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High-dose eteplirsen dose-escalation and randomized dose-comparison study in exon 51-skippable Duchenne muscular dystrophy
NL-OMON52516Phase 3Recruiting
- Sponsor
- Sarepta Therapeutics
- Start
- Jan 2023
- 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
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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 and Change from baseline in NSAA total score. Started Jan 2023; status: recruiting.
02
Drugs
eteplirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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