Summary

for people ages 16-80 (full criteria)
at La Jolla, California and other locations
study started
estimated completion:

Description

Summary

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 subjects with primary mitochondrial myopathy. This will be followed by an open-label treatment extension.

Official Title

A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension

Keywords

Primary Mitochondrial Myopathy Myopathy PMD Primary Mitochondrial Disease MTP-131 elamipretide Muscular Diseases Mitochondrial Myopathies elamipretide open label treatment

Eligibility

For people ages 16-80

PART 1:

Inclusion Criteria:

  • Willing and able to provide a signed informed consent form prior to participation in any trial-related procedures
  • Agrees to adhere to the trial requirements for the length of the trial, including the use of the elamipretide delivery system
  • Subject is ≥ 16 and ≤ 80 years of age
  • Enrolled in SPIMM-300
  • Diagnosed with PMM in the opinion of the investigator
  • Woman of childbearing potential must agree to use a highly effective method of birth control

Exclusion Criteria:

  • Subject has myopathic signs and or/symptoms due to a neuropathic process or gait problem that would interfere with the 6MWT, in the opinion of the Investigator
  • Female who are pregnant, planning to become pregnant, or breastfeeding/lactating
  • At Screening, the estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2

  • Subject has undergone an in-patient hospitalization within the 30 days prior to the Baseline Visit or has a planned hospitalization or a surgical procedure during the trial.
  • Subject has clinically significant cardiac disease or prior interventional procedure and/or respiratory disease (medical history or current clinical findings) within 3 months of the Baseline Visit, in the opinion of the Investigator.
  • Subject has QTc elongation (using the correction factor utilized at the clinical site)defined as a QTc >450 msec in male subjects and >480 msec in female subjects.
  • ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following:
  • First degree AV-block
  • Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
  • Right bundle branch block
  • Subject has severe vision impairment that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements
  • Subject has a seizure disorder that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements.
  • Active malignancy or any other cancer from which the subject has been disease-free for< 2 years.
  • Subject has a solid organ transplant and/or is currently receiving treatment with therapy for immunosuppression, in the opinion of the Investigator.
  • Subject has been previously diagnosed with human immunodeficiency virus (HIV),hepatitis B, or hepatitis C infection.
  • Subject has a history of a systemic eosinophilic illness and/or an eosinophil count>1,000 cells x106/L at the Screening Visit.

  • Subject is currently participating or has participated in an interventional clinical trial (i.e.,investigational product or device, stem cell therapy, gene therapy) within 30 days of the Baseline Visit; or is currently enrolled in a non-interventional clinical trial (except for SPIMM-300) at the Baseline Visit which, in the opinion of the Investigator, may be potentially confounding with results of the current trial(e.g., exercise therapy trial).
  • Subject has previously received elamipretide (MTP-131), for any reason.
  • Subject has a history of active substance abuse during the year before the Baseline Visit, in the opinion of the Investigator.
  • Subject has 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 trial requirements.

PART 2:

Continuation Criteria:

  • Subjects must continue to be able and willing to adhere to the trial requirements.
  • Subject is appropriate to continue in Part 2 (i.e. subject was compliant in Part 1),in the opinion of the Investigator.
  • Subject has not had a serious adverse event (SAE)/serious adverse device effect (SADE)attributed to the elamipretide delivery system.
  • Subject has not permanently discontinued the elamipretide delivery system.

Locations

  • University of California San Diego
    La Jolla California 92093 United States
  • Stanford University
    Palo Alto California 94304 United States
  • Children's Hospital Colorado
    Aurora Colorado 80045 United States
  • Seattle Children's Hospital
    Seattle Washington 98105 United States
  • Baylor College of Medicine/Texas Children's Hospital
    Houston Texas 77030 United States
  • University of Texas Health Science Center
    Houston Texas 77030 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Stealth BioTherapeutics Inc.
ID
NCT03323749
Phase
Phase 3
Study Type
Interventional
Last Updated
July 25, 2018