Current partner codePEPTIDESDE
PeptideStat
Menu

Vendors

Compare pricesVendor directoryVendor reviewsDiscount codesLab tests & COAs

Research

ResearchDatabaseClinical trials

Tools

All calculatorsReconstitutionAccumulationUnit converterPeptide chemistry
NCT02367014·Phase 1 / Phase 2·INTERVENTIONAL

Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy

ElamipretideOtherCompleted

Status

Completed

Phase

Phase 1 / Phase 2

Enrollment

36

Locations

4

Results

Posted

Publications

0

Study summary

What the protocol is testing.

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.

Full detailed description

This multi-center, randomized, double-blind, placebo-controlled study enrolled 36 subjects into 3 cohorts of 12 subjects each to evaluate treatment with 3 ascending doses of intravenous elamipretide (0.01, 0.10, and 0.25 mg/kg/hr infused for 2 hours). After each cohort, a Safety Monitoring Board (SMB) determined if dose escalation to the next higher dose of elamipretide was warranted. Each cohort went through 3 distinct periods: Screening, Treatment, and Follow-up. The Screening Period started with informed consent and may have lasted up to 40 days. During this period, screening procedures to determine subject eligibility for the study occurred, including confirmation of disease, which incorporated a committee review of the investigator-submitted diagnosis and genetic results. The Treatment Period began on Day 1 (Visit 2) and lasted for 5 days (until Day 5 \[Visit 6\]). Within each cohort, 9 subjects were randomized to active drug and 3 subjects were randomized to placebo on Day 1 and subjects received treatment once a day for 5 consecutive days. Safety, tolerability, and efficacy measures were performed at pre-specified times. The Follow-up Period began at the time of discharge on Day 5. Subjects returned to the study center for the Follow-up Visit on Day 7 (+1 day).

Interventions

Treatment arms and agents.

DRUG

elamipretide (low dose)

elamipretide (0.01 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days

DRUG

elamipretide (intermediate dose)

elamipretide (0.10 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days

DRUG

elamipretide (high dose)

elamipretide (0.25 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days

DRUG

Placebo

placebo (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

Timeline

From registration to results.

  1. First posted

    Feb 20, 2015

  2. Study start

    Feb 2015

  3. Primary completion

    Apr 2016

  4. Study completion

    Apr 2016

  5. Results posted

    Dec 27, 2019

  6. Registry updated

    Dec 27, 2019

Outcomes

What the study measures.

Primary outcomes

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

Time frame · 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 outcomes

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

Time frame · 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).

Change in Ventilatory Efficiency (VE/VCO2 Slope)

Time frame · 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).

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

Time frame · 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).

Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio)

Time frame · 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).

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

Time frame · 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).

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

Time frame · 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).

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

Time frame · 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).

Change in Peak Respiratory Exchange Ratio (VCO2/VO2)

Time frame · 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).

Change in Peak Respiratory Rate (Breaths/Min)

Time frame · 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).

Change in Peak Ventilation (L/Min)

Time frame · Baseline, Day 5

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).

Eligibility

Who can take part.

Minimum age
16 Years
Maximum age
65 Years
Sex
ALL
Healthy volunteers
No

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.

Study locations

4 registered sites.

United States. Showing up to 24 locations stored in the fast local snapshot.

University of California

San Diego, California, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Akron Children's Hospital

Akron, Ohio, United States

Children's Hospital of Pittsburg of UPMC

Pittsburgh, Pennsylvania, United States

Publications

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

ClinicalTrials.gov ↗Search PubMed ↗Search FDA ↗Search EMA ↗

Related trials

More studies on Elamipretide.

Related PeptideStat pages

Put the record in context.

Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.

TrialsElamipretide trial dashboard →