Safety, Tolerability, and Efficacy of MTP-131 for the Tre... | NCT02367014

Label	Type	Description	Intervention Names
Low Dose	Experimental	elamipretide 0.01 mg/kg/hr infused for 2 hours for 5 days	Drug: elamipretide (low dose)
Intermediate dose	Experimental	elamipretide 0.10 mg/kg/hr infused for 2 hours for 5 days	Drug: elamipretide (intermediate dose)
High dose	Experimental	elamipretide 0.25 mg/kg/hr infused for 2 hours for 5 days	Drug: elamipretide (high dose)
Placebo	Placebo Comparator	In each cohort, subjects received either IV elamipretide given once daily for 2 hours for 5 days or matching placebo.	Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
elamipretide (low dose)	Drug	elamipretide (0.01 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days	Low Dose

Measure	Description	Time Frame
Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT)	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).	Assessed at Baseline, Day 5 (end-of-treatment visit)

Measure	Description	Time Frame
Change in Maximum Oxygen Uptake (ml/kg/Min)	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).	Baseline, Day 5
Change in Ventilatory Efficiency (VE/VCO2 Slope)

Facility	Status	City	State	ZIP	Country	Contacts
University of California		San Diego	California	92093	United States
Massachusetts General Hospital

ID	Title	Description
FG000	Low Dose	MTP-131: MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
FG001	Intermediate Dose	MTP-131: MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days

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

Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT)

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).

Posted

Least Squares Mean

Full Range

meters

Assessed at Baseline, Day 5 (end-of-treatment visit)

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Units	Counts
Participants	OG0009 OG0019 OG0029 OG003

Title

Denominators

Categories

Title	Measurements
	OG00013.5(-72 to 86) OG00136.5(-20 to 107) OG00264.5(4 to 133) OG003

Secondary

Change in Maximum Oxygen Uptake (ml/kg/Min)

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).

All participants for whom maximum oxygen uptake was measured at baseline and Day 5

Posted

Mean

Standard Deviation

ml/kg/min

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Ventilatory Efficiency (VE/VCO2 Slope)

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).

All participants for whom VE/VCO2 slope measurements were recorded at Baseline and Day 5

Posted

Mean

Standard Deviation

VE/VCO2 slope

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Aerobic Efficiency (ΔO2 Consumption/Δ Work Ratio)

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).

All participants for whom aerobic efficiency measurements were recorded at Baseline and Day 5

Posted

Mean

Standard Deviation

ΔO2 consumption/Δwork ratio

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio)

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).

All participants for whom oxygen utilization measurements were recorded at Baseline and Day 5

Posted

Mean

Standard Deviation

ΔVO2/ΔlogVE ratio

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Oxygen Uptake Kinetics (Mean Response Time as Measured by Seconds)

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).

Participants for whom oxygen uptake kinetics was measured

Posted

Mean

Standard Deviation

seconds

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Pre-exercise Lactate Levels (mg/dL)

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).

Participants for whom pre-exercise lactate levels were measured

Posted

Mean

Standard Deviation

mg/dL

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Post-exercise Lactate Levels (mg/dL)

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).

Participants for whom post- exercise lactate levels were measured

Posted

Mean

Standard Deviation

mg/dL

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Peak Respiratory Exchange Ratio (VCO2/VO2)

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).

Participants for whom peak respiratory exchange ratio was measured.

Posted

Mean

Standard Deviation

VCO2/VO2 ratio

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Peak Respiratory Rate (Breaths/Min)

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).

Participants for whom peak respiratory rate was measured

Posted

Mean

Standard Deviation

breaths/min

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Peak Ventilation (L/Min)

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).

Participants for whom peak ventilation was measured.

Posted

Mean

Standard Deviation

L/min

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Peak Heart Rate (Beats/Min)

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).

Participants for whom peak heart rate was measured

Posted

Mean

Standard Deviation

beats/min

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Peak Oxygen Saturation (% O2-saturated Hemoglobin)

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).

Participants for whom peak oxygen saturation was measured.

Posted

Mean

Standard Deviation

percentage of O2-saturated hemoglobin

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Peak Systolic Blood Pressure (mmHg)

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).

Participants for whom peak systolic blood pressure was measured.

Posted

Mean

Standard Deviation

mmHg

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Peak Diastolic Blood Pressure (mmHg)

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).

Participants for whom peak diastolic blood pressure was measured

Posted

Mean

Standard Deviation

mmHg

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Peak Borg Dyspnea

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).

Participants for whom peak Borg dyspnea was measured.

Posted

Mean

Standard Deviation

Units on a scale

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in VO2 Anaerobic Threshold (mL)

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).

Participants for whom VO2 was measured.

Posted

Mean

Standard Deviation

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Watts

Change in watts from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).

Participants for whom watts was measured

Posted

Mean

Standard Deviation

watts

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Temperature (°C)

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).

Participants for whom temperature was measured

Posted

Mean

Standard Deviation

°C

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in ECG-PR Interval (Msec)

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).

Participants for whom PR interval was measured

Posted

Mean

Standard Deviation

msec

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in ECG-QRS Complex (Msec)

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).

Participants for whom QRS complex was measured

Posted

Mean

Standard Deviation

msec

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in ECG-QT Interval (Msec)

Change in QT interval as measured by ECG in msec from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).

Participants for whom QT interval was measured

Posted

Mean

Standard Deviation

msec

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in ECG-QTc Interval (Msec)

Change in QTc interval as measured by ECG in msec from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).

Participants for whom QTc interval was measured

Posted

Mean

Standard Deviation

msec

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Number of Participants Who Had Suicide Ideation, Suicidal Behavior, or Non-suicidal Self-injurious Behavior Post-screening.

Number of participants with suicide ideation, suicidal behavior, or non-suicidal self-injurious behavior post-screening as measured on Days 1-5, and Day 7 on the Columbia Suicide Severity Rating Scale (CSSRS). A yes/no binary response is utilized in the following ten categories: 1 - Wish to be Dead; 2 - Non-specific Active Suicidal Thoughts; 3 - Active Suicidal Ideation with Any Methods (Not Plan) without Intent to Act; 4 - Active Suicidal Ideation with Some Intent to Act, without Specific Plan; 5 - Active Suicidal Ideation with Specific Plan and Intent; 6 - Preparatory Acts or Behavior; 7 - Aborted Attempt; 8 - Interrupted Attempt; Category 9 - Actual Attempt (non-fatal); 10 - Completed Suicide. A yes/no binary response is also utilized in assessing self-injurious behavior without suicidal intent. A lower score means a better outcome whereas a higher score means a worse outcome.

Participants for whom CSSR was measured.

Posted

Count of Participants

Participants

Days 1-5 and Day 7.

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Creatine Phosphokinase (IU/L)

Change in Creatine Phosphokinase as measured by IU/L from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).

Participants for whom creatine phosphokinase was measured

Posted

Mean

Standard Deviation

IU/L

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Alanine Aminotransferase (ALT) (U/L)

Change in Alanine aminotransferase (ALT) as measured by (U/L) from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).

Participants for whom Alanine aminotransferase was measured

Posted

Mean

Standard Deviation

U/L

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Aspartate Aminotransferase (AST) (U/L)

Change in aspartate aminotransferase (AST) as measured by U/L from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).

Participants for whom aspartate aminotransferase was measured

Posted

Mean

Standard Deviation

U/L

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Secondary

Change in Eosinophils (10^9 Cells/L)

Change in eosinophils as measured by (10^9 cells/L) from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).

Participants for whom eosinophils was measured

Posted

Mean

Standard Deviation

10^9 cells/L

Baseline, Day 5

ID	Title	Description
OG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days
OG003	Placebo	Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

ID	Title	Description
BG000	Low Dose	MTP-131: MTP-131 (low dose) administered as single day intravenous infusion over 2 hours for 5 days
BG001	Intermediate Dose	MTP-131: MTP-131 (intermediate dose) administered as single day intravenous infusion over 2 hours for 5 days
BG002	High Dose	MTP-131: MTP-131 (high dose) administered as single day intravenous infusion over 2 hours for 5 days
BG003	Placebo	Placebo: Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days
BG004	Total	Total of all reporting groups

ID	Title	Description	Deaths (At Risk)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Low Dose	MTP-131 (low dose) 0.01 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days	9	9	7	9
EG001	Intermediate Dose	MTP-131 (intermediate dose) 0.10 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days	9	9	7	9
EG002	High Dose	MTP-131 (high dose) 0.25 mg/kg/hour administered as single day intravenous infusion over 2 hours for 5 days	9	9	5	9
EG003	Placebo	Placebo: Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days	9	9	5	9

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Headache	Nervous system disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0011 affected9 at risk EG0022 affected9 at risk EG0032 affected9 at risk
Dizziness	Nervous system disorders	MedDRA (19.0)	Systematic Assessment	EG0002 affected9 at risk EG0011 affected9 at risk EG0020 affected9 at risk EG003
Abdominal pain	Gastrointestinal disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0012 affected9 at risk EG0020 affected9 at risk EG003
Flatulence	Gastrointestinal disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Muscle spasms	Musculoskeletal and connective tissue disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Constipation	Gastrointestinal disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Nausea	Gastrointestinal disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0011 affected9 at risk EG0020 affected9 at risk EG003
Injection site bruising	General disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Thirst	General disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Vessel puncture site haemorrhage	General disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0011 affected9 at risk EG0020 affected9 at risk EG003
Nasopharyngitis	Infections and infestations	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Urinary tract infection	Infections and infestations	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0011 affected9 at risk EG0020 affected9 at risk EG003
Viral upper respiratory tract	Infections and infestations	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Contusion	Injury, poisoning and procedural complications	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Ligament sprain	Injury, poisoning and procedural complications	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Blood creatine phosphokinase increased	Investigations	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0021 affected9 at risk EG003
Eosinophil count increased	Investigations	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0011 affected9 at risk EG0020 affected9 at risk EG003
Hepatic enzyme increased	Investigations	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Liver function test increased	Investigations	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Increased appetite	Metabolism and nutrition disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Neck pain	Musculoskeletal and connective tissue disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0021 affected9 at risk EG003
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0011 affected9 at risk EG0020 affected9 at risk EG003
Somnolence	Nervous system disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Migraine	Nervous system disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0021 affected9 at risk EG003
Myoclonus	Nervous system disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0021 affected9 at risk EG003
Presyncope	Nervous system disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0011 affected9 at risk EG0020 affected9 at risk EG003
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0011 affected9 at risk EG0021 affected9 at risk EG003
Hyperhydrosis	Skin and subcutaneous tissue disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Pain of skin	Skin and subcutaneous tissue disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Petechiae	Skin and subcutaneous tissue disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0021 affected9 at risk EG003
Skin tightness	Skin and subcutaneous tissue disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Flushing	Vascular disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003
Hypotension	Vascular disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0011 affected9 at risk EG0020 affected9 at risk EG003
Pallor	Vascular disorders	MedDRA (19.0)	Systematic Assessment	EG0000 affected9 at risk EG0011 affected9 at risk EG0020 affected9 at risk EG003
Phlebitis	Vascular disorders	MedDRA (19.0)	Systematic Assessment	EG0001 affected9 at risk EG0010 affected9 at risk EG0020 affected9 at risk EG003

ID	Term
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases
D009468	Neuromuscular Diseases
D009422	Nervous System Diseases
D028361	Mitochondrial Diseases
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases