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Phase 3 dose-finding study of high-dose eteplirsen in ambulant exon 51-skipping Duchenne muscular dystrophy
NL-OMON58904Phase 3Completed
- Sponsor
- Sarepta Therapeutics
- Start
- Nov 2022
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of eteplirsen in Duchenne muscular dystrophy (DMD) (5 arms), sponsored by Sarepta Therapeutics. Primary endpoints: Incidence of adverse events, Incidence of adverse events of special interest and 7 more. Started Nov 2022; status: completed.
02
Drugs
eteplirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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