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Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage 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: NCT02530905
Recruitment Status : Completed
First Posted : August 21, 2015
Results First Posted : May 17, 2021
Last Update Posted : May 17, 2021
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Duchenne Muscular Dystrophy
Interventions Drug: SRP-4045
Drug: Placebo
Enrollment 12
Recruitment Details The study was conducted at 3 sites in United States.
Pre-assignment Details Study conducted in 2 parts: Part 1 (Double-Blind Period [DBP]) and Part 2 (Open Label Extension Period [OLEP]). When Part 1 was completed and cumulative safety data was reviewed by an independent Data Safety Monitoring Board (DSMB), Part 2 was conducted.
Arm/Group Title Double-Blind Period: Placebo Double-Blind Period: Casimersen Open Label Extension Period: Casimersen
Hide Arm/Group Description Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) characterized by deletions amenable to exon 45 skipping received placebo-matched to casimersen intravenous (IV) infusions, once weekly over approximately 12 weeks in the double-blind period. Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received weekly IV infusions of casimersen at four escalating dose levels, each for at least 2 weeks: 4 milligrams per kilograms (mg/kg) during Week 1 to Week 2, followed by 10 mg/kg during Week 3 to Week 4, followed by 20 mg/kg during Week 5 to Week 6, followed by 30 mg/kg beginning at Week 7 and continued over approximately Week 12 in the double-blind period. All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Period Title: Double-Blind Period (12 Weeks)
Started 4 8 0
Received 4 mg/kg 0 8 0
Received 10 mg/kg 0 8 0
Received 20 mg/kg 0 8 0
Received 30 mg/kg 0 8 0
Completed 4 8 0
Not Completed 0 0 0
Period Title: Open Label Extension Period (132 Weeks)
Started 0 0 12 [1]
Completed 0 0 11
Not Completed 0 0 1
Reason Not Completed
Withdrawal by Subject             0             0             1
[1]
All participants from the double-blind period amenable to exon 45 skipping were enrolled to open-label extension period.
Arm/Group Title Double-Blind Period: Placebo Double-Blind Period: Casimersen Total
Hide Arm/Group Description Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received placebo-matched to casimersen IV infusions, once weekly over approximately 12 weeks in the double-blind period. Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received weekly IV infusions of casimersen at four escalating dose levels, each for at least 2 weeks: 4 mg/kg during Week 1 to Week 2, followed by 10 mg/kg during Week 3 to Week 4, followed by 20 mg/kg during Week 5 to Week 6, followed by 30 mg/kg beginning at Week 7 and continued over approximately Week 12 in the double-blind period. Total of all reporting groups
Overall Number of Baseline Participants 4 8 12
Hide Baseline Analysis Population Description
Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in double-blind period.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 8 participants 12 participants
12.0  (2.16) 14.4  (3.29) 13.6  (3.09)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 8 participants 12 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
4
 100.0%
8
 100.0%
12
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 8 participants 12 participants
Hispanic or Latino
0
   0.0%
1
  12.5%
1
   8.3%
Not Hispanic or Latino
4
 100.0%
7
  87.5%
11
  91.7%
Unknown or Not Reported
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 8 participants 12 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
2
  25.0%
2
  16.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
4
 100.0%
6
  75.0%
10
  83.3%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Hide Description Adverse event (AE) was any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. AEs also included abnormal physical examination findings (Physical examination were conducted per protocol and any clinically significant abnormal findings were recorded in medical history if pre-existing or addressed as an AE if new or worsening). TEAEs was defined as AEs that started, worsened, or became serious on or after the start of first infusion through 148 weeks. Number of participants with TEAEs were reported.
Time Frame Baseline up to Week 148
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in both double-blind and open-label extension periods.
Arm/Group Title Double-Blind Period: Placebo Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received placebo-matched to casimersen IV infusions, once weekly over approximately 12 weeks in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 4 8 8 8 8 12
Measure Type: Count of Participants
Unit of Measure: Participants
4
 100.0%
5
  62.5%
3
  37.5%
3
  37.5%
7
  87.5%
12
 100.0%
2.Primary Outcome
Title Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs
Hide Description Laboratory parameters included serum chemistry (hepatic chemistry and renal chemistry), hematology, coagulation, and urinalysis. Number of participants with potentially clinically significant abnormal finding were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potentially clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Time Frame Baseline up to Week 148
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in both double-blind and open-label extension periods.
Arm/Group Title Double-Blind Period: Placebo Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received placebo-matched to casimersen IV infusions, once weekly over approximately 12 weeks in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 4 8 8 8 8 12
Measure Type: Count of Participants
Unit of Measure: Participants
Serum chemistry: Hepatic chemistry
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Serum chemistry: Renal chemistry
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Hematolgy: Leukocytes
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
2
  25.0%
4
  33.3%
Hematolgy: Neutrophils
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
4
  50.0%
6
  50.0%
Coagulation: Platelet count
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
2
  25.0%
2
  16.7%
Urinalysis
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   8.3%
3.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs
Hide Description Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potential clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Time Frame Baseline up to Week 148
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in both double-blind and open-label extension periods.
Arm/Group Title Double-Blind Period: Placebo Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received placebo-matched to casimersen IV infusions, once weekly over approximately 12 weeks in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period
Overall Number of Participants Analyzed 4 8 8 8 8 12
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
  12.5%
1
   8.3%
4.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Abnormalities in Electrocardiogram (ECG) Reported as TEAEs
Hide Description Twelve-lead ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECG reported as TEAEs were presented here. The Investigator determined whether abnormal assessment results were potentially clinically significant or not.
Time Frame Baseline up to Week 148
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in both double-blind and open-label extension periods.
Arm/Group Title Double-Blind Period: Placebo Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received placebo-matched to casimersen IV infusions, once weekly over approximately 12 weeks in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 4 8 8 8 8 12
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   8.3%
5.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Abnormalities in Echocardiograms (ECHO)
Hide Description Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study.The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECHO were reported.
Time Frame Baseline up to Week 148
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in both double-blind and open-label extension periods.
Arm/Group Title Double-Blind Period: Placebo Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received placebo-matched to casimersen IV infusions, once weekly over approximately 12 weeks in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 4 8 8 8 8 12
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
6.Secondary Outcome
Title Maximum Plasma Concentration (Cmax) of Casimersen
Hide Description Maximum Concentration (Cmax) of casimersen in plasma was evaluated.
Time Frame Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic (PK) set included all randomized participants who received the planned full dose of study drug (casimersen) and for whom there are adequate PK samples from which to estimate PK parameters. Data was not planned to be collected and analyzed for placebo arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 8 8 8 8 12
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: Nanogram per milliliter (ng/mL)
13700
(25.0%)
39400
(11.7%)
64400
(27.4%)
119000
(33.6%)
115000
(31.5%)
7.Secondary Outcome
Title Time to Reach Maximum Plasma Concentration (Tmax) of Casimersen
Hide Description Time to reach maximum plasma concentration (Tmax) of casimersen was evaluated.
Time Frame Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Hide Outcome Measure Data
Hide Analysis Population Description
PK set included all randomized participants who received the planned full dose of study drug (casimersen or placebo) and for whom there are adequate PK samples from which to estimate PK parameters. Data was not planned to be collected and analyzed for placebo arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 8 8 8 8 12
Median (Full Range)
Unit of Measure: Hour
1.11
(0.92 to 1.22)
1.03
(0.93 to 1.17)
1.03
(0.83 to 1.22)
0.94
(0.83 to 1.18)
0.95
(0.78 to 1.08)
8.Secondary Outcome
Title Area Under Concentration-time Curve From Time of Dosing to the Last Measurable Concentration (AUClast) of Casimersen in Plasma
Hide Description Area under the concentration-time curve from time zero to the last quantifiable concentration of casimersen in plasma were evaluated.
Time Frame Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Hide Outcome Measure Data
Hide Analysis Population Description
PK set included all randomized participants who received the planned full dose of study drug (casimersen or placebo) and for whom there are adequate PK samples from which to estimate PK parameters. Data was not planned to be collected and analyzed for placebo arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 8 8 8 8 12
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: Hour*nanogram per milliliter (hr*ng/mL)
23100
(29.5%)
59500
(16.2%)
101000
(17.7%)
188000
(27.4%)
182000
(33.8%)
9.Secondary Outcome
Title Area Under Concentration-Time Curve From Time Zero Pre-dose to Twenty-Four Hours Post-dose (AUC0-24) of Casimersen in Plasma
Hide Description Area under concentration-time curve from time zero pre-dose to twenty-four hours post-dos of casimersen in plasma were evaluated.
Time Frame Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Hide Outcome Measure Data
Hide Analysis Population Description
PK set included all randomized participants who received the planned full dose of study drug (casimersen or placebo) and for whom there are adequate PK samples from which to estimate PK parameters. Data was not planned to be collected and analyzed for placebo arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 8 8 8 8 12
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: h*ng/mL
23200
(29.4%)
59500
(16.1%)
101000
(17.7%)
188000
(27.4%)
182000
(33.8%)
10.Secondary Outcome
Title Area Under the Concentration-Time Curve From Time Zero Extrapolated to the Infinity (AUCinf) of Casimersen in Plasma
Hide Description Area under the concentration-time curve from time zero extrapolated to the infinity of casimersen in plasma was evaluated.
Time Frame Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Hide Outcome Measure Data
Hide Analysis Population Description
PK set included all randomized participants who received the planned full dose of study drug (casimersen or placebo) and for whom there are adequate PK samples from which to estimate PK parameters. Here, "Overall number of participants analyzed" = number of participants evaluable for this outcome. Data was not planned to be collected and analyzed for placebo arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 8 7 8 8 12
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: h*ng/mL
23300
(29.5%)
58300
(16.0%)
101000
(17.7%)
189000
(27.5%)
182000
(33.9%)
11.Secondary Outcome
Title Apparent Volume of Distribution at Steady State (Vss) of Casimersen
Hide Description Volume of distribution was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Apparent volume of distribution at steady state of casimersen was evaluated.
Time Frame Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Hide Outcome Measure Data
Hide Analysis Population Description
PK set included all randomized participants who received the planned full dose of study drug (casimersen or placebo) and for whom there are adequate PK samples from which to estimate PK parameters. Here, "Overall number of participants analyzed" = number of participants evaluable for this outcome. Data was not planned to be collected and analyzed for placebo arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 8 7 8 8 12
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: Liter per kilogram (L/kg)
0.369
(24.4%)
0.343
(12.5%)
0.407
(23.4%)
0.319
(31.4%)
0.367
(28.9%)
12.Secondary Outcome
Title Elimination Half-life (T1/2) of Casimersen
Hide Description T1/2 is the time measured for the plasma concentration of drug to decrease by one half. T1/2 of casimersen was evaluated.
Time Frame Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Hide Outcome Measure Data
Hide Analysis Population Description
PK set included all randomized participants who received the planned full dose of study drug (casimersen or placebo) and for whom there are adequate PK samples from which to estimate PK parameters. Here, "Overall number of participants analyzed" = number of participants evaluable for this outcome. Data was not planned to be collected and analyzed for placebo arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 8 7 8 8 12
Mean (Standard Deviation)
Unit of Measure: Hour
2.92  (0.985) 3.29  (0.640) 3.71  (0.616) 3.82  (0.741) 3.45  (0.359)
13.Secondary Outcome
Title Total Clearance (CL) of Casimersen
Hide Description Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood. CL of casimersen was evaluated.
Time Frame Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Hide Outcome Measure Data
Hide Analysis Population Description
PK set included all randomized participants who received the planned full dose of study drug (casimersen or placebo) and for whom there are adequate PK samples from which to estimate PK parameters. Here, "Overall number of participants analyzed" = number of participants evaluable for this outcome. Data was not planned to be collected and analyzed for placebo arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 8 7 8 8 12
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: Liters per hour per kilogram (L/h/kg)
0.177
(29.1%)
0.181
(15.9%)
0.205
(18.5%)
0.163
(27.7%)
0.180
(35.0%)
14.Secondary Outcome
Title Mean Residence Time Extrapolated to Infinity (MRTinf) of Casimersen
Hide Description MRTinf = AUMCinf/AUCinf - T/2, where T was the infusion duration, and AUMCinf was the area under the first moment curve from time 0 extrapolated to infinite time, calculated using the linear/log trapezoidal method. MRTinf of casimersen was evaluated.
Time Frame Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Hide Outcome Measure Data
Hide Analysis Population Description
PK set included all randomized participants who received the planned full dose of study drug (casimersen or placebo) and for whom there are adequate PK samples from which to estimate PK parameters. Here, "Overall number of participants analyzed" = number of participants evaluable for this outcome. Data was not planned to be collected and analyzed for placebo arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
Overall Number of Participants Analyzed 8 7 8 8 12
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: Hour
2.08
(13.5%)
1.89
(16.1%)
1.98
(12.7%)
1.96
(16.6%)
2.04
(10.3%)
15.Secondary Outcome
Title Double-Blind Period: Renal Clearance (CLR) of Casimersen
Hide Description Renal clearance was calculated using the partial AUC0-24 from the non-compartmental analysis in plasma and AE0-24. AUC0-24 was defined as area under the plasma concentration-time curve, from time 0 to 24 hours after completion of dosing. AE0-24 was defined as total cumulative amount excreted from 0 to 24 hours. Summarized data of all urine collection intervals are reported.
Time Frame 0 to 4, 4 to 8, 8 to 12, and 12 to 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 and 12 (for 30 mg/kg arm) in double-blind period
Hide Outcome Measure Data
Hide Analysis Population Description
PK set included all randomized participants who received the planned full dose of study drug (casimersen or placebo) and for whom there are adequate PK samples from which to estimate PK parameters. Here, "Overall number of participants analyzed"= number of participants evaluable for this outcome. Data was not planned to be collected and analyzed for placebo and open-label extension period arm.
Arm/Group Title Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg
Hide Arm/Group Description:
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period.
Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period.
Overall Number of Participants Analyzed 8 8 7 8
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: L/h/kg
0.137
(25.8%)
0.162
(22.6%)
0.209
(29.0%)
0.177
(34.2%)
Time Frame From start of study drug administration up to Week 148
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Double-Blind Period: Placebo Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Hide Arm/Group Description Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received placebo-matched to casimersen IV infusions, once weekly over approximately 12 weeks in the double-blind period. Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 4 mg/kg once weekly for 2 weeks (i.e. Week 1 to Week 2) in the double-blind period. Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 10 mg/kg once weekly for 2 weeks (i.e. Week 3 to Week 4) in the double-blind period. Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 20 mg/kg once weekly for 2 weeks (i.e. Week 5 to Week 6) in the double-blind period. Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping received casimersen IV infusions, at a dose level of 30 mg/kg once weekly from Week 7 to Week 12 in the double-blind period. All participants who completed double blind period were enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.
All-Cause Mortality
Double-Blind Period: Placebo Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/8 (0.00%)   0/8 (0.00%)   0/8 (0.00%)   0/8 (0.00%)   0/12 (0.00%) 
Hide Serious Adverse Events
Double-Blind Period: Placebo Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/8 (0.00%)   0/8 (0.00%)   0/8 (0.00%)   1/8 (12.50%)   3/12 (25.00%) 
Infections and infestations             
Septic embolus * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
bacteraemia * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Injury, poisoning and procedural complications             
Femur fracture * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Tibia fracture * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Vascular disorders             
vena cava thrombosis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
1
Term from vocabulary, MedDRA (17.1)
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Double-Blind Period: Placebo Double-Blind Period: Casimersen 4 mg/kg Double-Blind Period: Casimersen 10 mg/kg Double-Blind Period: Casimersen 20 mg/kg Double-Blind Period: Casimersen 30 mg/kg Open Label Extension Period: Casimersen 30 mg/kg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   4/4 (100.00%)   5/8 (62.50%)   3/8 (37.50%)   3/8 (37.50%)   7/8 (87.50%)   12/12 (100.00%) 
Cardiac disorders             
Atrial thrombosis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Left ventricular dysfunction * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Ventricular tachycardia * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Endocrine disorders             
Delayed puberty * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Eye disorders             
Cataract * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Gastrointestinal disorders             
Vomiting * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  2/8 (25.00%)  2/8 (25.00%)  4/12 (33.33%) 
Nausea * 1  0/4 (0.00%)  1/8 (12.50%)  1/8 (12.50%)  1/8 (12.50%)  0/8 (0.00%)  3/12 (25.00%) 
Abdominal pain * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Abdominal rigidity * 1  0/4 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Dental caries * 1  0/4 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Dyspepsia * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  2/12 (16.67%) 
Gingival pain * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Abdominal pain upper * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  2/12 (16.67%) 
Constipation * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Diarrhoea * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Haemorrhoids * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
General disorders             
Device dislocation * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Pyrexia * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  2/12 (16.67%) 
Thrombosis in device * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Application site bruise * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Application site dermatitis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Application site erythema * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Catheter site bruise * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Catheter site inflammation * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Catheter site pain * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Fatigue * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Injection site bruising * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Non-cardiac chest pain * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Immune system disorders             
Seasonal allergy * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Infections and infestations             
Bronchitis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  2/12 (16.67%) 
Candida infection * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Ear infection * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Gastroenteritis viral * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Influenza * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Nasopharyngitis * 1  1/4 (25.00%)  1/8 (12.50%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  9/12 (75.00%) 
Tinea versicolour * 1  1/4 (25.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Upper respiratory tract infection * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  4/12 (33.33%) 
Hordeolum * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  2/12 (16.67%) 
Acarodermatitis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Eye infection * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Labyrinthitis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Otitis externa * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Pharyngitis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Sinusitis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Staphylococcal infection * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Tinea pedis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Injury, poisoning and procedural complications             
Procedural pain * 1  1/4 (25.00%)  0/8 (0.00%)  0/8 (0.00%)  2/8 (25.00%)  3/8 (37.50%)  4/12 (33.33%) 
Incision site pain * 1  0/4 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Infusion related reaction * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Limb injury * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Ligament sprain * 1  1/4 (25.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/12 (0.00%) 
Tibia fracture * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  2/12 (16.67%) 
Ankle fracture * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Concussion * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Fibula fracture * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Investigations             
Blood calcium decreased * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Blood potassium decreased * 1  0/4 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Neutrophil count increased * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  1/12 (8.33%) 
Urine calcium * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Blood pressure increased * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Weight increased * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Metabolism and nutrition disorders             
Iron deficiency * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Musculoskeletal and connective tissue disorders             
Neck pain * 1  0/4 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Pain in extremity * 1  1/4 (25.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  3/12 (25.00%) 
Back pain * 1  1/4 (25.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  2/12 (16.67%) 
Musculoskeletal pain * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Myalgia * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)             
Skin papilloma * 1  1/4 (25.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Nervous system disorders             
Headache * 1  0/4 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  0/8 (0.00%)  2/8 (25.00%)  4/12 (33.33%) 
Dizziness * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Drug withdrawal headache * 1  0/4 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Migraine * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Paraesthesia * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Presyncope * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Psychiatric disorders             
Anxiety * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Renal and urinary disorders             
Nephrolithiasis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Respiratory, thoracic and mediastinal disorders             
Upper respiratory tract congestion * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  1/12 (8.33%) 
Oropharyngeal pain * 1  1/4 (25.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  3/12 (25.00%) 
Cough * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  4/12 (33.33%) 
Nasal congestion * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Skin and subcutaneous tissue disorders             
Dermatitis contact * 1  2/4 (50.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  2/12 (16.67%) 
Rash * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  3/12 (25.00%) 
Acne * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Eczema * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Erythema * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Psoriasis * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Skin hyperpigmentation * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Vitiligo * 1  0/4 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Vascular disorders             
Flushing * 1  0/4 (0.00%)  1/8 (12.50%)  0/8 (0.00%)  0/8 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
1
Term from vocabulary, MedDRA (17.1)
*
Indicates events were collected by non-systematic assessment
[Not Specified]
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 most restrictive relevant agreement provides that the PI can only publish the study results with the approval of Sponsor.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Director
Organization: Sarepta Therapeutics, Inc.
Phone: 1-800-690-2003
EMail: clinicaltrials@sarepta.com
Layout table for additonal information
Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02530905    
Other Study ID Numbers: 4045-101
First Submitted: August 10, 2015
First Posted: August 21, 2015
Results First Submitted: April 23, 2021
Results First Posted: May 17, 2021
Last Update Posted: May 17, 2021