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Trial record 9 of 29 for:    skipping | "muscular dystrophy, duchenne and becker types"

Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00844597
Recruitment Status : Completed
First Posted : February 16, 2009
Results First Posted : October 6, 2015
Last Update Posted : October 6, 2015
Sponsor:
Collaborator:
British Medical Research Council
Information provided by (Responsible Party):
Sarepta Therapeutics

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Duchenne Muscular Dystrophy
Intervention Drug: AVI-4658 for Injection
Enrollment 19

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort 1 - 0.5 mg/kg/wk Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10.0 mg/kg/wk Cohort 6 - 20.0 mg/kg/wk
Hide Arm/Group Description Subjects in this group received a 0.5 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group will receive a 1.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group will receive a 2.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group will receive a 4.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group will receive a 10.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group will receive a 20.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period
Period Title: Overall Study
Started 4 2 2 3 4 4
Completed 4 2 2 2 4 4
Not Completed 0 0 0 1 0 0
Reason Not Completed
Adverse Event             0             0             0             1             0             0
Arm/Group Title Cohort 1 - 0.5mg/kg/wk Cohort 2 - 1.0mg/kg/wk Cohort 3 - 2.0mg/kg/wk Cohort 4 - 4.0mg/kg/wk Cohort 5 - 10.0mg/kg/wk Cohort 6 - 20.0mg/kg/wk Total
Hide Arm/Group Description Subjects in this group received a 0.5 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group received a 1.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group received a 2.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group received a 4.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group received a 10.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Subjects in this group received a 20.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period Total of all reporting groups
Overall Number of Baseline Participants 4 2 2 3 4 4 19
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 2 participants 2 participants 3 participants 4 participants 4 participants 19 participants
8.3  (0.50) 6.0  (0.0) 11.0  (2.83) 9.7  (0.58) 8.8  (2.75) 8.8  (1.26) 8.7  (1.91)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 2 participants 2 participants 3 participants 4 participants 4 participants 19 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
4
 100.0%
2
 100.0%
2
 100.0%
3
 100.0%
4
 100.0%
4
 100.0%
19
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 2 participants 2 participants 3 participants 4 participants 4 participants 19 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
4
 100.0%
2
 100.0%
2
 100.0%
3
 100.0%
4
 100.0%
4
 100.0%
19
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 2 participants 2 participants 3 participants 4 participants 4 participants 19 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
1
  50.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   5.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
4
 100.0%
1
  50.0%
2
 100.0%
3
 100.0%
4
 100.0%
4
 100.0%
18
  94.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Body Weight  
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 4 participants 2 participants 2 participants 3 participants 4 participants 4 participants 19 participants
33  (4.09) 23.7  (3.46) 42.6  (6.36) 40.1  (19.00) 34.6  (14.01) 33.0  (8.71) 34.5  (10.84)
Height  
Mean (Standard Deviation)
Unit of measure:  Centimeters
Number Analyzed 4 participants 2 participants 2 participants 3 participants 4 participants 4 participants 19 participants
127.3  (5.05) 110.7  (4.45) 126.9  (5.09) 126.9  (14.40) 123.7  (10.04) 126.5  (7.18) 124.5  (8.99)
1.Primary Outcome
Title Safety and Tolerability
Hide Description Number of subjects with 1 or more Treatment Emergent Adverse Event that are possibly related to the investigational drug
Time Frame Baseline to 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety Population - Any patient who received at least one dose of the study drug.
Arm/Group Title Open Label Treatment Arm
Hide Arm/Group Description:

AVI-4658 for Injection: AVI-4658 for Injection, is packaged as 100 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline as follows:

Cohort 1: 0.5mg/kg once weekly for 12 weeks; Cohort 2: 1.0mg/kg once weekly for 12 weeks; Cohort 3: 2.0mg/kg once weekly for 12 weeks; Cohort 4: 4.0mg/kg once weekly for 12 weeks; Cohort 5: 10.0mg/kg once weekly for 12 weeks; Cohort 6: 20.0mg/kg once weekly for 12 weeks

Overall Number of Participants Analyzed 19
Measure Type: Number
Unit of Measure: participants
14
2.Primary Outcome
Title Treatment Emergent Adverse Events
Hide Description Number of Patients with Treatment Emergent Adverse Events
Time Frame from Baseline to Follow up (27 weeks)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety Population
Arm/Group Title Open Label Treatment Arm
Hide Arm/Group Description:

Subjects will be sequentially allocated to one of 6 dose level cohorts and will receive 12 weekly IV infusions of AVI-4658 in 50 mL of normal saline solution over a 60-minute period

AVI-4658 for Injection: AVI-4658 for Injection, is packaged as 100 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline as follows:

Cohort 1: 0.5mg/kg once weekly for 12 weeks; Cohort 2: 1.0mg/kg once weekly for 12 weeks; Cohort 3: 2.0mg/kg once weekly for 12 weeks; Cohort 4: 4.0mg/kg once weekly for 12 weeks; Cohort 5: 10.0mg/kg once weekly for 12 weeks; Cohort 6: 20.0mg/kg once weekly for 12 weeks

Overall Number of Participants Analyzed 19
Measure Type: Number
Unit of Measure: participants
19
3.Secondary Outcome
Title Pharmacokinetics - Mean Peak Plasma Concentration of AVI-4658 After Administration
Hide Description Standard Pharmacokinetic parameters estimated using non-compartmental modeling of plasma concentration data.
Time Frame Samples were taken: 30 minutes pre dose; and at 5 (±1), 15 (±2), 30 (±5), 60 (±5), and 90 (±5) minutes; and 2, 4, 6, 8, 12, and 24 hours (all ± 15 minutes) post dose at Weeks 1, 6, and 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PK Evaluable Population: Included all patients who provided at least 1 PK sample. The reportable PK population included those patients with at least Cmax, Tmax, and AUC0-24 computed from 1 or more of the 3 sampling days (1st, 6th, 12th dose [Weeks 1, 6, and 12]).
Arm/Group Title Open Label Treatment Arm
Hide Arm/Group Description:

AVI-4658 for Injection: AVI-4658 for Injection, is packaged as 100 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline as follows:

Cohort 1: 0.5mg/kg once weekly for 12 weeks; Cohort 2: 1.0mg/kg once weekly for 12 weeks; Cohort 3: 2.0mg/kg once weekly for 12 weeks; Cohort 4: 4.0mg/kg once weekly for 12 weeks; Cohort 5: 10.0mg/kg once weekly for 12 weeks; Cohort 6: 20.0mg/kg once weekly for 12 weeks

Overall Number of Participants Analyzed 19
Mean (Standard Deviation)
Unit of Measure: ng/mL
39000  (16900)
4.Secondary Outcome
Title Efficacy of Eteplirsen Over 12 Weeks of Dosing
Hide Description Efficacy was defined as an estimated change in the percentage of dystrophin positive fibers (assessed by IHC) at Week 14 from Baseline after 12 weekly doses of eterplirsen. This outcome measure represents the number of patients to show an increase in the percentage of dystrophin-positive fibers.
Time Frame Biopsies were taken at Baseline and Week 14
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Per Protocol Population - Included all patients who received all 12 doses of study treatment.
Arm/Group Title Open Label Treatment Arm
Hide Arm/Group Description:

Subjects will be sequentially allocated to one of 6 dose level cohorts and will receive 12 weekly IV infusions of AVI-4658 in 50 mL of normal saline solution over a 60-minute period

AVI-4658 for Injection: AVI-4658 for Injection, is packaged as 100 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline as follows:

Cohort 1: 0.5mg/kg once weekly for 12 weeks; Cohort 2: 1.0mg/kg once weekly for 12 weeks; Cohort 3: 2.0mg/kg once weekly for 12 weeks; Cohort 4: 4.0mg/kg once weekly for 12 weeks; Cohort 5: 10.0mg/kg once weekly for 12 weeks; Cohort 6: 20.0mg/kg once weekly for 12 weeks

Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: participants
11
5.Post-Hoc Outcome
Title Adverse Events >15%
Hide Description Adverse events that occurred in >15% of overall patient population across dose level arms.
Time Frame 27 Weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety Population
Arm/Group Title Cohort 1 - 0.5 mg/kg/wk Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10.0 mg/kg/wk Cohort 6 - 20.0 mg/kg/wk
Hide Arm/Group Description:
Subjects in this group received a 0.5 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period
Subjects in this group received a 1.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period
Subjects in this group received a 2.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period
Subjects in this group received a 4.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period
Subjects in this group received a 10.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period
Subjects in this group received a 20.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period
Overall Number of Participants Analyzed 4 2 2 3 4 4
Measure Type: Number
Unit of Measure: Events
Cardiomyopathy 0 0 0 1 0 2
Tachycardia 0 0 0 1 0 2
Abdominal Pain 0 1 0 1 1 0
Nausea 0 0 1 1 0 1
Vomiting 0 0 1 1 1 0
Influenza like illness 0 0 2 0 1 0
Rhinitis 1 0 0 1 4 1
Upper respiratory tract infection 2 1 0 1 2 2
Fall 2 0 0 2 0 1
Arthralgia 1 0 1 1 0 0
Back Pain 1 0 1 2 2 1
Myalgia 1 1 1 0 1 0
Dizziness 0 0 1 1 0 1
Headache 2 1 2 0 2 1
Time Frame Adverse Event data for this study was collected from Baseline to Follow-up (27 Weeks)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1 - 0.5 mg/kg/wk Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10.0 mg/kg/wk Cohort 6 - 20.0 mg/kg/wk
Hide Arm/Group Description Subjects in this group received a 0.5 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period Subjects in this group received a 1.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period Subjects in this group received a 2.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period Subjects in this group received a 4.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period Subjects in this group received a 10.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period Subjects in this group received a 20.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60 minute period
All-Cause Mortality
Cohort 1 - 0.5 mg/kg/wk Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10.0 mg/kg/wk Cohort 6 - 20.0 mg/kg/wk
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Cohort 1 - 0.5 mg/kg/wk Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10.0 mg/kg/wk Cohort 6 - 20.0 mg/kg/wk
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/4 (0.00%)      0/2 (0.00%)      1/2 (50.00%)      1/3 (33.33%)      0/4 (0.00%)      0/4 (0.00%)    
Gastrointestinal disorders             
Post-Operative Nausea and Vomiting *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Musculoskeletal and connective tissue disorders             
Fracture of Left Medial Malleolus *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cohort 1 - 0.5 mg/kg/wk Cohort 2 - 1.0 mg/kg/wk Cohort 3 - 2.0 mg/kg/wk Cohort 4 - 4.0 mg/kg/wk Cohort 5 - 10.0 mg/kg/wk Cohort 6 - 20.0 mg/kg/wk
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/4 (100.00%)      2/2 (100.00%)      2/2 (100.00%)      3/3 (100.00%)      4/4 (100.00%)      4/4 (100.00%)    
Blood and lymphatic system disorders             
Platelet anisocytosis *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Cardiac disorders             
Tachycardia *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 2/4 (50.00%)  2
Cardiomyopathy *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Sinus tachycardia *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Ear and labyrinth disorders             
Ear Pain *  1/4 (25.00%)  1 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Gastrointestinal disorders             
Nausea *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 1/3 (33.33%)  1 0/4 (0.00%)  0 1/4 (25.00%)  1
Vomiting *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 1/3 (33.33%)  1 1/4 (25.00%)  1 0/4 (0.00%)  0
Abdominal Pain *  0/4 (0.00%)  0 1/2 (50.00%)  1 0/2 (0.00%)  0 1/3 (33.33%)  1 1/4 (25.00%)  1 0/4 (0.00%)  0
Abdominal pain - Upper *  1/4 (25.00%)  1 0/2 (0.00%)  0 1/2 (50.00%)  1 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Constipation *  1/4 (25.00%)  1 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Diarrhoea *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Enteritis *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Lip dry *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
General disorders             
Influenza like illness *  0/4 (0.00%)  0 0/2 (0.00%)  0 2/2 (100.00%)  2 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Abasia *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Application site rash *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Catheter Site Pain *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Disease progression * [1]  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 1/4 (25.00%)  1
Fatigue *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Infusion related reaction *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Pyrexia *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Vaccination site pain *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Vessel puncture site haematoma *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Infections and infestations             
Rhinitis *  1/4 (25.00%)  1 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 4/4 (100.00%)  4 1/4 (25.00%)  1
Upper respiratory tract infection *  2/4 (50.00%)  2 1/2 (50.00%)  1 0/2 (0.00%)  0 1/3 (33.33%)  1 2/4 (50.00%)  2 2/4 (50.00%)  2
Bronchitis *  0/4 (0.00%)  0 1/2 (50.00%)  1 0/2 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Hordeolum *  0/4 (0.00%)  0 1/2 (50.00%)  1 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Incision site infection *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Nasopharyngitis *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Respiratory tract infection viral *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Tinea infection *  0/4 (0.00%)  0 1/2 (50.00%)  1 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Viral Infection *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Injury, poisoning and procedural complications             
Fall *  2/4 (50.00%)  2 0/2 (0.00%)  0 0/2 (0.00%)  0 2/3 (66.67%)  2 0/4 (0.00%)  0 1/4 (25.00%)  1
Ankle Fracture *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Arthropod bite *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Arthropod sting *  1/4 (25.00%)  1 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Contusion *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Excoriation *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Head Injury *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Heat Stroke *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Lumbar vertebral fracture *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 1/4 (25.00%)  1
Post procedural haematoma *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Procedural Pain *  2/4 (50.00%)  2 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Vaccination complication *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Metabolism and nutrition disorders             
Decreased appetitie *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Musculoskeletal and connective tissue disorders             
Back pain *  1/4 (25.00%)  1 0/2 (0.00%)  0 1/2 (50.00%)  1 2/3 (66.67%)  2 2/4 (50.00%)  2 1/4 (25.00%)  1
Myalgia *  1/4 (25.00%)  1 1/2 (50.00%)  1 1/2 (50.00%)  1 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Arthralgia *  1/4 (25.00%)  1 0/2 (0.00%)  0 1/2 (50.00%)  1 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Coccydynia *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Musculoskeletal pain *  1/4 (25.00%)  1 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Osteopenia *  1/4 (25.00%)  1 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Pain in extremity *  1/4 (25.00%)  1 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Nervous system disorders             
Headache *  2/4 (50.00%)  2 1/2 (50.00%)  1 2/2 (100.00%)  2 0/3 (0.00%)  0 2/4 (50.00%)  2 1/4 (25.00%)  1
Dizziness *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 1/3 (33.33%)  1 0/4 (0.00%)  0 1/4 (25.00%)  1
Renal and urinary disorders             
Enuresis *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Respiratory, thoracic and mediastinal disorders             
Asthma *  0/4 (0.00%)  0 1/2 (50.00%)  1 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Cough *  2/4 (50.00%)  2 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Epistaxis *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Productive cough *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Skin and subcutaneous tissue disorders             
Drug eruption *  0/4 (0.00%)  0 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Skin irritation *  1/4 (25.00%)  1 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
Vascular disorders             
Haematoma *  1/4 (25.00%)  1 0/2 (0.00%)  0 0/2 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Pallor *  0/4 (0.00%)  0 0/2 (0.00%)  0 1/2 (50.00%)  1 0/3 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
[1]
One subject had a reduction in fractional shortening due DMD progression One subject had a deterioration of clinical signs of DMD
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" #5 for a summary of frequent and related AEs.
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 PI required written permission to publish any information relating to the trial and if given permission to acknowledge the sponsor's contribution and ownership of the materials.
Results Point of Contact
Name/Title: Edward M. Kaye, MD, Interim CEO, SVP & Chief Medical Officer
Organization: Sarepta Therapeutics
Phone: 617-274-4003
Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT00844597     History of Changes
Other Study ID Numbers: AVI-4658-28
First Submitted: December 24, 2008
First Posted: February 16, 2009
Results First Submitted: June 8, 2015
Results First Posted: October 6, 2015
Last Update Posted: October 6, 2015