SS-31 research: studies, doses and safety

• First 12 weeks, Barth syndrome trial: In the small Barth syndrome trial, injection-site reactions were common and daily elamipretide did not beat placebo on walking or fatigue at 12 weeks.
• Extension weeks 12-24: Knee strength rose during the uncontrolled Barth syndrome extension, which cannot separate drug effects from other changes.
• Extension week 168: Eight remaining Barth syndrome participants still showed knee-strength gains, but there was no placebo group for comparison.

Forzinity is used daily for eligible people with Barth syndrome; its label gives no fixed cycle.

45 human records include ongoing trials: 28 registered treatment studies, 9 other FDA studies, 7 case reports or series and 1 historical comparison. Records can share participants.

Key studies

• TAZPOWER: Barth crossover: The randomized phase failed; later uncontrolled muscle-strength gains supported accelerated approval. (source)
• TAZPOWER: long extension: Walking gains persisted among eight long-term completers. (source)
• MMPOWER: IV dose finding: The unadjusted main comparison narrowly missed significance. (source)
• MMPOWER-2: daily injections: The walking endpoint missed; several fatigue measures improved. (source)
• MMPOWER-3: phase 3: Neither walking distance nor fatigue improved versus placebo. (source)
• MMPOWER-3: genetic subgroups: Post hoc genetic signals led to NuPOWER. (source)