Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy (NCT02367014) | Clinical Trial Compass
CompletedPhase 1/2
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
United States36 participantsStarted 2015-02
Plain-language 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.
Who can participate
Age range
16 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 diastoli…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT)
Timeframe: Assessed at Baseline, Day 5 (end-of-treatment visit)