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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: July 31, 2012
Last verified: July 2012
History of Changes
  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:

U.S. FDA Resources

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: December 2013
Estimated Primary Completion Date: September 2013 (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: July 31, 2012
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 May 19, 2013