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Trial record 42 of 997 for:    BMD

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

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: NCT01540409
Recruitment Status : Completed
First Posted : February 28, 2012
Results First Posted : July 10, 2019
Last Update Posted : July 29, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Duchenne Muscular Dystrophy (DMD)
Intervention Drug: AVI-4658 (Eteplirsen)
Enrollment 12
Recruitment Details The study was conducted at 12 centers in the United States. Overall, 12 participants who completed parent study 4658-us-201 (NCT01396239) were enrolled between July 2011 and February 2012 in this extension study (4658-us-202; NCT01540409). A 4-week open label period (Week 24-28) was observed between the parent and extension study.
Pre-assignment Details Participants who received placebo in 4658-us-201 study were randomized in 1:1 ratio in this extension study to receive either eteplirsen 30 or 50 milligram per kilogram (mg/kg) and, those who received eteplirsen 30 or 50 mg/kg in 4658-us-201 received same treatment in this extension study.
Arm/Group Title Eteplirsen 30 mg/kg Eteplirsen 50 mg/kg
Hide Arm/Group Description Participants who received 30 milligram per kilogram (mg/kg) eteplirsen or placebo once weekly, intravenous (IV) infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 30 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study. Participants who received 50 mg/kg eteplirsen or placebo once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 50 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study.
Period Title: Overall Study
Started 6 6
Completed 6 6
Not Completed 0 0
Arm/Group Title Eteplirsen 30 mg/kg Eteplirsen 50 mg/kg Total
Hide Arm/Group Description Participants who received 30 mg/kg eteplirsen or placebo once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 30 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study. Participants who received 50 mg/kg eteplirsen or placebo once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 50 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study. Total of all reporting groups
Overall Number of Baseline Participants 6 6 12
Hide Baseline Analysis Population Description
The safety population included all randomized participants who received any amount of study drug.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 6 participants 12 participants
9.2  (0.75) 8.8  (1.47) 9.0  (1.13)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 12 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
6
 100.0%
6
 100.0%
12
 100.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 6 participants 6 participants 12 participants
6
 100.0%
6
 100.0%
12
 100.0%
1.Primary Outcome
Title Change From Baseline in the 6 Minute Walk Test (6MWT) at Week 240
Hide Description This study used a modified version of the 6MWT test procedure described in American Thoracic Society (ATS) 2002 guidelines, specifically adapted for patients with Duchenne muscular dystrophy. The participant was asked to walk a set course of 25 meters for 6 minutes (timed) and the distance walked in meters was recorded. Increases from baseline in 6MWT distance are indicative of improvement and decreases from baseline indicate worsening. Baseline here corresponds to the baseline in the parent study (4658-us-201, NCT01396239).
Time Frame Parent Baseline and Week 240
Hide Outcome Measure Data
Hide Analysis Population Description
The Intent-to-Treat Population (ITT) population included all participants randomized into parent study 4658-us-201. Here, “Overall Number of Participants Analyzed” signifies participants evaluable for this outcome measure. Results are reported below in 2 reporting groups based on evaluation period (Week 240) as applicable for this outcome measure.
Arm/Group Title Eteplirsen 30 mg/kg Eteplirsen 50 mg/kg
Hide Arm/Group Description:
Participants who received 30 mg/kg eteplirsen or placebo once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 30 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study.
Participants who received 50 mg/kg eteplirsen or placebo once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 50 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study.
Overall Number of Participants Analyzed 4 3
Mean (Standard Deviation)
Unit of Measure: Meters
-199.0  (113.25) -258.0  (175.65)
2.Primary Outcome
Title Change From Baseline in the Percentage of Dystrophin Positive Fibers (PDPF) at Week 48
Hide Description Dystrophin expression as assessed by percent dystrophin positive fibers was measured by immunohistochemistry (IHC) technique using primary anti-dystrophin antibody. Percent change from baseline is the arithmetic difference of the treatment time point minus baseline divided by baseline calculated for individual subjects. Baseline here corresponds to the baseline in the parent study (4658-us-201, NCT01396239).
Time Frame Parent Baseline and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included all participants randomized into parent study 4658-us-201. Results are reported below in 3 reporting groups of placebo to eteplirsen, eteplirsen 30 mg/kg, and eteplirsen 50 mg/kg, respectively, based on evaluation period (Week 48) as applicable for this outcome measure.
Arm/Group Title Placebo to Eteplirsen Eteplirsen 30 mg/kg Eteplirsen 50 mg/kg
Hide Arm/Group Description:
Participants who received eteplirsen matched placebo once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), followed by 30 or 50 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study.
Participants who received 30 mg/kg eteplirsen once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 30 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study.
Participants who received 50 mg/kg eteplirsen once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 50 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study.
Overall Number of Participants Analyzed 4 4 4
Mean (Standard Deviation)
Unit of Measure: Percentage of Dystrophin Positive Fibers
37.70  (12.602) 51.69  (7.089) 42.93  (13.433)
Time Frame Parent Baseline up to 240 weeks (cumulative Study 4658-us-201 + 4658-us-202)
Adverse Event Reporting Description Only treatment-emergent adverse events with an onset date on or after the date of first dose of study drug were reported. Relatedness of Serious Adverse Events (SAEs) to study medication was determined by the Investigator.
 
Arm/Group Title Eteplirsen 30 mg/kg Eteplirsen 50 mg/kg
Hide Arm/Group Description Participants who received 30 mg/kg eteplirsen or placebo once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 30 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study. Participants who received 50 mg/kg eteplirsen or placebo once weekly, IV infusion for 24 weeks in the parent study 4658-us-201 (NCT01396239), continued the same treatment with 50 mg/kg eteplirsen once weekly for 212 weeks (up to Week 240) in this extension study.
All-Cause Mortality
Eteplirsen 30 mg/kg Eteplirsen 50 mg/kg
Affected / at Risk (%) Affected / at Risk (%)
Total   0/6 (0.00%)   0/6 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Eteplirsen 30 mg/kg Eteplirsen 50 mg/kg
Affected / at Risk (%) Affected / at Risk (%)
Total   4/6 (66.67%)   2/6 (33.33%) 
Injury, poisoning and procedural complications     
Tibia Fracture * 1 [1]  0/6 (0.00%)  1/6 (16.67%) 
Femur Fracture * 1 [1]  3/6 (50.00%)  1/6 (16.67%) 
Musculoskeletal and connective tissue disorders     
Scoliosis * 1 [2]  2/6 (33.33%)  0/6 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Acute respiratory failure * 1 [2]  1/6 (16.67%)  0/6 (0.00%) 
1
Term from vocabulary, MedDRA (14.0)
*
Indicates events were collected by non-systematic assessment
[1]
Not related to study medication. All events are related to patients falling.
[2]
Not related to study medication.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Eteplirsen 30 mg/kg Eteplirsen 50 mg/kg
Affected / at Risk (%) Affected / at Risk (%)
Total   6/6 (100.00%)   6/6 (100.00%) 
Blood and lymphatic system disorders     
Anaemia * 1  2/6 (33.33%)  0/6 (0.00%) 
Cardiac disorders     
Tachycardia * 1  1/6 (16.67%)  0/6 (0.00%) 
Congenital, familial and genetic disorders     
Cryptorchism * 1  1/6 (16.67%)  0/6 (0.00%) 
Ear and labyrinth disorders     
Cerumen impaction * 1  1/6 (16.67%)  0/6 (0.00%) 
Motion sickness * 1  0/6 (0.00%)  1/6 (16.67%) 
Tympanic membrane disorder * 1  0/6 (0.00%)  1/6 (16.67%) 
Endocrine disorders     
Goitre * 1  0/6 (0.00%)  1/6 (16.67%) 
Growth hormone deficiency * 1  0/6 (0.00%)  1/6 (16.67%) 
Cushingoid * 1  0/6 (0.00%)  2/6 (33.33%) 
Eye disorders     
Cataract * 1  0/6 (0.00%)  2/6 (33.33%) 
Cataract subcapsular * 1  1/6 (16.67%)  0/6 (0.00%) 
Conjunctivitis * 1  1/6 (16.67%)  0/6 (0.00%) 
Erythema of eyelid * 1  0/6 (0.00%)  1/6 (16.67%) 
Hypermetropia * 1  1/6 (16.67%)  0/6 (0.00%) 
Gastrointestinal disorders     
Vomiting * 1  5/6 (83.33%)  4/6 (66.67%) 
Abdominal pain upper * 1  1/6 (16.67%)  3/6 (50.00%) 
Dyspepsia * 1  3/6 (50.00%)  1/6 (16.67%) 
Abdominal pain * 1  0/6 (0.00%)  2/6 (33.33%) 
Constipation * 1  2/6 (33.33%)  2/6 (33.33%) 
Diarrhoea * 1  1/6 (16.67%)  2/6 (33.33%) 
Nausea * 1  2/6 (33.33%)  3/6 (50.00%) 
Oral pain * 1  1/6 (16.67%)  2/6 (33.33%) 
Abdominal discomfort * 1  1/6 (16.67%)  1/6 (16.67%) 
Dental caries * 1  1/6 (16.67%)  0/6 (0.00%) 
Dysphagia * 1  1/6 (16.67%)  0/6 (0.00%) 
Flatulence * 1  1/6 (16.67%)  0/6 (0.00%) 
Food poisoning * 1  0/6 (0.00%)  1/6 (16.67%) 
Haemorrhoids * 1  0/6 (0.00%)  1/6 (16.67%) 
Lip swelling * 1  0/6 (0.00%)  1/6 (16.67%) 
Retained deciduous tooth * 1  1/6 (16.67%)  0/6 (0.00%) 
Tooth impacted * 1  0/6 (0.00%)  1/6 (16.67%) 
Toothache * 1  1/6 (16.67%)  0/6 (0.00%) 
Gastritis * 1  1/6 (16.67%)  0/6 (0.00%) 
Tooth disorder * 1  1/6 (16.67%)  0/6 (0.00%) 
General disorders     
Pyrexia * 1  1/6 (16.67%)  2/6 (33.33%) 
Catheter site pain * 1  2/6 (33.33%)  2/6 (33.33%) 
Infusion site extravasation * 1  2/6 (33.33%)  2/6 (33.33%) 
Device occlusion * 1  1/6 (16.67%)  1/6 (16.67%) 
Infusion site haematoma * 1  1/6 (16.67%)  1/6 (16.67%) 
Oedema peripheral * 1  2/6 (33.33%)  0/6 (0.00%) 
Thrombosis in device * 1  2/6 (33.33%)  1/6 (16.67%) 
Application site erythema * 1  0/6 (0.00%)  1/6 (16.67%) 
Application site pruritus * 1  0/6 (0.00%)  1/6 (16.67%) 
Catheter site haematoma * 1  0/6 (0.00%)  2/6 (33.33%) 
Catheter site haemorrhage * 1  0/6 (0.00%)  1/6 (16.67%) 
Catheter site inflammation * 1  0/6 (0.00%)  1/6 (16.67%) 
Catheter site related reaction * 1  0/6 (0.00%)  1/6 (16.67%) 
Chest pain * 1  1/6 (16.67%)  0/6 (0.00%) 
Influenza like illness * 1  1/6 (16.67%)  0/6 (0.00%) 
Infusion site pain * 1  0/6 (0.00%)  1/6 (16.67%) 
Infusion site rash * 1  1/6 (16.67%)  0/6 (0.00%) 
Injection site pain * 1  0/6 (0.00%)  1/6 (16.67%) 
Irritability * 1  1/6 (16.67%)  0/6 (0.00%) 
Malaise * 1  0/6 (0.00%)  1/6 (16.67%) 
Non-cardiac chest pain * 1  1/6 (16.67%)  0/6 (0.00%) 
Pain * 1  1/6 (16.67%)  2/6 (33.33%) 
Swelling * 1  0/6 (0.00%)  1/6 (16.67%) 
Disease progression * 1  0/6 (0.00%)  1/6 (16.67%) 
Infusion site urticaria * 1  1/6 (16.67%)  0/6 (0.00%) 
Immune system disorders     
Hypersensitivity * 1  0/6 (0.00%)  1/6 (16.67%) 
Seasonal allergy * 1  0/6 (0.00%)  1/6 (16.67%) 
Infections and infestations     
Nasopharyngitis * 1  4/6 (66.67%)  5/6 (83.33%) 
Upper respiratory tract infection * 1  2/6 (33.33%)  4/6 (66.67%) 
Gastroenteritis viral * 1  1/6 (16.67%)  2/6 (33.33%) 
Hordeolum * 1  0/6 (0.00%)  3/6 (50.00%) 
Influenza * 1  0/6 (0.00%)  3/6 (50.00%) 
Post procedural cellulitis * 1  1/6 (16.67%)  1/6 (16.67%) 
Rhinitis * 1  0/6 (0.00%)  1/6 (16.67%) 
Viral infection * 1  1/6 (16.67%)  2/6 (33.33%) 
Candidiasis * 1  1/6 (16.67%)  0/6 (0.00%) 
Folliculitis * 1  0/6 (0.00%)  1/6 (16.67%) 
Gastroenteritis * 1  1/6 (16.67%)  0/6 (0.00%) 
Incision site infection * 1  0/6 (0.00%)  1/6 (16.67%) 
Pharyngitis streptococcal * 1  1/6 (16.67%)  0/6 (0.00%) 
Pneumonia * 1  1/6 (16.67%)  0/6 (0.00%) 
Sinusitis * 1  1/6 (16.67%)  0/6 (0.00%) 
Tinea capitis * 1  1/6 (16.67%)  0/6 (0.00%) 
Tinea pedis * 1  1/6 (16.67%)  0/6 (0.00%) 
Viral upper respiratory tract infection * 1  1/6 (16.67%)  0/6 (0.00%) 
Abscess * 1  0/6 (0.00%)  1/6 (16.67%) 
Abscess limb * 1  0/6 (0.00%)  1/6 (16.67%) 
Bacterial disease carrier * 1  1/6 (16.67%)  0/6 (0.00%) 
Localised infection * 1  0/6 (0.00%)  1/6 (16.67%) 
Paraspinal abscess * 1  0/6 (0.00%)  1/6 (16.67%) 
Pharyngitis * 1  1/6 (16.67%)  0/6 (0.00%) 
Injury, poisoning and procedural complications     
Procedural pain * 1  5/6 (83.33%)  6/6 (100.00%) 
Contusion * 1  4/6 (66.67%)  4/6 (66.67%) 
Arthropod bite * 1  3/6 (50.00%)  2/6 (33.33%) 
Excoriation * 1  2/6 (33.33%)  3/6 (50.00%) 
Joint injury * 1  2/6 (33.33%)  1/6 (16.67%) 
Joint sprain * 1  0/6 (0.00%)  3/6 (50.00%) 
Muscle strain * 1  2/6 (33.33%)  2/6 (33.33%) 
Fall * 1  1/6 (16.67%)  0/6 (0.00%) 
Foot fracture * 1  1/6 (16.67%)  1/6 (16.67%) 
Incision site haemorrhage * 1  1/6 (16.67%)  1/6 (16.67%) 
Incision site pain * 1  1/6 (16.67%)  0/6 (0.00%) 
Arthropod sting * 1  1/6 (16.67%)  0/6 (0.00%) 
Back injury * 1  1/6 (16.67%)  0/6 (0.00%) 
Burns first degree * 1  0/6 (0.00%)  1/6 (16.67%) 
Cardiac function disturbance postoperative * 1  1/6 (16.67%)  0/6 (0.00%) 
Compression fracture * 1  1/6 (16.67%)  0/6 (0.00%) 
Concussion * 1  0/6 (0.00%)  1/6 (16.67%) 
Head injury * 1  0/6 (0.00%)  1/6 (16.67%) 
Laceration * 1  1/6 (16.67%)  1/6 (16.67%) 
Limb injury * 1  1/6 (16.67%)  1/6 (16.67%) 
Lip injury * 1  0/6 (0.00%)  1/6 (16.67%) 
Lower limb fracture * 1  1/6 (16.67%)  0/6 (0.00%) 
Nail injury * 1  1/6 (16.67%)  0/6 (0.00%) 
Post procedural haematoma * 1  0/6 (0.00%)  1/6 (16.67%) 
Postoperative respiratory distress * 1  1/6 (16.67%)  0/6 (0.00%) 
Radius fracture * 1  1/6 (16.67%)  0/6 (0.00%) 
Sunburn * 1  0/6 (0.00%)  1/6 (16.67%) 
Thermal burn * 1  0/6 (0.00%)  1/6 (16.67%) 
Tibia fracture * 1  1/6 (16.67%)  1/6 (16.67%) 
Humerus fracture * 1  1/6 (16.67%)  0/6 (0.00%) 
Scratch * 1  0/6 (0.00%)  1/6 (16.67%) 
Torus fracture * 1  0/6 (0.00%)  1/6 (16.67%) 
Ulna fracture * 1  1/6 (16.67%)  0/6 (0.00%) 
Investigations     
Activated partial thromboplastin time prolonged * 1  0/6 (0.00%)  4/6 (66.67%) 
C-reactive protein increased * 1  2/6 (33.33%)  2/6 (33.33%) 
Blood glucose increased * 1  2/6 (33.33%)  1/6 (16.67%) 
Blood creatine phosphokinase increased * 1  2/6 (33.33%)  0/6 (0.00%) 
Body height below normal * 1  1/6 (16.67%)  1/6 (16.67%) 
Activated partial thromboplastin time abnormal * 1  0/6 (0.00%)  1/6 (16.67%) 
Blood creatinine increased * 1  0/6 (0.00%)  1/6 (16.67%) 
Blood urea increased * 1  0/6 (0.00%)  1/6 (16.67%) 
Lymphocyte count decreased * 1  0/6 (0.00%)  1/6 (16.67%) 
Neutrophil count increased * 1  0/6 (0.00%)  1/6 (16.67%) 
Red blood cells urine positive * 1  0/6 (0.00%)  1/6 (16.67%) 
White blood cell count decreased * 1  0/6 (0.00%)  1/6 (16.67%) 
Wound healing normal * 1  0/6 (0.00%)  1/6 (16.67%) 
Metabolism and nutrition disorders     
Hypokalaemia * 1  2/6 (33.33%)  2/6 (33.33%) 
Dehydration * 1  2/6 (33.33%)  1/6 (16.67%) 
Obesity * 1  2/6 (33.33%)  0/6 (0.00%) 
Vitamin D deficiency * 1  2/6 (33.33%)  0/6 (0.00%) 
Musculoskeletal and connective tissue disorders     
Pain in extremity * 1  4/6 (66.67%)  4/6 (66.67%) 
Arthralgia * 1  5/6 (83.33%)  4/6 (66.67%) 
Back pain * 1  6/6 (100.00%)  3/6 (50.00%) 
Muscle spasms * 1  2/6 (33.33%)  2/6 (33.33%) 
Musculoskeletal pain * 1  2/6 (33.33%)  2/6 (33.33%) 
Myalgia * 1  1/6 (16.67%)  2/6 (33.33%) 
Muscular weakness * 1  0/6 (0.00%)  3/6 (50.00%) 
Bone pain * 1  1/6 (16.67%)  0/6 (0.00%) 
Musculoskeletal chest pain * 1  1/6 (16.67%)  0/6 (0.00%) 
Neck pain * 1  0/6 (0.00%)  1/6 (16.67%) 
Scoliosis * 1  1/6 (16.67%)  0/6 (0.00%) 
Tendon disorder * 1  0/6 (0.00%)  1/6 (16.67%) 
Tendonitis * 1  0/6 (0.00%)  1/6 (16.67%) 
Foot deformity * 1  0/6 (0.00%)  1/6 (16.67%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Skin papilloma * 1  0/6 (0.00%)  1/6 (16.67%) 
Nervous system disorders     
Headache * 1  4/6 (66.67%)  5/6 (83.33%) 
Balance disorder * 1  2/6 (33.33%)  3/6 (50.00%) 
Dizziness * 1  1/6 (16.67%)  1/6 (16.67%) 
Psychomotor hyperactivity * 1  0/6 (0.00%)  1/6 (16.67%) 
Sinus headache * 1  0/6 (0.00%)  1/6 (16.67%) 
Somnolence * 1  1/6 (16.67%)  0/6 (0.00%) 
Syncope * 1  0/6 (0.00%)  1/6 (16.67%) 
Psychiatric disorders     
Anxiety disorder * 1  1/6 (16.67%)  0/6 (0.00%) 
Insomnia * 1  0/6 (0.00%)  1/6 (16.67%) 
Agitation * 1  1/6 (16.67%)  0/6 (0.00%) 
Renal and urinary disorders     
Proteinuria * 1  2/6 (33.33%)  4/6 (66.67%) 
Glycosuria * 1  0/6 (0.00%)  1/6 (16.67%) 
Hypercalciuria * 1  0/6 (0.00%)  1/6 (16.67%) 
Polyuria * 1  1/6 (16.67%)  0/6 (0.00%) 
Pollakiuria * 1  0/6 (0.00%)  1/6 (16.67%) 
Reproductive system and breast disorders     
Pelvic pain * 1  1/6 (16.67%)  0/6 (0.00%) 
Testicular pain * 1  1/6 (16.67%)  0/6 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Oropharyngeal pain * 1  4/6 (66.67%)  4/6 (66.67%) 
Nasal congestion * 1  5/6 (83.33%)  2/6 (33.33%) 
Cough * 1  2/6 (33.33%)  4/6 (66.67%) 
Epistaxis * 1  1/6 (16.67%)  2/6 (33.33%) 
Pharyngeal erythema * 1  1/6 (16.67%)  1/6 (16.67%) 
Rhinorrhoea * 1  0/6 (0.00%)  3/6 (50.00%) 
Sleep apnoea syndrome * 1  3/6 (50.00%)  0/6 (0.00%) 
Upper respiratory tract congestion * 1  2/6 (33.33%)  0/6 (0.00%) 
Productive cough * 1  0/6 (0.00%)  1/6 (16.67%) 
Respiratory disorder * 1  1/6 (16.67%)  0/6 (0.00%) 
Sinus congestion * 1  1/6 (16.67%)  0/6 (0.00%) 
Sneezing * 1  1/6 (16.67%)  0/6 (0.00%) 
Upper-airway cough syndrome * 1  0/6 (0.00%)  1/6 (16.67%) 
Skin and subcutaneous tissue disorders     
Dermatitis contact * 1  2/6 (33.33%)  1/6 (16.67%) 
Rash * 1  3/6 (50.00%)  1/6 (16.67%) 
Ecchymosis * 1  1/6 (16.67%)  2/6 (33.33%) 
Erythema * 1  1/6 (16.67%)  2/6 (33.33%) 
Papule * 1  1/6 (16.67%)  1/6 (16.67%) 
Alopecia * 1  0/6 (0.00%)  1/6 (16.67%) 
Dermatitis bullous * 1  1/6 (16.67%)  0/6 (0.00%) 
Ingrowing nail * 1  1/6 (16.67%)  1/6 (16.67%) 
Intertrigo * 1  1/6 (16.67%)  0/6 (0.00%) 
Keloid scar * 1  0/6 (0.00%)  1/6 (16.67%) 
Nail discolouration * 1  1/6 (16.67%)  0/6 (0.00%) 
Nail dystrophy * 1  1/6 (16.67%)  0/6 (0.00%) 
Petechiae * 1  1/6 (16.67%)  0/6 (0.00%) 
Pruritus * 1  1/6 (16.67%)  1/6 (16.67%) 
Rash papular * 1  1/6 (16.67%)  0/6 (0.00%) 
Skin erosion * 1  1/6 (16.67%)  0/6 (0.00%) 
Urticaria * 1  0/6 (0.00%)  1/6 (16.67%) 
Urticaria thermal * 1  1/6 (16.67%)  0/6 (0.00%) 
Vascular disorders     
Haematoma * 1  1/6 (16.67%)  1/6 (16.67%) 
Flushing * 1  0/6 (0.00%)  1/6 (16.67%) 
1
Term from vocabulary, MedDRA (14.0)
*
Indicates events were collected by non-systematic assessment
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.
There is an agreement between the Principal Investigators and the Sponsor (or its agents) that restricts the PIs' rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Medical Director
Organization: Sarepta Therapeutics, Inc.
Phone: +1-888-727-3782
Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT01540409     History of Changes
Other Study ID Numbers: 4658-us-202
First Submitted: February 23, 2012
First Posted: February 28, 2012
Results First Submitted: June 20, 2019
Results First Posted: July 10, 2019
Last Update Posted: July 29, 2019