartate Aminotransferase (AST) (U/L) |
5.6; -2.2; -12.6; -4.4 | — |
| SECONDARY Change in Eosinophils (10^9 Cells/L) |
0.06; 0.08; -0.01; 0.04 | — |
Summary
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.
Eligibility Criteria
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 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.
Data sourced from ClinicalTrials.gov (NCT02367014). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.