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Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

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.
 
ClinicalTrials.gov Identifier: NCT02286947
Recruitment Status : Completed
First Posted : November 10, 2014
Results First Posted : February 20, 2019
Last Update Posted : July 29, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE October 30, 2014
First Posted Date  ICMJE November 10, 2014
Results First Submitted Date  ICMJE January 30, 2019
Results First Posted Date  ICMJE February 20, 2019
Last Update Posted Date July 29, 2019
Actual Study Start Date  ICMJE November 2014
Actual Primary Completion Date April 21, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 30, 2019)
Number of Participants With Treatment Emergent Adverse Events [ Time Frame: From first dose of drug up to 100 weeks ]
An adverse event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. A serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; Required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug (up to 100 weeks) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.
Original Primary Outcome Measures  ICMJE
 (submitted: November 5, 2014)
Number of patients with treatment emergent adverse events. [ Time Frame: 96 weeks ]
Change History Complete list of historical versions of study NCT02286947 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 30, 2019)
  • Number of Participants With Potentially Clinically Significant Laboratory Abnormalities [ Time Frame: Baseline up to 100 weeks ]
    Laboratory parameters included hematology, clinical chemistry, urinalysis and coagulation. Data is only reported for parameters in which at least 1 participant had potentially clinically significant abnormal findings. Incr=increase; LLN=lower limit of normal; ULN=upper limit of normal; GGT=gamma glutamyl transferase
  • Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs [ Time Frame: Baseline up to 100 weeks ]
    Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Data is only reported for parameters in which at least 1 participant had potentially clinically significant abnormal vital sign findings.
  • Number of Participants With at Least One Potentially Clinically Significant Abnormalities in Physical Examinations [ Time Frame: Baseline up to 100 weeks ]
    Physical examinations, full and brief, were performed by the Investigator, a physician Sub-Investigator, or a Nurse Practitioner (if licensed in the state or province to perform physical examinations). Full physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; skin; lymph nodes; and musculoskeletal and neurological systems. Brief physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; and skin.
  • Number of Participants With Abnormalities in Electrocardiograms (ECGs) [ Time Frame: Baseline up to 100 weeks ]
    Twelve-lead ECGs and Holter ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the patient was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel using a central vendor according to prespecified criteria. The Investigator reviewed the results of the centrally read ECG report and determined if the findings were clinically significant. Data is only reported for parameters in which at least 1 participant had potentially clinically significant abnormal ECG findings. msec=milliseconds; QTcF=QT interval corrected with Fridericia's method
  • Number of Participants With Abnormalities in Echocardiograms (ECHO) [ Time Frame: Baseline up to 100 weeks ]
    Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. The ECHO was reviewed and interpreted by medically qualified personnel using a central vendor according to prespecified criteria. Ejection fraction was noted. The Investigator reviewed the results of the ECHO report and determined if the findings were clinically significant. LEVF=left ventricular ejection fraction
Original Secondary Outcome Measures  ICMJE
 (submitted: November 5, 2014)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
Official Title  ICMJE 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 participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Detailed Description

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

Participants will be evaluated for inclusion during a Screening/Baseline period of up to 4 weeks. Eligible participants 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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Muscular Dystrophy, Duchenne
Intervention  ICMJE Drug: Eteplirsen
Eteplirsen solution for IV infusion
Other Names:
  • AVI-4658
  • EXONDYS 51®
Study Arms  ICMJE Experimental: Eteplirsen 30 mg/kg
Participants will receive eteplirsen 30 mg/kg/week intravenous (IV) infusions, weekly, for up to 96 weeks.
Intervention: Drug: Eteplirsen
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 30, 2019)
24
Original Estimated Enrollment  ICMJE
 (submitted: November 5, 2014)
20
Actual Study Completion Date  ICMJE March 23, 2018
Actual Primary Completion Date April 21, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 7 Years to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02286947
Other Study ID Numbers  ICMJE 4658-204
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sarepta Therapeutics, Inc.
Study Sponsor  ICMJE Sarepta Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics, Inc.
Verification Date July 2019

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