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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: April 19, 2020
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 findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia.
• 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.
ICER Releases Draft Evidence Report on Treatments for Duchenne Muscular DystrophyThe Institute for Clinical and Economic ...
Results at 96 Weeks of a Phase IIb Extension Study of the Exon-Skipping Drug Eteplirsen in Patients with Duchenne Mu...Objective: The objective of this study ...
Eteplirsen is Granted Accelerated Approval in UShttps://www.youtube.com/watch?v=Orig6mpu...
ICER Document Reviews Effectiveness of 3 Duchenne MD TherapiesThe Institute for Clinical and Economic ...
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 SkippingThis is a multicenter, open-label, dose-...
Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular...Background: Duchenne muscular dystrophy...
Sarepta Therapeutics and Clinigen launch a managed access program to treat patients with Duchenne muscular dystrophy...Sarepta Therapeutics, Inc., a U.S. bioph...