SPIMD-301: Efficacy and Safety of Elamipretide in Subjects with Primary Mitochondrial Disease

Status: Closed to recruitment. In analysis.

Stealth Biotherapeutics logo, a power icon with a mitochondrian for the switch

Background

Primary mitochondrial disease (PMD) most commonly affects tissues in the body that require lots of energy, including the brain, muscle, heart, retina and cochlea, to name a few. Patients who present with PMD during adulthood tend to demonstrate the following clinical signs and symptoms: muscle weakness, damage to nerves (peripheral neuropathy), coordination, balance and speech (ataxia) and progressive external ophthalmoplegia (PEO).

There are currently no approved treatments for patients with PMD. Elamipretide is a drug that has been shown to improve cell and organ function across a spectrum of diseases including PMD. It does this by targeting the inner lining (membrane) of mitochondria where energy production occurs and stabilises it, leading to overall improvement in the function of the cell and organ.

Objectives

The aims for this study are to:

  • Evaluate the effect of the study drug (elamipretide vs placebo) on the distance walked (in meters) on the 6-Minute Walk Test (6MWT).
  • Evaluate the effect of the study drug (elamipretide vs placebo) measured by changes in the following study assessments:
    • Total time (in seconds) the Five-Times Sit-to-Stand Test (5XSST)
    • Total time (in seconds) the Triple Timed Up-and-Go Test (3TUG)
    • Patient Global Impression (PGI) of Change Scale
  • Evaluate the safety and tolerability of the study drug (elamipretide).

Research aims:

The main aim of this study is to learn how well the study drug works and how safe the study drug is compared with placebo. A placebo is an inactive material that looks like the study drug but does not have any active study drug.

Who can take part?

  • Individuals with a confirmed nuclear mitochondrial DNA mutation (aged 18 to 70 years old).
  • Individuals with myopathy, progressive external opthalmoplegia (PEO), exercise intolerance and/or skeletal muscle weakness.

What is involved?

Participants will be required to take part in three parts of the study (approximate total of 56 weeks):

  • Screening period (Duration: up to 28 days)
    • Treatment period (Duration: 48 weeks)
    • Follow-up period (Duration: 4 weeks)

We will ask participants to:

  • Attend at least 7 visits to Addenbrooke’s Hospital, Cambridge over a 56-week period
  • Inject study medication (elamipretide or placebo) daily (at the same time of day) during the treatment period, following instructions provided
  • Complete all study assessments, including medical history, physical examinations, blood samples, quality of life questionnaires and exercise assessments

Study Website

For further information about the study, please visit the Stealth BioTherapeutics webpages.

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