Clinical Trial: Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Disease (PMD)

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: 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 Assessing long-term safety and tolerability through collection of adverse events



Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Changes in distance walked on Six Minute Walk Test [ Time Frame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 260 weeks ]
  • Changes in Triple Timed up and Go Test [ Time Frame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 260 weeks ]
  • Changes in 5X Sit to Stand [ Time Frame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 260 weeks ]
  • Changes in Scale for the Assessment and Rating of Ataxia [ Time Frame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 260 weeks ]
  • Changes in the NeuroQOL Fatigue Questionnaire [ Time Frame: up to 260 weeks ]
  • Changes in the Patient Global Assessment [ Time Frame: up to 260 Weeks ]
  • Changes in Primary Mitochondrial Disease Symptom Assessment [ Time Frame: Up to 260 weeks ]
  • Changes in Work Limitations Questionnaire [ Time Frame: Up to 260 Weeks ]
  • Changes in Physician Global Assessment [ Time Frame: Up to 260 Weeks ]


Original Secondary Outcome: Same as current

Information By: Stealth BioTherapeutics Inc.

Dates:
Date Received: November 18, 2016
Date Started: December 2016
Date Completion: December 2021
Last Updated: March 1, 2017
Last Verified: March 2017