An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

Phase 3

Withdrawn

Conditions: Duchenne disease
Duchenne muscular dystrophy
10028302

Registration Number: NL-OMON43602

Lead Sponsor: BioMarin

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Withdrawn

Sex: Not specified

Target Recruitment: 14

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

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.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>To evaluate the long-term safety and tolerability of subcutaneous or<br /><br>intravenous drisapersen in subjects with DMD correctable by drisapersen-induced<br /><br>DMD exon 51 skipping who have previously participated in an eligible study. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>• To evaluate the long-term efficacy of subcutaneous drisapersen at a dose of 6<br /><br>mg/kg/week.<br /><br>• To evaluate the long-term impact on functional outcomes of continued<br /><br>treatment with drisapersen.<br /><br>• To evaluate the long-term safety and efficacy of an intermittent dosing<br /><br>option in those subjects unable to tolerate drisapersen 6 mg/kg/week.<br /><br>• To evaluate the long-term safety and efficacy of an intravenous dosing option<br /><br>in those subjects unable to tolerate subcutaneous administration of<br /><br>drisapersen.</p><br>