A Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Mitochondrial Disease Previously Treated in the Stealth BioTherapeutics SPIMM-201 Study

2016-06-20 18:53:21 | BioPortfolio


This is a Phase 2, randomized, double-blind, placebo-controlled crossover study which will enroll up to 36 subjects (anticipated) with genetically confirmed mitochondrial disease who have completed participation in the SPIMM-201 (MMPOWER) study. Subjects will be randomized (1:1) to one of two sequence groups: 4-weeks of treatment with 40 mg elamipretide administered once daily subcutaneously in Treatment Period 1 followed by 4-weeks of treatment with placebo administered once daily subcutaneously in Treatment Period 2 (separated by 4-week washout period), or vice versa

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment


Primary Mitochondrial Disease


Elamipretide, Placebo


University of California
San Diego
United States


Enrolling by invitation


Stealth BioTherapeutics Inc.

Results (where available)

View Results


Published on BioPortfolio: 2016-06-20T18:53:21-0400

Clinical Trials [1005 Associated Clinical Trials listed on BioPortfolio]

A Trial to Evaluate the Safety and Efficacy of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Extension

This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in sub...

A Multicenter, Open-Label Phase 2 Extension Trial to Characterize the Long-term Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Disease (PMD)

This is a Phase 2 Open-Label extension study to evaluate the long term safety and tolerability of daily elamipretide injections in patients with genetically confirmed Primary Mitochondrial...

The Effect of Multiple Injections of Elamipretide on Various Measures of Heart Function in Patients With Chronic Heart Failure With a Reduced Ejection Fraction

This is multi-center, randomized, double-blind, placebo-controlled study in patients with stable heart failure with reduced ejection fraction (HFrEF) to evaluate the effects of multiple su...

A Phase 2 Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Elamipretide in Subjects With Age-related Macular Degeneration With Geographic Atrophy

A randomized, double-masked, placebo controlled study to evaluate the safety, efficacy and pharmacokinetics of elamipretide in subjects with Age-Related Macular Degeneration with Geographi...

An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)

This is an observational study of patients with Primary Mitochondrial Disease with either signs or symptoms suggestive of myopathy. The Investigator will identify potential patients throug...

PubMed Articles [22920 Associated PubMed Articles listed on BioPortfolio]

Cocoa to Improve Walking Performance in Older People With Peripheral Artery Disease: The Cocoa-Pad Pilot Randomized Clinical Trial.

Cocoa and its major flavanol component, epicatechin, have therapeutic properties that may improve limb perfusion and increase calf muscle mitochondrial activity in people with lower extremity peripher...

Efficacy and placebo response of repetitive transcranial magnetic stimulation for primary insomnia.

Repetitive transcranial magnetic stimulation (rTMS) has been considered a promising technique for the treatment of primary insomnia. However, its efficacy and placebo response remains unclear due to l...

The "mitochondrial contact site and cristae organising system" (MICOS) in health and human disease.

The "Mitochondrial Contact Site and Cristae Organising System" (MICOS) is an essential protein complex that promotes the formation, maintenance and stability of mitochondrial cristae. As such, loss of...

Activities of mitochondrial respiratory chain complexes in platelets of patients with Alzheimer's disease and depressive disorder.

We analyzed activities of complex I, II, III, and IV, and citrate synthase (CS) in patients with major depressive disorder (MDD) or Alzheimer's disease (AD) presenting with or without depression. Asso...

Renovascular disease induces mitochondrial damage in swine scattered tubular cells.

Scattered tubular-like cells (STCs) contribute to repair neighboring injured renal tubular cells. Mitochondria mediate STC biology and function, but might be injured by the ambient milieu. We hypothes...

Medical and Biotech [MESH] Definitions

In vitro fertilization technique that uses mitochondrial DNA from a healthy donor in order to prevent the transmission of MITOCHONDRIAL DISEASE.

Specific practices for the prevention of disease or mental disorders in susceptible individuals or populations. These include HEALTH PROMOTION, including mental health; protective procedures, such as COMMUNICABLE DISEASE CONTROL; and monitoring and regulation of ENVIRONMENTAL POLLUTANTS. Primary prevention is to be distinguished from SECONDARY PREVENTION and TERTIARY PREVENTION.

Mitochondrial anion carrier proteins that function as dimers and form proton channels in the INNER MITOCHONDRIAL MEMBRANE which creates proton leaks and uncouples OXIDATIVE PHOSPHORYLATION from ATP synthesis, resulting in the generation of heat instead of ATP.

Mucocellular carcinoma of the ovary, usually metastatic from the gastrointestinal tract, characterized by areas of mucoid degeneration and the presence of signet-ring-like cells. It accounts for 30%-40% of metastatic cancers to the ovaries and possibly 1%-2% of all malignant ovarian tumors. The lesions may not be discovered until the primary disease is advanced, and most patients die of their disease within a year. In some cases, a primary tumor is not found. (From Dorland, 27th ed; Holland et al., Cancer Medicine, 3d ed, p1685)

Misunderstanding among individuals, frequently research subjects, of scientific methods such as randomization and placebo controls.

More From BioPortfolio on "A Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Mitochondrial Disease Previously Treated in the Stealth BioTherapeutics SPIMM-201 Study"

Quick Search

Relevant Topic

Biotechnology Products
BioPortfolio lists over 550 biotechnology products - please open Direct topic pages: Actos Advair Biopharmaceuticals Biosimilars Biotherapeutics GMO Crops Lipitor ...

Searches Linking to this Trial