Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
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ClinicalTrials.gov Identifier: NCT01396239 |
Recruitment Status :
Completed
First Posted : July 18, 2011
Results First Posted : November 9, 2015
Last Update Posted : March 30, 2020
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Sponsor:
Sarepta Therapeutics, Inc.
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.
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Tracking Information | ||||
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First Submitted Date ICMJE | July 8, 2011 | |||
First Posted Date ICMJE | July 18, 2011 | |||
Results First Submitted Date ICMJE | July 2, 2013 | |||
Results First Posted Date ICMJE | November 9, 2015 | |||
Last Update Posted Date | March 30, 2020 | |||
Study Start Date ICMJE | July 2011 | |||
Actual Primary Completion Date | February 2012 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
Change in the Number (%) of Dystrophin Positive Fibers [ Time Frame: After 12 weeks for 4 patients who received 50 mg/kg and 2 patients who received placebo. After 24 weeks for 4 patients who received 30 mg/kg and 2 patients who received placebo. ] The primary efficacy endpoint will be based on the pre-treatment and post-treatment change in the number (%) of dystrophin positive fibers as measured in the muscle biopsy tissue on immunohistochemistry (IHC).
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Original Primary Outcome Measures ICMJE |
Dystrophin positive fibers as measured in the muscle biopsy tissue on IHC [ Time Frame: 24 weeks ] The primary efficacy endpoint will be based on the pre-treatment and post-treatment percent of dystrophin positive fibers as measure in the muscle biopsy tissue on IHC.
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Change History | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
Key secondary: CD3, CD4, and CD8 lymphocyte counts as measured in the muscle biopsy tissue [ Time Frame: 24 weeks ] 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.
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Current Other Pre-specified Outcome Measures | Not Provided | |||
Original Other Pre-specified Outcome Measures | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients | |||
Official Title ICMJE | A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability and Pharmacokinetics Study of AVI-4658(Eteplirsen),in the Treatment of Ambulant Subjects With Duchenne Muscular Dystrophy | |||
Brief Summary | This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophy (DMD). | |||
Detailed Description | For details regarding the roll-over extension study 4658-us-202, please refer to NCT01540409. | |||
Study Type ICMJE | Interventional | |||
Study Phase ICMJE | Phase 2 | |||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Condition ICMJE | Duchenne Muscular Dystrophy | |||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Completed | |||
Actual Enrollment ICMJE |
12 | |||
Original Estimated Enrollment ICMJE | Same as current | |||
Actual Study Completion Date ICMJE | June 2012 | |||
Actual Primary Completion Date | February 2012 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Major Inclusion and Exclusion Criteria: Inclusion Criteria: A subject must meet all of the following criteria to be eligible for this study.
Exclusion Criteria: A subject who meets any of the following criteria will be excluded from this study.
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Sex/Gender ICMJE |
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Ages ICMJE | 7 Years to 13 Years (Child) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT01396239 | |||
Other Study ID Numbers ICMJE | 4658-us-201 07-2484 ( Other Identifier: Office of Orphan Drug Development ) |
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Has Data Monitoring Committee | No | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE | Not Provided | |||
Current Responsible Party | Sarepta Therapeutics, Inc. | |||
Original Responsible Party | Adrienne Ste. Marie, Sr. Project Manager, AVI BioPharma, Inc. | |||
Current Study Sponsor ICMJE | Sarepta Therapeutics, Inc. | |||
Original Study Sponsor ICMJE | Same as current | |||
Collaborators ICMJE | Not Provided | |||
Investigators ICMJE |
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PRS Account | Sarepta Therapeutics, Inc. | |||
Verification Date | March 2020 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |