Trial Outcomes & Findings for Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients (NCT NCT02530905)

NCT ID: NCT02530905

Last Updated: 2021-05-17

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 12 participants
• Primary outcome timeframe: Baseline up to Week 148
• Results posted on: 2021-05-17

Participant Flow

The study was conducted at 3 sites in United States.

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.

Participant milestones

Measure	Double-Blind Period: Placebo 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.	Double-Blind Period: Casimersen 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.	Open Label Extension Period: Casimersen 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.
Double-Blind Period (12 Weeks) STARTED	4	8	0
Double-Blind Period (12 Weeks) Received 4 mg/kg	0	8	0
Double-Blind Period (12 Weeks) Received 10 mg/kg	0	8	0
Double-Blind Period (12 Weeks) Received 20 mg/kg	0	8	0
Double-Blind Period (12 Weeks) Received 30 mg/kg	0	8	0
Double-Blind Period (12 Weeks) COMPLETED	4	8	0
Double-Blind Period (12 Weeks) NOT COMPLETED	0	0	0
Open Label Extension Period (132 Weeks) STARTED	0	0	12
Open Label Extension Period (132 Weeks) COMPLETED	0	0	11
Open Label Extension Period (132 Weeks) NOT COMPLETED	0	0	1

Reasons for withdrawal

Measure	Double-Blind Period: Placebo 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.	Double-Blind Period: Casimersen 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.	Open Label Extension Period: Casimersen 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.
Open Label Extension Period (132 Weeks) Withdrawal by Subject	0	0	1

Baseline Characteristics

Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

Baseline characteristics by cohort

Measure	Double-Blind Period: Placebo n=4 Participants 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.	Double-Blind Period: Casimersen n=8 Participants 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 n=12 Participants Total of all reporting groups
Age, Continuous	12.0 Years STANDARD_DEVIATION 2.16 • n=5 Participants	14.4 Years STANDARD_DEVIATION 3.29 • n=7 Participants	13.6 Years STANDARD_DEVIATION 3.09 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	8 Participants n=7 Participants	12 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants	7 Participants n=7 Participants	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants	6 Participants n=7 Participants	10 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline up to Week 148

Population: 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.

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.

Outcome measures

Measure	Double-Blind Period: Placebo n=4 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=8 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg n=12 Participants 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.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	4 Participants	5 Participants	3 Participants	3 Participants	7 Participants	12 Participants

PRIMARY outcome

Timeframe: Baseline up to Week 148

Population: 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.

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.

Outcome measures

Measure	Double-Blind Period: Placebo n=4 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=8 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg n=12 Participants 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.
Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs Serum chemistry: Hepatic chemistry	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs Serum chemistry: Renal chemistry	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs Hematolgy: Leukocytes	0 Participants	0 Participants	0 Participants	0 Participants	2 Participants	4 Participants
Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs Hematolgy: Neutrophils	0 Participants	0 Participants	0 Participants	0 Participants	4 Participants	6 Participants
Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs Coagulation: Platelet count	0 Participants	0 Participants	0 Participants	0 Participants	2 Participants	2 Participants
Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs Urinalysis	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants

PRIMARY outcome

Timeframe: Baseline up to Week 148

Population: 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.

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.

Outcome measures

Measure	Double-Blind Period: Placebo n=4 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=8 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg n=12 Participants 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.
Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants	1 Participants

PRIMARY outcome

Timeframe: Baseline up to Week 148

Population: 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.

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.

Outcome measures

Measure	Double-Blind Period: Placebo n=4 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=8 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg n=12 Participants 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.
Number of Participants With Potentially Clinically Significant Abnormalities in Electrocardiogram (ECG) Reported as TEAEs	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants

PRIMARY outcome

Timeframe: Baseline up to Week 148

Population: 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.

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.

Outcome measures

Measure	Double-Blind Period: Placebo n=4 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=8 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg n=12 Participants 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.
Number of Participants With Potentially Clinically Significant Abnormalities in Echocardiograms (ECHO)	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 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

Population: 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.

Maximum Concentration (Cmax) of casimersen in plasma was evaluated.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=12 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Maximum Plasma Concentration (Cmax) of Casimersen	13700 Nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 25.0	39400 Nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 11.7	64400 Nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 27.4	119000 Nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 33.6	115000 Nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 31.5	—

SECONDARY outcome

Timeframe: 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

Population: 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.

Time to reach maximum plasma concentration (Tmax) of casimersen was evaluated.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=12 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Time to Reach Maximum Plasma Concentration (Tmax) of Casimersen	1.11 Hour Interval 0.92 to 1.22	1.03 Hour Interval 0.93 to 1.17	1.03 Hour Interval 0.83 to 1.22	0.94 Hour Interval 0.83 to 1.18	0.95 Hour Interval 0.78 to 1.08	—

SECONDARY outcome

Timeframe: 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

Population: 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.

Area under the concentration-time curve from time zero to the last quantifiable concentration of casimersen in plasma were evaluated.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=12 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Area Under Concentration-time Curve From Time of Dosing to the Last Measurable Concentration (AUClast) of Casimersen in Plasma	23100 Hour*nanogram per milliliter (hr*ng/mL) Geometric Coefficient of Variation 29.5	59500 Hour*nanogram per milliliter (hr*ng/mL) Geometric Coefficient of Variation 16.2	101000 Hour*nanogram per milliliter (hr*ng/mL) Geometric Coefficient of Variation 17.7	188000 Hour*nanogram per milliliter (hr*ng/mL) Geometric Coefficient of Variation 27.4	182000 Hour*nanogram per milliliter (hr*ng/mL) Geometric Coefficient of Variation 33.8	—

SECONDARY outcome

Timeframe: 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

Population: 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.

Area under concentration-time curve from time zero pre-dose to twenty-four hours post-dos of casimersen in plasma were evaluated.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=12 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Area Under Concentration-Time Curve From Time Zero Pre-dose to Twenty-Four Hours Post-dose (AUC0-24) of Casimersen in Plasma	23200 h*ng/mL Geometric Coefficient of Variation 29.4	59500 h*ng/mL Geometric Coefficient of Variation 16.1	101000 h*ng/mL Geometric Coefficient of Variation 17.7	188000 h*ng/mL Geometric Coefficient of Variation 27.4	182000 h*ng/mL Geometric Coefficient of Variation 33.8	—

SECONDARY outcome

Timeframe: 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

Population: 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.

Area under the concentration-time curve from time zero extrapolated to the infinity of casimersen in plasma was evaluated.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=7 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=12 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Area Under the Concentration-Time Curve From Time Zero Extrapolated to the Infinity (AUCinf) of Casimersen in Plasma	23300 h*ng/mL Geometric Coefficient of Variation 29.5	58300 h*ng/mL Geometric Coefficient of Variation 16.0	101000 h*ng/mL Geometric Coefficient of Variation 17.7	189000 h*ng/mL Geometric Coefficient of Variation 27.5	182000 h*ng/mL Geometric Coefficient of Variation 33.9	—

SECONDARY outcome

Timeframe: 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

Population: 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.

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.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=7 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=12 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Apparent Volume of Distribution at Steady State (Vss) of Casimersen	0.369 Liter per kilogram (L/kg) Geometric Coefficient of Variation 24.4	0.343 Liter per kilogram (L/kg) Geometric Coefficient of Variation 12.5	0.407 Liter per kilogram (L/kg) Geometric Coefficient of Variation 23.4	0.319 Liter per kilogram (L/kg) Geometric Coefficient of Variation 31.4	0.367 Liter per kilogram (L/kg) Geometric Coefficient of Variation 28.9	—

SECONDARY outcome

Timeframe: 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

Population: 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.

T1/2 is the time measured for the plasma concentration of drug to decrease by one half. T1/2 of casimersen was evaluated.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=7 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=12 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Elimination Half-life (T1/2) of Casimersen	2.92 Hour Standard Deviation 0.985	3.29 Hour Standard Deviation 0.640	3.71 Hour Standard Deviation 0.616	3.82 Hour Standard Deviation 0.741	3.45 Hour Standard Deviation 0.359	—

SECONDARY outcome

Timeframe: 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

Population: 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.

Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood. CL of casimersen was evaluated.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=7 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=12 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Total Clearance (CL) of Casimersen	0.177 Liters per hour per kilogram (L/h/kg) Geometric Coefficient of Variation 29.1	0.181 Liters per hour per kilogram (L/h/kg) Geometric Coefficient of Variation 15.9	0.205 Liters per hour per kilogram (L/h/kg) Geometric Coefficient of Variation 18.5	0.163 Liters per hour per kilogram (L/h/kg) Geometric Coefficient of Variation 27.7	0.180 Liters per hour per kilogram (L/h/kg) Geometric Coefficient of Variation 35.0	—

SECONDARY outcome

Timeframe: 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

Population: 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.

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.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=7 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg n=12 Participants 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Mean Residence Time Extrapolated to Infinity (MRTinf) of Casimersen	2.08 Hour Geometric Coefficient of Variation 13.5	1.89 Hour Geometric Coefficient of Variation 16.1	1.98 Hour Geometric Coefficient of Variation 12.7	1.96 Hour Geometric Coefficient of Variation 16.6	2.04 Hour Geometric Coefficient of Variation 10.3	—

SECONDARY outcome

Timeframe: 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

Population: 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.

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.

Outcome measures

Measure	Double-Blind Period: Placebo n=8 Participants 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 10 mg/kg n=7 Participants 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 Participants 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.	Double-Blind Period: Casimersen 30 mg/kg 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.	Open Label Extension Period: Casimersen 30 mg/kg 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.
Double-Blind Period: Renal Clearance (CLR) of Casimersen	0.137 L/h/kg Geometric Coefficient of Variation 25.8	0.162 L/h/kg Geometric Coefficient of Variation 22.6	0.209 L/h/kg Geometric Coefficient of Variation 29.0	0.177 L/h/kg Geometric Coefficient of Variation 34.2	—	—

Adverse Events

Double-Blind Period: Placebo

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Double-Blind Period: Casimersen 4 mg/kg

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Double-Blind Period: Casimersen 10 mg/kg

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Double-Blind Period: Casimersen 20 mg/kg

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Double-Blind Period: Casimersen 30 mg/kg

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Open Label Extension Period: Casimersen 30 mg/kg

Serious events: 3 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Measure	Double-Blind Period: Placebo n=4 participants at risk 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 participants at risk 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 participants at risk 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 participants at risk 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.	Double-Blind Period: Casimersen 30 mg/kg n=8 participants at risk 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.	Open Label Extension Period: Casimersen 30 mg/kg n=12 participants at risk 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.
Infections and infestations Septic embolus	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Vascular disorders vena cava thrombosis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations bacteraemia	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Femur fracture	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Tibia fracture	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148

Other adverse events

Measure	Double-Blind Period: Placebo n=4 participants at risk 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.	Double-Blind Period: Casimersen 4 mg/kg n=8 participants at risk 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.	Double-Blind Period: Casimersen 10 mg/kg n=8 participants at risk 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.	Double-Blind Period: Casimersen 20 mg/kg n=8 participants at risk 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.	Double-Blind Period: Casimersen 30 mg/kg n=8 participants at risk 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.	Open Label Extension Period: Casimersen 30 mg/kg n=12 participants at risk 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.
Gastrointestinal disorders Constipation	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Diarrhoea	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Haemorrhoids	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Skin and subcutaneous tissue disorders Rash	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	25.0% 3/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Abdominal pain upper	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	16.7% 2/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Vomiting	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	25.0% 2/8 • From start of study drug administration up to Week 148	25.0% 2/8 • From start of study drug administration up to Week 148	33.3% 4/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Nausea	0.00% 0/4 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	25.0% 3/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Abdominal pain	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Abdominal rigidity	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Dental caries	0.00% 0/4 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Dyspepsia	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	16.7% 2/12 • From start of study drug administration up to Week 148
Gastrointestinal disorders Gingival pain	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Bronchitis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	16.7% 2/12 • From start of study drug administration up to Week 148
Infections and infestations Candida infection	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Ear infection	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Gastroenteritis viral	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Influenza	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Nasopharyngitis	25.0% 1/4 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	75.0% 9/12 • From start of study drug administration up to Week 148
Infections and infestations Tinea versicolour	25.0% 1/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Procedural pain	25.0% 1/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	25.0% 2/8 • From start of study drug administration up to Week 148	37.5% 3/8 • From start of study drug administration up to Week 148	33.3% 4/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Incision site pain	0.00% 0/4 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Infusion related reaction	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Limb injury	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Ligament sprain	25.0% 1/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/12 • From start of study drug administration up to Week 148
Investigations Blood calcium decreased	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Investigations Blood potassium decreased	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Investigations Neutrophil count increased	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Investigations Urine calcium	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Nervous system disorders Headache	0.00% 0/4 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	25.0% 2/8 • From start of study drug administration up to Week 148	33.3% 4/12 • From start of study drug administration up to Week 148
Nervous system disorders Dizziness	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Nervous system disorders Drug withdrawal headache	0.00% 0/4 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Nervous system disorders Migraine	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Device dislocation	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Pyrexia	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	16.7% 2/12 • From start of study drug administration up to Week 148
General disorders Thrombosis in device	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Musculoskeletal and connective tissue disorders Neck pain	0.00% 0/4 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Musculoskeletal and connective tissue disorders Pain in extremity	25.0% 1/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	25.0% 3/12 • From start of study drug administration up to Week 148
Musculoskeletal and connective tissue disorders Back pain	25.0% 1/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	16.7% 2/12 • From start of study drug administration up to Week 148
Metabolism and nutrition disorders Iron deficiency	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Skin papilloma	25.0% 1/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Respiratory, thoracic and mediastinal disorders Upper respiratory tract congestion	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	25.0% 1/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	25.0% 3/12 • From start of study drug administration up to Week 148
Vascular disorders Flushing	0.00% 0/4 • From start of study drug administration up to Week 148	12.5% 1/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Skin and subcutaneous tissue disorders Dermatitis contact	50.0% 2/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	16.7% 2/12 • From start of study drug administration up to Week 148
Infections and infestations Upper respiratory tract infection	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	33.3% 4/12 • From start of study drug administration up to Week 148
Infections and infestations Hordeolum	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	16.7% 2/12 • From start of study drug administration up to Week 148
Infections and infestations Acarodermatitis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Eye infection	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Labyrinthitis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Otitis externa	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Pharyngitis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Sinusitis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Staphylococcal infection	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Infections and infestations Tinea pedis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Skin and subcutaneous tissue disorders Acne	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Skin and subcutaneous tissue disorders Eczema	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Skin and subcutaneous tissue disorders Erythema	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Skin and subcutaneous tissue disorders Psoriasis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Skin and subcutaneous tissue disorders Skin hyperpigmentation	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Skin and subcutaneous tissue disorders Vitiligo	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Tibia fracture	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	16.7% 2/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Ankle fracture	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Concussion	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Injury, poisoning and procedural complications Fibula fracture	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Musculoskeletal and connective tissue disorders Musculoskeletal pain	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Application site bruise	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Application site dermatitis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Application site erythema	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Catheter site bruise	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Catheter site inflammation	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Catheter site pain	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Fatigue	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Injection site bruising	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
General disorders Non-cardiac chest pain	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Investigations Blood pressure increased	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Investigations Weight increased	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Nervous system disorders Paraesthesia	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Nervous system disorders Presyncope	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	33.3% 4/12 • From start of study drug administration up to Week 148
Respiratory, thoracic and mediastinal disorders Nasal congestion	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Cardiac disorders Atrial thrombosis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Cardiac disorders Left ventricular dysfunction	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Cardiac disorders Ventricular tachycardia	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Endocrine disorders Delayed puberty	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Eye disorders Cataract	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Immune system disorders Seasonal allergy	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Psychiatric disorders Anxiety	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148
Renal and urinary disorders Nephrolithiasis	0.00% 0/4 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	0.00% 0/8 • From start of study drug administration up to Week 148	8.3% 1/12 • From start of study drug administration up to Week 148

Additional Information

Medical Director

Sarepta Therapeutics, Inc.

Phone: 1-800-690-2003

Email: clinicaltrials@sarepta.com

Results disclosure agreements

• Principal investigator is a sponsor employee The most restrictive relevant agreement provides that the PI can only publish the study results with the approval of Sponsor.
• Publication restrictions are in place

Restriction type: OTHER