Duchenne disease Duchenne muscular dystrophy
Conditions
Interventions
The primary dosing arm is drisapersen 6 mg/kg as subcutaneous (SC) injection(s)
once a week. All subjects starting with subcutaneous injections will receive a
loading dose of twice weekly 6mg/kg dri
Sponsors
BioMarin
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Any subject who has been previously treated with drisapersen or eteplirsen 2. Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on glucocorticoids for the duration of this study. Changes to or cessation of glucocorticoids will be at the discretion of the investigator conducting this study in consultation with the subject/parent and Medical Monitor.
Exclusion criteria
Exclusion criteria: 1.Use of anticoagulants, anti-thrombotics or antiplatelet agents 2.Use of any investigatoional product within 6 months prior to the start of study (except for eteplirsen) 3.History of significant medical disorder which may confound the interpretation of safety data (e.g. current or history of renal or liver disease/impairment, history of inflammatory illness) 4.Symptomatic cardiomyopathy. 5.A platelet count under the lower limit of normal (LLN) at start of this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the long-term safety and tolerability of subcutaneous or intravenous drisapersen in subjects with DMD correctable by drisapersen-induced DMD exon 51 skipping who have previously participated in an eligible study. | — |
Secondary
| Measure | Time frame |
|---|---|
| • To evaluate the long-term efficacy of subcutaneous drisapersen at a dose of 6 mg/kg/week. • To evaluate the long-term impact on functional outcomes of continued treatment with drisapersen. • To evaluate the long-term safety and efficacy of an intermittent dosing option in those subjects unable to tolerate drisapersen 6 mg/kg/week. • To evaluate the long-term safety and efficacy of an intravenous dosing option in those subjects unable to tolerate subcutaneous administration of drisapersen. | — |
Countries
Netherlands
Outcome results
None listed