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Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics
ClinicalTrials.gov Identifier:
NCT01396239
First received: July 8, 2011
Last updated: October 12, 2015
Last verified: July 2015
Results First Received: July 2, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: AVI-4658 (Eteplirsen)
Other: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
AVI-4658 (Eteplirsen) 50 mg/kg 50 mg/kg eteplirsen for 24 weeks
Placebo - Week 12 Biopsy Placebo for 24 Weeks with muscle Biopsy at Week 12
AVI-4658 (Eteplirsen) 30 mg/kg 30 mg/kg eteplirsen for 24 weeks
Placebo - Week 24 Biopsy Placebo for 24 weeks with muscle biopsy at Week 24

Participant Flow:   Overall Study
    AVI-4658 (Eteplirsen) 50 mg/kg   Placebo - Week 12 Biopsy   AVI-4658 (Eteplirsen) 30 mg/kg   Placebo - Week 24 Biopsy
STARTED   4   2   4   2 
COMPLETED   4   2   4   2 
NOT COMPLETED   0   0   0   0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
AVI-4658 (Eteplirsen) 30 mg/kg 30 mg/kg eteplirsen for 24 weeks
AVI-4658 (Eteplirsen) 50 mg/kg 50 mg/kg eteplirsen for 24 weeks
Placebo Placebo: phosphate buffered saline solution identical in appearance to eteplirsen for 24 weeks
Total Total of all reporting groups

Baseline Measures
   AVI-4658 (Eteplirsen) 30 mg/kg   AVI-4658 (Eteplirsen) 50 mg/kg   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 4   4   4   12 
Age 
[Units: Participants]
       
<=18 years   4   4   4   12 
Between 18 and 65 years   0   0   0   0 
>=65 years   0   0   0   0 
Age 
[Units: Years]
Mean (Standard Deviation)
 9.3  (0.50)   8.5  (1.29)   8.5  (1.73)   8.8  (1.22) 
Gender 
[Units: Participants]
       
Female   0   0   0   0 
Male   4   4   4   12 
Region of Enrollment 
[Units: Participants]
       
United States   4   4   4   12 


  Outcome Measures
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1.  Primary:   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. ]

2.  Secondary:   Change From Baseline: 6 Minute Walk Test (6MWT) - Intent to Treat Population (ITT)   [ Time Frame: 24 weeks ]

3.  Secondary:   Change From Baseline: 6 Minute Walk Test (6MWT) - Modified Intent to Treat Population (mITT)   [ Time Frame: 24 weeks ]

4.  Post-Hoc:   Adverse Events >30%   [ Time Frame: 24 Weeks ]

5.  Post-Hoc:   Frequency of AEs Related to Eteplirsen   [ Time Frame: 24 Weeks ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Due to the small number of study participants, a single adverse event (AE) in 1 patient exceeds the reporting threshold of 5%. Refer to the "Post-Hoc Outcome Measures" #4 and #5 for a summary of frequent and related AEs.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Edward M. Kaye MD, Interim CEO, SVP & Chief Medical Officer
Organization: Sarepta Therapeutics, Inc.
phone: 1-617-274-4003
e-mail: EKaye@Sarepta.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT01396239     History of Changes
Other Study ID Numbers: 4658-us-201
07-2484 ( Other Identifier: Office of Orphan Drug Development )
Study First Received: July 8, 2011
Results First Received: July 2, 2013
Last Updated: October 12, 2015
Health Authority: United States: Food and Drug Administration