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NCT Number: NCT02286947

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

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.

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Key information

Conditions

Muscular Dystrophy, Duchenne Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

7 year–21 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Ronald Reagan UCLA Medical Center, Los Angeles, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Eteplirsen

Drug

Eteplirsen solution for IV infusion

Other names: AVI-4658, EXONDYS 51®

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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

  2. 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.

  3. 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.

  4. 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

  5. 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

Sponsors and collaborators

Lead sponsor

Sarepta Therapeutics, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2014
Primary completion
2017
Study completion
2018
First posted
Nov 10, 2014
Registry last updated
Mar 30, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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