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Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02286947
First Posted: November 10, 2014
Last Update Posted: October 4, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Sarepta Therapeutics
October 30, 2014
November 10, 2014
October 4, 2017
November 2014
April 2017   (Final data collection date for primary outcome measure)
Number of patients with treatment emergent adverse events. [ Time Frame: up to 144 weeks ]
Number of patients with treatment emergent adverse events. [ Time Frame: 96 weeks ]
Complete list of historical versions of study NCT02286947 on ClinicalTrials.gov Archive Site
  • 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 tin 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 ]
  • 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 ]
Not Provided
Not Provided
 
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy
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.

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.

Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Muscular Dystrophy, Duchenne
Drug: eteplirsen
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Other Names:
  • AVI-4658
  • EXONDYS 51®
Experimental: Advanced Stage Intervention Group
Approximately 20 patients will receive weekly infusions of eteplirsen 30 mg/kg.
Intervention: Drug: eteplirsen
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
20
December 2017
April 2017   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male 7 - 21 years of age
  • Diagnosis of DMD with a mutation that is amenable to exon 51 skipping, confirmed by a genetic report
  • Stable dose of oral corticosteroids for at least 24 weeks or has not received corticosteroids for at least 24 weeks
  • Non-ambulatory, or incapable of walking ≥300 meters on the 6-Minute Walk Test (6MWT).
  • Score of ≤4 on the Brooke Score for Arms and Shoulders.
  • Stable cardiac and pulmonary function
  • Use of contraceptives for sexually active males throughout the study
  • Willing to provide consent and comply with the study

Exclusion Criteria:

  • Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
  • Previous treatment with SMT C1100/BMN 195 at any time.
  • Previous treatment with drisapersen (PRO051) within the last 6 months.
  • Participation in any other DMD interventional clinical study within 12 weeks
  • Major change in physiotherapy regimen within the past 3 months
  • Major surgery within 3 months
  • Presence of other clinically significant illness
  • Use of an aminoglycoside antibiotic within 12 weeks or the need for this antibiotic or statin during study
  • Forced vital capacity % predicted [FVC % predicted] <40%, or requiring daytime ventilation.
  • Require antiarrhythmic and/or antidiuretic therapy for heart failure.
  • Have a left ventricular ejection fraction (LVEF) of <40%.
  • Prior or ongoing medical condition that could adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
Sexes Eligible for Study: Male
7 Years to 21 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT02286947
4658-204
No
Not Provided
Not Provided
Sarepta Therapeutics
Sarepta Therapeutics
Not Provided
Study Director: Chris Mix, MD Sarepta Therapeutics
Study Director: Catherine Stehman-Breen, MD, MS Sarepta Therapeutics
Sarepta Therapeutics
September 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP