Clinical Trial: Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy

Brief Summary: The primary objective of this study is to explore safety and tolerability of eteplirsen in patients with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The exploratory objectives are to evaluate the effect of eteplirsen on pulmonary function tests (PFTs) and other functional clinical measures.

Detailed Summary:

This is an open-label, multi-center study to explore the safety and tolerability of eteplirsen injection in patients with advanced stage DMD with confirmed genetic mutations amenable to treatment by exon 51 skipping.

Patients will be evaluated for inclusion during a Screening/Baseline period of up to 4 weeks. Eligible patients will receive once weekly intravenous (IV) infusions of 30 mg/kg eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks).

Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. Exploratory efficacy assessments, including PFTs, upper extremity testing, and other measurements of functional status, will occur at functional assessment visits every 12 weeks over the first year of treatment and approximately every 24 weeks over the second year of treatment.


Sponsor: Sarepta Therapeutics

Current Primary Outcome: Number of patients with treatment emergent adverse events. [ Time Frame: up to 144 weeks ]

Original Primary Outcome: Number of patients with treatment emergent adverse events. [ Time Frame: 96 weeks ]

Current Secondary Outcome:

  • Number of patients with clinical laboratory abnormalities [ Time Frame: up to 144 weeks ]
  • Number of patients with abnormalities in vital signs and ECG [ Time Frame: up to 144 weeks ]
  • Mean Change from baseline in maximum inspiratory pressure (MIP) % predicted [ Time Frame: Baseline to Week 96 ]
  • Mean Change from baseline in maximum expiratory pressure (MEP) % predicted [ Time Frame: Baseline to Week 96 ]


Original Secondary Outcome:

  • Number of patients with clinical laboratory abnormalities [ Time Frame: 96 weeks ]
  • Number of patients with abnormalities in vital signs and ECG [ Time Frame: 96 weeks ]
  • Mean Change from baseline in maximum inspiratory pressure (MIP) % predicted [ Time Frame: Baseline to Week 96 ]
  • Mean Change from baseline in maximum expiratory pressure (MEP) % predicted [ Time Frame: Baseline to Week 96 ]


Information By: Sarepta Therapeutics

Dates:
Date Received: October 30, 2014
Date Started: November 2014
Date Completion: September 2017
Last Updated: January 10, 2017
Last Verified: January 2017