Loading clinical trials...
Loading clinical trials...
An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Safety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients. Clinical efficacy, including functional tests and MRI, will be assessed at regularly scheduled study visits. Patients will undergo one baseline and one follow-up muscle biopsy. Population and serial PK will be collected.
Age
4 - 6 years
Sex
MALE
Healthy Volunteers
No
Neuromuscular Research Center of Arizona
Phoenix, Arizona, United States
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States
University of California, Davis Medical Center
Sacramento, California, United States
Stanford University Medical Center
Stanford, California, United States
University of Florida, Shands Hospital
Gainesville, Florida, United States
Rare Disease Research Center
Atlanta, Georgia, United States
Children's Hospital of Atlanta
Atlanta, Georgia, United States
University of Iowa Children's Hospital
Iowa City, Iowa, United States
St. Louis Children's Hospital
St Louis, Missouri, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Start Date
June 30, 2015
Primary Completion Date
December 17, 2018
Completion Date
December 17, 2018
Last Updated
January 25, 2021
33
ACTUAL participants
eteplirsen
DRUG
Lead Sponsor
Sarepta Therapeutics, Inc.
NCT07037862
NCT07129954
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.
View ClinicalTrials.gov Terms and ConditionsNCT06925269