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A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy
study id #: NCT03985878
condition: Duchenne Muscular Dystrophy
status: enrolling by invitationpurpose:
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male DMD patients who have successfully completed the 96-week eteplirsen study: Study 4658-102 (NCT03218995)
mechanism of action: Exon-skipping to promote dystrophin production
last updated: December 11, 2019
start date: June 26, 2019
estimated completion: November 2026
phase of development: Phase 2
size / enrollment: 15
- Incidence of Adverse Events (AEs) [ Time Frame: Up to 288 weeks ]
- Incidence of Death Due to Adverse Events [ Time Frame: Up to 288 weeks ]
- Incidence of Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 288 weeks ]
AESIs will be defined as any AE that is of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor is appropriate. AESIs will include infusion-related reactions, hypersensitivity, and renal events.
• Eligible Sexes: male
• Patient successfully completes 96 weeks of treatment in Study 4658-102.
• Patient has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the patient, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.
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