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 invitation

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)

intervention: Eteplirsen

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03985878

last updated: December 04, 2019

start date: June 26, 2019

estimated completion: November 2026

phase of development: Phase 2

size / enrollment: 15

primary outcomes:

• 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.

inclusion criteria:
• Patient successfully completes 96 weeks of treatment in Study 4658-102.

exclusion criteria:
• 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.

sponsor: Sarepta Therapeutics, Inc.

• Belgium
  Universitair Ziekenhuis Gent
  
  
• France
  Hopital Trousseau, Bâtiment lemariey
  
  
• Italy
  Fondazione Policlinico Universitario A Gemelli
  
  
• United Kingdom
  Dubowitz Neuromuscular Centre, UCL-Institute of Child Health