Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
Overview
- Phase
- Phase 3
- Intervention
- Casimersen
- Conditions
- Duchenne Muscular Dystrophy
- Sponsor
- Sarepta Therapeutics, Inc.
- Enrollment
- 171
- Locations
- 50
- Primary Endpoint
- Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
- Status
- Terminated
- Last Updated
- 2 years ago
Overview
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).
Investigators
Eligibility Criteria
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.
Exclusion Criteria
- Not provided
Arms & Interventions
Casimersen
Patients amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.
Intervention: Casimersen
Golodirsen
Patients amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.
Intervention: Golodirsen
Outcomes
Primary Outcomes
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
Time Frame: 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.