Modifying muscle metabolic dysregulation in inclusion body myositis with pioglitazone: a single-arm trial - PubMed
5 hours ago
- #inclusion body myositis
- #pioglitazone
- #metabolic dysregulation
- Single-arm, open-label phase 1 trial evaluated pioglitazone in inclusion body myositis (IBM) patients.
- Primary outcome: Change in PPARGC1A expression and related metabolic pathways after 16 weeks of treatment.
- Pioglitazone reversed metabolic abnormalities, enhancing oxidative phosphorylation in muscle.
- Clinical outcomes unchanged overall, but a subset showed slower decline in IBM-FRS and m-TUG.
- Adverse effects included myalgia and heart failure exacerbation.
- Trial terminated early due to COVID-19 pandemic.
- Supported by Ira T. Discovery Fund and Peter and Carmen Lucia Buck Foundation Myositis Discovery Fund.