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