A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

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NCT03992430 · readout in 73 d

Sponsored by Sarepta Therapeutics, Inc. (industry) · SRPT — their whole pipeline →.

Phase

Phase 3

Status

Active, not recruiting

Study type

Interventional

Design

Randomized · Quadruple · Treatment

Enrollment

160actual

Sites

Countries

Colombia, Czechia, Denmark +23

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Dates

each axis at the precision it was filed, with the registry's own basis

Date

Filed

Basis

What it is

Start

2020-07-13

actual

When the study began enrolling

Primary completion

2026-10-31

estimated

The date the last participant is measured for the primary outcome — the readout window

Study completion

2026-10-31

estimated

The whole study's end, after follow-up

First posted

2019-06-20

actual

When this record first appeared on the registry

Results posted

—

When the sponsor posted results to the registry

Record updated

2026-02-27

actual

The sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

Muscular Dystrophy, Duchenne

Intervention

Drug: Eteplirsen — also filed as AVI-4658, EXONDYS 51, EXONDYS

Primary outcomes

what the primary-completion date above is the date OF

Part 1: Incidence of Adverse Events (AEs)

measured Up to Week 148

Part 2: Change From Baseline at Week 144 in the NSAA Total Score (for Final Analysis)

measured Baseline, Week 144

Part 2: Change from Baseline at Week 72 or Week 96 in NSAA Total Score (for Conditional Efficacy Interim Analysis)

measured Baseline, Week 72 or Week 96

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