©2024 Stanford Medicine
Study of Eteplirsen in DMD Patients
Recruiting
I'm InterestedTrial ID: NCT02255552,25513,19502,26554
Purpose
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658)
in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51.
Additional objectives include evaluation of safety, biomarkers and the long-term effects of
eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Official Title
An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
Eligibility
Inclusion Criteria:
- Male 7-16 years old
- Diagnosed with DMD, genotypically confirmed
- Stable dose of corticosteroids for at least 24 weeks
- Have intact right and left alternative upper muscle groups
- Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)
- Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and
LVEF of greater than 50%
Exclusion Criteria:
- Previous treatment with drisapersen or any other RNA antisense agent or any gene
therapy within the last 6 months
- Participation in any other DMD interventional clinical study within 12 weeks
- Major surgery within 3 months
- Presence of other clinically significant illness
- Major change in the physical therapy regime within 3 months
Other inclusion/exclusion criteria apply.
Intervention(s):
drug: eteplirsen
Recruiting
I'm InterestedContact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
John W. Day, MD, PhD