Duchenne Muscular Dystrophy
Conditions
Keywords
Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 53, Exon 45, Duchenne
Brief summary
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
Interventions
DRUGCasimersen
Casimersen solution for IV infusion
DRUGGolodirsen
Golodirsen solution for IV infusion
Sponsors
Sarepta Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
7 Years to 23 Years
Healthy volunteers
No
Inclusion criteria
* Completed a clinical trial evaluating casimersen or golodirsen, per protocol. * Is between 7 and 23 years of age, inclusive, at enrollment. Other inclusion/
Exclusion criteria
apply.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) | Up to 33 days after the last infusion of study drug (up to approximately 149 weeks) | A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module. |
Countries
Belgium, Bulgaria, Canada, Czechia, France, Germany, Israel, Italy, Poland, Spain, Sweden, United Kingdom, United States
Participant flow
Recruitment details
Participants amenable to exon 53 or exon 45 skipping were enrolled into this study from Study 4045-101 (NCT02530905), Study 4053-101 (NCT02310906), and Study 4045-301 (NCT02500381).
Participants by arm
| Arm | Count |
|---|---|
| Golodirsen Participants amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen received open-label golodirsen IV infusions, weekly, at 30 mg/kg for up to 144 weeks. | 74 |
| Casimersen Participants amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen received open-label casimersen IV infusions, weekly, at 30 mg/kg for up to 144 weeks. | 97 |
| Total | 171 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Death | 1 | 0 |
| Overall Study | Study Terminated by Sponsor | 41 | 65 |
| Overall Study | Withdrawal by Subject | 6 | 5 |
Baseline characteristics
| Characteristic | Total | Casimersen | Golodirsen |
|---|---|---|---|
| Age, Continuous | 12.0 years STANDARD_DEVIATION 2.4 | 12.2 years STANDARD_DEVIATION 2.57 | 11.7 years STANDARD_DEVIATION 2.14 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 16 Participants | 6 Participants | 10 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 143 Participants | 89 Participants | 54 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 12 Participants | 2 Participants | 10 Participants |
| Race/Ethnicity, Customized American Indian or Alaska Native | 1 participants | 0 participants | 1 participants |
| Race/Ethnicity, Customized Asian | 11 participants | 7 participants | 4 participants |
| Race/Ethnicity, Customized Black | 1 participants | 0 participants | 1 participants |
| Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander | 1 participants | 1 participants | 0 participants |
| Race/Ethnicity, Customized Other | 6 participants | 3 participants | 3 participants |
| Race/Ethnicity, Customized White | 151 participants | 86 participants | 65 participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 171 Participants | 97 Participants | 74 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 74 | 0 / 97 |
| other Total, other adverse events | 63 / 74 | 93 / 97 |
| serious Total, serious adverse events | 12 / 74 | 22 / 97 |
Outcome results
Primary
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.
Time frame: Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
Population: Safety Analysis Set: All enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Golodirsen | Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) | 12 Participants |
| Casimersen | Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) | 22 Participants |