Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
|ClinicalTrials.gov Identifier: NCT02286947|
Recruitment Status : Completed
First Posted : November 10, 2014
Results First Posted : February 20, 2019
Last Update Posted : May 7, 2019
|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophy, Duchenne||Drug: Eteplirsen||Phase 2|
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 :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy|
|Actual Study Start Date :||November 2014|
|Actual Primary Completion Date :||April 21, 2017|
|Actual Study Completion Date :||March 23, 2018|
Experimental: Eteplirsen 30 mg/kg
Participants will receive eteplirsen 30 mg/kg/week intravenous (IV) infusions, weekly, for up to 96 weeks.
Eteplirsen solution for IV infusion
- 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.
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02286947
|United States, California|
|Ronald Reagan UCLA Medical Center|
|Los Angeles, California, United States, 90095|
|University of California, Davis Medical Center|
|Sacramento, California, United States, 95817|
|United States, Iowa|
|University of Iowa Children's Hospital|
|Iowa City, Iowa, United States, 52242|
|United States, Maryland|
|Kennedy Krieger Institute|
|Baltimore, Maryland, United States, 21205|
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|United States, Missouri|
|St. Louis Children's Hospital|
|Saint Louis, Missouri, United States, 63110|
|United States, New York|
|University of Rochester Medical Center|
|Rochester, New York, United States, 14642|
|United States, Ohio|
|Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|
|United States, Washington|
|Seattle Children's Hospital|
|Seattle, Washington, United States, 98105|
|Study Director:||Medical Director||Sarepta Therapeutics, Inc.|