Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
Study ID: STU-2019-0588
Patients with Duchenne muscular dystrophy (DMD) who are amenable to treatment by skipping exons 45 or 53 and who have been participating in a clinical trial evaluating casimersen (SRP-4045) or golodirsen (SRP-4053) will be eligible to transfer into this long-term extension (LTe) study. Patients can enroll in the LTe only after completing the original study, per protocol. every effort will be made to ensure that the patient will not experience any interruption in dosing of casimersen or golodirsen during the transition from the original study to the LTe. Patients who are enrolled in a treatment group receiving either casimersen or golodirsen will continue to receive 30 mg/kg of either casimersen or golodirsen once-weekly by intravenous (iV) infusion. Patients who are enrolled in a placebo group within their original study will initiate treatment with 30 mg/kg of either casimersen or golodirsen, depending upon their genotype, once-weekly by iV infusion starting at Week 1 [See protocol for complete text]
Primary objective * To evaluate the safety and tolerability of long-term treatment with 30 mg/kg of casimersen or golodirsen. exploratory objectives * To evaluate changes in physical function with long-term treatment with 30 mg/kg of casimersen or golodirsen. * To evaluate changes in pulmonary function with long-term treatment with 30 mg/kg of casimersen or golodirsen. * To evaluate immunogenicity of long-term treatment with 30 mg/kg casimersen or golodirsen.