Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
Sponsor
Sarepta Therapeutics, Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT01540409
Collaborator
(none)
12
11
1
65.6
1.1
0
Study Details
Study Description
Brief Summary
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection 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 or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
This is an open label, multiple dose extension study to assess the ongoing efficacy, safety, and tolerability of weekly intravenous (IV) infusions of eteplirsen in DMD subjects who have successfully completed Study 4658-us 201.
Subjects will have the opportunity to enroll in this study during the last visit of Study 4658-us-201 (Week 28). Eligible subjects will receive once weekly IV infusions of eteplirsen (50 or 30 mg/kg) for an additional 212 weeks. Subjects will receive the same dose of eteplirsen they received in Study 4658-us-201. Subjects will thereafter continue to receive once weekly IV infusions of eteplirsen for up to an additional 72 week period (through week 284). If commercial eteplirsen becomes available during this additional 72 week period, participation in the study will be discontinued as subjects transition to commercial eteplirsen.
Safety, efficacy, pharmacokinetic (PK), and biomarker assessments will be performed at scheduled visits; adverse events (AEs) and concomitant medications and therapies will be continuously monitored.
If review of data from this open label study suggests that continued treatment with eteplirsen is warranted, this study may be extended by protocol amendment or subjects who successfully complete this study may have the opportunity to participate in a separate follow on, open label eteplirsen study.
Study Design
Study Type:
Interventional
Actual Enrollment
:
12 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (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
Actual Study Start Date
:
Feb 27, 2012
Actual Primary Completion Date
:
Apr 15, 2016
Actual Study Completion Date
:
Aug 16, 2017
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: AVI-4658 (Eteplirsen) Multiple-Dose Extension Study |
Drug: AVI-4658 (Eteplirsen)
Eteplirsen will be administered once weekly via an IV infusion. There are two treatment groups, 30 mg/kg and 50 mg/kg.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Change From Baseline in the 6 Minute Walk Test (6MWT) at Week 240 [Parent Baseline and Week 240]
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).
- Change From Baseline in the Percentage of Dystrophin Positive Fibers (PDPF) at Week 48 [Parent Baseline and Week 48]
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).
Eligibility Criteria
Criteria
Ages Eligible for Study:
7 Years
to 13 Years
Sexes Eligible for Study:
Male
Accepts Healthy Volunteers:
No
Inclusion Criteria:
A subject must meet all of the following criteria to be eligible for this study.
-
The subject and/or their parent/legal guardian are willing and able to provide signed informed consent.
-
The subject has successfully completed 28 weeks of treatment in Study 4658-US-201.
-
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.
- 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
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Miller Children's Hospital | Long Beach | California | United States | 90806 |
| 2 | University of Florida Clinical Research Center | Gainesville | Florida | United States | 32610 |
| 3 | Rush University Medical Center | Chicago | Illinois | United States | 60612 |
| 4 | Massachusetts General Hospital | Boston | Massachusetts | United States | 02114 |
| 5 | Washington University Medical School | Saint Louis | Missouri | United States | 63110 |
| 6 | Summerwood Pediatrics/Infusacare Medical Services | Liverpool | New York | United States | 13088 |
| 7 | Levine Children's Hospital | Charlotte | North Carolina | United States | 28203 |
| 8 | Nationwide Children's Hospital | Columbus | Ohio | United States | 43205 |
| 9 | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | United States | 15224 |
| 10 | Children's Specialty Group, Pediatric Neurology | Norfolk | Virginia | United States | 23510 |
| 11 | Osceola Medical Center | Osceola | Wisconsin | United States | 54020 |
Sponsors and Collaborators
- Sarepta Therapeutics, Inc.
Investigators
- Study Director: Medical Director, Sarepta Therapeutics, Inc.
Study Documents (Full-Text)
None provided.More Information
Additional Information:
- Mendell, 2013, Annals of Neurology
- Mendell, 2016, Annals of Neurology
- Kinane, 2018, Journal of Neuromuscular Diseases
- Charleston, 2018, Neurology
Publications
None provided.Responsible Party:
Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT01540409
Other Study ID Numbers:
- 4658-us-202
First Posted:
Feb 28, 2012
Last Update Posted:
Mar 30, 2020
Last Verified:
Mar 1, 2020
Keywords provided by Sarepta Therapeutics, Inc.
Additional relevant MeSH terms:
Study Results
Participant Flow
| 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 Detail | 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 |
|---|---|---|
| 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 |
Baseline Characteristics
| Arm/Group Title | Eteplirsen 30 mg/kg | Eteplirsen 50 mg/kg | Total |
|---|---|---|---|
| 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 Participants | 6 | 6 | 12 |
| Age (years) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [years] |
9.2
(0.75)
|
8.8
(1.47)
|
9.0
(1.13)
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
0
0%
|
0
0%
|
0
0%
|
| Male |
6
100%
|
6
100%
|
12
100%
|
| Region of Enrollment (Count of Participants) | |||
| United States |
6
100%
|
6
100%
|
12
100%
|
Outcome Measures
| Title | Change From Baseline in the 6 Minute Walk Test (6MWT) at Week 240 |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 4 | 3 |
| Mean (Standard Deviation) [Meters] |
-199.0
(113.25)
|
-258.0
(175.65)
|
| Title | Change From Baseline in the Percentage of Dystrophin Positive Fibers (PDPF) at Week 48 |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|---|
| 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. |
| Measure Participants | 4 | 4 | 4 |
| Mean (Standard Deviation) [Percentage of Dystrophin Positive Fibers] |
37.70
(12.602)
|
51.69
(7.089)
|
42.93
(13.433)
|
Adverse Events
| 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 | ||
| 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 (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/6 (0%) | 0/6 (0%) | ||
Serious Adverse Events |
||||
| Eteplirsen 30 mg/kg | Eteplirsen 50 mg/kg | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 4/6 (66.7%) | 2/6 (33.3%) | ||
| Injury, poisoning and procedural complications | ||||
| Tibia Fracture | 0/6 (0%) | 1/6 (16.7%) | ||
| Femur Fracture | 3/6 (50%) | 1/6 (16.7%) | ||
| Musculoskeletal and connective tissue disorders | ||||
| Scoliosis | 2/6 (33.3%) | 0/6 (0%) | ||
| Respiratory, thoracic and mediastinal disorders | ||||
| Acute respiratory failure | 1/6 (16.7%) | 0/6 (0%) | ||
Other (Not Including Serious) Adverse Events |
||||
| Eteplirsen 30 mg/kg | Eteplirsen 50 mg/kg | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 6/6 (100%) | 6/6 (100%) | ||
| Blood and lymphatic system disorders | ||||
| Anaemia | 2/6 (33.3%) | 0/6 (0%) | ||
| Cardiac disorders | ||||
| Tachycardia | 1/6 (16.7%) | 0/6 (0%) | ||
| Congenital, familial and genetic disorders | ||||
| Cryptorchism | 1/6 (16.7%) | 0/6 (0%) | ||
| Ear and labyrinth disorders | ||||
| Cerumen impaction | 1/6 (16.7%) | 0/6 (0%) | ||
| Motion sickness | 0/6 (0%) | 1/6 (16.7%) | ||
| Tympanic membrane disorder | 0/6 (0%) | 1/6 (16.7%) | ||
| Endocrine disorders | ||||
| Goitre | 0/6 (0%) | 1/6 (16.7%) | ||
| Growth hormone deficiency | 0/6 (0%) | 1/6 (16.7%) | ||
| Cushingoid | 0/6 (0%) | 2/6 (33.3%) | ||
| Eye disorders | ||||
| Cataract | 0/6 (0%) | 2/6 (33.3%) | ||
| Cataract subcapsular | 1/6 (16.7%) | 0/6 (0%) | ||
| Conjunctivitis | 1/6 (16.7%) | 0/6 (0%) | ||
| Erythema of eyelid | 0/6 (0%) | 1/6 (16.7%) | ||
| Hypermetropia | 1/6 (16.7%) | 0/6 (0%) | ||
| Gastrointestinal disorders | ||||
| Vomiting | 5/6 (83.3%) | 4/6 (66.7%) | ||
| Abdominal pain upper | 1/6 (16.7%) | 3/6 (50%) | ||
| Dyspepsia | 3/6 (50%) | 1/6 (16.7%) | ||
| Abdominal pain | 0/6 (0%) | 2/6 (33.3%) | ||
| Constipation | 2/6 (33.3%) | 2/6 (33.3%) | ||
| Diarrhoea | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Nausea | 2/6 (33.3%) | 3/6 (50%) | ||
| Oral pain | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Abdominal discomfort | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Dental caries | 1/6 (16.7%) | 0/6 (0%) | ||
| Dysphagia | 1/6 (16.7%) | 0/6 (0%) | ||
| Flatulence | 1/6 (16.7%) | 0/6 (0%) | ||
| Food poisoning | 0/6 (0%) | 1/6 (16.7%) | ||
| Haemorrhoids | 0/6 (0%) | 1/6 (16.7%) | ||
| Lip swelling | 0/6 (0%) | 1/6 (16.7%) | ||
| Retained deciduous tooth | 1/6 (16.7%) | 0/6 (0%) | ||
| Tooth impacted | 0/6 (0%) | 1/6 (16.7%) | ||
| Toothache | 1/6 (16.7%) | 0/6 (0%) | ||
| Gastritis | 1/6 (16.7%) | 0/6 (0%) | ||
| Tooth disorder | 1/6 (16.7%) | 0/6 (0%) | ||
| General disorders | ||||
| Pyrexia | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Catheter site pain | 2/6 (33.3%) | 2/6 (33.3%) | ||
| Infusion site extravasation | 2/6 (33.3%) | 2/6 (33.3%) | ||
| Device occlusion | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Infusion site haematoma | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Oedema peripheral | 2/6 (33.3%) | 0/6 (0%) | ||
| Thrombosis in device | 2/6 (33.3%) | 1/6 (16.7%) | ||
| Application site erythema | 0/6 (0%) | 1/6 (16.7%) | ||
| Application site pruritus | 0/6 (0%) | 1/6 (16.7%) | ||
| Catheter site haematoma | 0/6 (0%) | 2/6 (33.3%) | ||
| Catheter site haemorrhage | 0/6 (0%) | 1/6 (16.7%) | ||
| Catheter site inflammation | 0/6 (0%) | 1/6 (16.7%) | ||
| Catheter site related reaction | 0/6 (0%) | 1/6 (16.7%) | ||
| Chest pain | 1/6 (16.7%) | 0/6 (0%) | ||
| Influenza like illness | 1/6 (16.7%) | 0/6 (0%) | ||
| Infusion site pain | 0/6 (0%) | 1/6 (16.7%) | ||
| Infusion site rash | 1/6 (16.7%) | 0/6 (0%) | ||
| Injection site pain | 0/6 (0%) | 1/6 (16.7%) | ||
| Irritability | 1/6 (16.7%) | 0/6 (0%) | ||
| Malaise | 0/6 (0%) | 1/6 (16.7%) | ||
| Non-cardiac chest pain | 1/6 (16.7%) | 0/6 (0%) | ||
| Pain | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Swelling | 0/6 (0%) | 1/6 (16.7%) | ||
| Disease progression | 0/6 (0%) | 1/6 (16.7%) | ||
| Infusion site urticaria | 1/6 (16.7%) | 0/6 (0%) | ||
| Immune system disorders | ||||
| Hypersensitivity | 0/6 (0%) | 1/6 (16.7%) | ||
| Seasonal allergy | 0/6 (0%) | 1/6 (16.7%) | ||
| Infections and infestations | ||||
| Nasopharyngitis | 4/6 (66.7%) | 5/6 (83.3%) | ||
| Upper respiratory tract infection | 2/6 (33.3%) | 4/6 (66.7%) | ||
| Gastroenteritis viral | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Hordeolum | 0/6 (0%) | 3/6 (50%) | ||
| Influenza | 0/6 (0%) | 3/6 (50%) | ||
| Post procedural cellulitis | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Rhinitis | 0/6 (0%) | 1/6 (16.7%) | ||
| Viral infection | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Candidiasis | 1/6 (16.7%) | 0/6 (0%) | ||
| Folliculitis | 0/6 (0%) | 1/6 (16.7%) | ||
| Gastroenteritis | 1/6 (16.7%) | 0/6 (0%) | ||
| Incision site infection | 0/6 (0%) | 1/6 (16.7%) | ||
| Pharyngitis streptococcal | 1/6 (16.7%) | 0/6 (0%) | ||
| Pneumonia | 1/6 (16.7%) | 0/6 (0%) | ||
| Sinusitis | 1/6 (16.7%) | 0/6 (0%) | ||
| Tinea capitis | 1/6 (16.7%) | 0/6 (0%) | ||
| Tinea pedis | 1/6 (16.7%) | 0/6 (0%) | ||
| Viral upper respiratory tract infection | 1/6 (16.7%) | 0/6 (0%) | ||
| Abscess | 0/6 (0%) | 1/6 (16.7%) | ||
| Abscess limb | 0/6 (0%) | 1/6 (16.7%) | ||
| Bacterial disease carrier | 1/6 (16.7%) | 0/6 (0%) | ||
| Localised infection | 0/6 (0%) | 1/6 (16.7%) | ||
| Paraspinal abscess | 0/6 (0%) | 1/6 (16.7%) | ||
| Pharyngitis | 1/6 (16.7%) | 0/6 (0%) | ||
| Injury, poisoning and procedural complications | ||||
| Procedural pain | 5/6 (83.3%) | 6/6 (100%) | ||
| Contusion | 4/6 (66.7%) | 4/6 (66.7%) | ||
| Arthropod bite | 3/6 (50%) | 2/6 (33.3%) | ||
| Excoriation | 2/6 (33.3%) | 3/6 (50%) | ||
| Joint injury | 2/6 (33.3%) | 1/6 (16.7%) | ||
| Joint sprain | 0/6 (0%) | 3/6 (50%) | ||
| Muscle strain | 2/6 (33.3%) | 2/6 (33.3%) | ||
| Fall | 1/6 (16.7%) | 0/6 (0%) | ||
| Foot fracture | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Incision site haemorrhage | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Incision site pain | 1/6 (16.7%) | 0/6 (0%) | ||
| Arthropod sting | 1/6 (16.7%) | 0/6 (0%) | ||
| Back injury | 1/6 (16.7%) | 0/6 (0%) | ||
| Burns first degree | 0/6 (0%) | 1/6 (16.7%) | ||
| Cardiac function disturbance postoperative | 1/6 (16.7%) | 0/6 (0%) | ||
| Compression fracture | 1/6 (16.7%) | 0/6 (0%) | ||
| Concussion | 0/6 (0%) | 1/6 (16.7%) | ||
| Head injury | 0/6 (0%) | 1/6 (16.7%) | ||
| Laceration | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Limb injury | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Lip injury | 0/6 (0%) | 1/6 (16.7%) | ||
| Lower limb fracture | 1/6 (16.7%) | 0/6 (0%) | ||
| Nail injury | 1/6 (16.7%) | 0/6 (0%) | ||
| Post procedural haematoma | 0/6 (0%) | 1/6 (16.7%) | ||
| Postoperative respiratory distress | 1/6 (16.7%) | 0/6 (0%) | ||
| Radius fracture | 1/6 (16.7%) | 0/6 (0%) | ||
| Sunburn | 0/6 (0%) | 1/6 (16.7%) | ||
| Thermal burn | 0/6 (0%) | 1/6 (16.7%) | ||
| Tibia fracture | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Humerus fracture | 1/6 (16.7%) | 0/6 (0%) | ||
| Scratch | 0/6 (0%) | 1/6 (16.7%) | ||
| Torus fracture | 0/6 (0%) | 1/6 (16.7%) | ||
| Ulna fracture | 1/6 (16.7%) | 0/6 (0%) | ||
| Investigations | ||||
| Activated partial thromboplastin time prolonged | 0/6 (0%) | 4/6 (66.7%) | ||
| C-reactive protein increased | 2/6 (33.3%) | 2/6 (33.3%) | ||
| Blood glucose increased | 2/6 (33.3%) | 1/6 (16.7%) | ||
| Blood creatine phosphokinase increased | 2/6 (33.3%) | 0/6 (0%) | ||
| Body height below normal | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Activated partial thromboplastin time abnormal | 0/6 (0%) | 1/6 (16.7%) | ||
| Blood creatinine increased | 0/6 (0%) | 1/6 (16.7%) | ||
| Blood urea increased | 0/6 (0%) | 1/6 (16.7%) | ||
| Lymphocyte count decreased | 0/6 (0%) | 1/6 (16.7%) | ||
| Neutrophil count increased | 0/6 (0%) | 1/6 (16.7%) | ||
| Red blood cells urine positive | 0/6 (0%) | 1/6 (16.7%) | ||
| White blood cell count decreased | 0/6 (0%) | 1/6 (16.7%) | ||
| Wound healing normal | 0/6 (0%) | 1/6 (16.7%) | ||
| Metabolism and nutrition disorders | ||||
| Hypokalaemia | 2/6 (33.3%) | 2/6 (33.3%) | ||
| Dehydration | 2/6 (33.3%) | 1/6 (16.7%) | ||
| Obesity | 2/6 (33.3%) | 0/6 (0%) | ||
| Vitamin D deficiency | 2/6 (33.3%) | 0/6 (0%) | ||
| Musculoskeletal and connective tissue disorders | ||||
| Pain in extremity | 4/6 (66.7%) | 4/6 (66.7%) | ||
| Arthralgia | 5/6 (83.3%) | 4/6 (66.7%) | ||
| Back pain | 6/6 (100%) | 3/6 (50%) | ||
| Muscle spasms | 2/6 (33.3%) | 2/6 (33.3%) | ||
| Musculoskeletal pain | 2/6 (33.3%) | 2/6 (33.3%) | ||
| Myalgia | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Muscular weakness | 0/6 (0%) | 3/6 (50%) | ||
| Bone pain | 1/6 (16.7%) | 0/6 (0%) | ||
| Musculoskeletal chest pain | 1/6 (16.7%) | 0/6 (0%) | ||
| Neck pain | 0/6 (0%) | 1/6 (16.7%) | ||
| Scoliosis | 1/6 (16.7%) | 0/6 (0%) | ||
| Tendon disorder | 0/6 (0%) | 1/6 (16.7%) | ||
| Tendonitis | 0/6 (0%) | 1/6 (16.7%) | ||
| Foot deformity | 0/6 (0%) | 1/6 (16.7%) | ||
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ||||
| Skin papilloma | 0/6 (0%) | 1/6 (16.7%) | ||
| Nervous system disorders | ||||
| Headache | 4/6 (66.7%) | 5/6 (83.3%) | ||
| Balance disorder | 2/6 (33.3%) | 3/6 (50%) | ||
| Dizziness | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Psychomotor hyperactivity | 0/6 (0%) | 1/6 (16.7%) | ||
| Sinus headache | 0/6 (0%) | 1/6 (16.7%) | ||
| Somnolence | 1/6 (16.7%) | 0/6 (0%) | ||
| Syncope | 0/6 (0%) | 1/6 (16.7%) | ||
| Psychiatric disorders | ||||
| Anxiety disorder | 1/6 (16.7%) | 0/6 (0%) | ||
| Insomnia | 0/6 (0%) | 1/6 (16.7%) | ||
| Agitation | 1/6 (16.7%) | 0/6 (0%) | ||
| Renal and urinary disorders | ||||
| Proteinuria | 2/6 (33.3%) | 4/6 (66.7%) | ||
| Glycosuria | 0/6 (0%) | 1/6 (16.7%) | ||
| Hypercalciuria | 0/6 (0%) | 1/6 (16.7%) | ||
| Polyuria | 1/6 (16.7%) | 0/6 (0%) | ||
| Pollakiuria | 0/6 (0%) | 1/6 (16.7%) | ||
| Reproductive system and breast disorders | ||||
| Pelvic pain | 1/6 (16.7%) | 0/6 (0%) | ||
| Testicular pain | 1/6 (16.7%) | 0/6 (0%) | ||
| Respiratory, thoracic and mediastinal disorders | ||||
| Oropharyngeal pain | 4/6 (66.7%) | 4/6 (66.7%) | ||
| Nasal congestion | 5/6 (83.3%) | 2/6 (33.3%) | ||
| Cough | 2/6 (33.3%) | 4/6 (66.7%) | ||
| Epistaxis | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Pharyngeal erythema | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Rhinorrhoea | 0/6 (0%) | 3/6 (50%) | ||
| Sleep apnoea syndrome | 3/6 (50%) | 0/6 (0%) | ||
| Upper respiratory tract congestion | 2/6 (33.3%) | 0/6 (0%) | ||
| Productive cough | 0/6 (0%) | 1/6 (16.7%) | ||
| Respiratory disorder | 1/6 (16.7%) | 0/6 (0%) | ||
| Sinus congestion | 1/6 (16.7%) | 0/6 (0%) | ||
| Sneezing | 1/6 (16.7%) | 0/6 (0%) | ||
| Upper-airway cough syndrome | 0/6 (0%) | 1/6 (16.7%) | ||
| Skin and subcutaneous tissue disorders | ||||
| Dermatitis contact | 2/6 (33.3%) | 1/6 (16.7%) | ||
| Rash | 3/6 (50%) | 1/6 (16.7%) | ||
| Ecchymosis | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Erythema | 1/6 (16.7%) | 2/6 (33.3%) | ||
| Papule | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Alopecia | 0/6 (0%) | 1/6 (16.7%) | ||
| Dermatitis bullous | 1/6 (16.7%) | 0/6 (0%) | ||
| Ingrowing nail | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Intertrigo | 1/6 (16.7%) | 0/6 (0%) | ||
| Keloid scar | 0/6 (0%) | 1/6 (16.7%) | ||
| Nail discolouration | 1/6 (16.7%) | 0/6 (0%) | ||
| Nail dystrophy | 1/6 (16.7%) | 0/6 (0%) | ||
| Petechiae | 1/6 (16.7%) | 0/6 (0%) | ||
| Pruritus | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Rash papular | 1/6 (16.7%) | 0/6 (0%) | ||
| Skin erosion | 1/6 (16.7%) | 0/6 (0%) | ||
| Urticaria | 0/6 (0%) | 1/6 (16.7%) | ||
| Urticaria thermal | 1/6 (16.7%) | 0/6 (0%) | ||
| Vascular disorders | ||||
| Haematoma | 1/6 (16.7%) | 1/6 (16.7%) | ||
| Flushing | 0/6 (0%) | 1/6 (16.7%) | ||
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
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 |
| clinicaltrials@sarepta.com |
Responsible Party:
Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT01540409
Other Study ID Numbers:
- 4658-us-202
First Posted:
Feb 28, 2012
Last Update Posted:
Mar 30, 2020
Last Verified:
Mar 1, 2020