Clinical trial NCT02286947: Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
Official title: An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Muscular Dystrophy, Duchenne
Keywords: DMD
exon 51
dystrophin
dystrophy
eteplirsen
Duchenne
Lead sponsor: Sarepta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2014-11-10
Enrollment: 24
Sex: MALE
Minimum age (years): 7
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events
timeFrame: From first dose of drug up to 100 weeks
Secondary outcomes:
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities
timeFrame: Baseline up to 100 weeks
measure: Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs
timeFrame: Baseline up to 100 weeks
measure: Number of Participants With at Least One Potentially Clinically Significant Abnormalities in Physical Examinations
timeFrame: Baseline up to 100 weeks
measure: Number of Participants With Abnormalities in Electrocardiograms (ECGs)
timeFrame: Baseline up to 100 weeks
measure: Number of Participants With Abnormalities in Echocardiograms (ECHO)
timeFrame: Baseline up to 100 weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Ronald Reagan UCLA Medical Center
state: California
city: Sacramento
country: United States
facility: University of California, Davis Medical Center
state: California
city: Iowa City
country: United States
facility: University of Iowa Children's Hospital
state: Iowa
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: St. Louis Children's Hospital
state: Missouri
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: Sarepta Therapeutics, Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: