ELEVIDYS Phase 4 DMD Post-Marketing Safety Study
Summary
NIH registered a Phase 4 clinical trial (NCT07542314) on ClinicalTrials.gov evaluating acute liver injury rates in Duchenne Muscular Dystrophy patients receiving ELEVIDYS gene therapy with sirolimus added as adjunct prophylactic immunosuppression. The study lists ELEVIDYS, sirolimus, glucocorticoids, and antibiotics as interventions.
What changed
NIH posted a new Phase 4 clinical trial registration on ClinicalTrials.gov. The study will evaluate acute liver injury rates in Duchenne Muscular Dystrophy participants treated with ELEVIDYS, with sirolimus added as a prophylactic immunosuppression adjunct. Glucocorticoids and antibiotics are also listed as interventions.
Sponsors and investigators conducting gene therapy trials for neuromuscular diseases may wish to monitor this registry for emerging post-marketing safety data on ELEVIDYS hepatotoxicity profiles.
Archived snapshot
Apr 21, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
Phase 4 NCT07542314 Kind: PHASE4 Apr 21, 2026
Abstract
The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
Conditions: Duchenne Muscular Dystrophy
Interventions: ELEVIDYS, Sirolimus, Glucocorticoids, Antibiotics
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