Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics
ClinicalTrials.gov Identifier:
NCT01540409
First received: February 23, 2012
Last updated: October 17, 2013
Last verified: July 2012
  Purpose

The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 80 weeks of treatment with eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28-week eteplirsen study: Study 4658-US-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.


Condition Intervention Phase
Duchenne Muscular Dystrophy
Drug: AVI-4658 (Eteplirsen)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201

Resource links provided by NLM:


Further study details as provided by Sarepta Therapeutics:

Primary Outcome Measures:
  • The primary efficacy endpoint will be the change from baseline in the percent of dystrophin positive fibers in muscle biopsy tissue as measured by immunohistochemistry (IHC). [ Time Frame: 20 Weeks ] [ Designated as safety issue: Yes ]
    The primary efficacy endpoint will be based on the pre-treatment and post-treament percent of dystrophin positive fibers as measured in the muscle biopsy tissue on(IHC).


Secondary Outcome Measures:
  • The secondary efficacy endpoints will be the change from baseline in: CD3, CD4, and CD8 lymphocyte counts in muscle biopsy tissue; 6-Minute Walk Test (6-MWT) distance. [ Time Frame: 20,80 Weeks ] [ Designated as safety issue: Yes ]
    A key secondary efficacy endpoint will be based on the pre-treatment and post-treatment CD3, CD4, and CD8 lymphocyte counts as measured in the muscle biopsy tissue as well as a 6-Minute Walk Test(6-MWT)distance.


Enrollment: 12
Study Start Date: February 2012
Estimated Study Completion Date: July 2015
Estimated Primary Completion Date: April 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AVI-4658 (Eteplirsen)
Multiple-Dose Extension Study
Drug: AVI-4658 (Eteplirsen)
Eteplirsen will be administered once weekly via an IV infusion over a period of at least 60 minutes. Their are two treatment groups, 30 mg/kg and 50 mg/kg.

  Eligibility

Ages Eligible for Study:   7 Years to 13 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

A subject must meet all of the following criteria to be eligible for this study.

  1. The subject and/or their parent/legal guardian are willing and able to provide signed informed consent.
  2. The subject has successfully completed 28 weeks of treatment in Study 4658-US-201.
  3. The subject has a parent(s) or legal guardian(s) who is able to understand and comply with all of the study procedure requirements.

Exclusion Criteria:

A subject who meets any of the following criteria will be excluded from this study.

1. The subject has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the subject or make it unlikely that the course of treatment or follow-up would be completed or impair the assessment of study results.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01540409

Locations
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Sarepta Therapeutics
Investigators
Principal Investigator: Jerry R Mendell, MD Nationwide Childtren's Hospital
  More Information

No publications provided

Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT01540409     History of Changes
Other Study ID Numbers: 4658-us-202
Study First Received: February 23, 2012
Last Updated: October 17, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Sarepta Therapeutics:
DMD

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on April 20, 2014