Clinical trial NCT02420379: Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
Official title: An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy (DMD)
Keywords: DMD
Duchenne muscular dystrophy
eteplirsen
Dystrophin
exon 51
Lead sponsor: Sarepta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2015-04-17
Enrollment: 33
Sex: MALE
Minimum age (years): 4
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Detailed description: Safety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients.

Clinical efficacy, including functional tests and MRI, will be assessed at regularly scheduled study visits. Patients will undergo one baseline and one follow-up muscle biopsy.

Population and serial PK will be collected.

Eligibility criteria: Inclusion Criteria:

* Male 4-6 years of age.
* Diagnosis of DMD, genotypically confirmed.
* Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks.
* Intact right and left biceps muscles or two alternative upper arm muscle groups.
* Parent that is willing to provide consent and comply with study procedures.

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 or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months.
* Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities.
* Presence of other clinically significant illness.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation
timeFrame: Baseline up to 100 weeks
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs
timeFrame: Baseline up to 100 weeks
measure: Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs
timeFrame: Baseline up to 100 weeks
measure: Number of Participants With at Least One Abnormal Physical Examination Finding
timeFrame: Baseline up to 100 weeks
measure: Number of Participants With Abnormalities in Electrocardiograms (ECGs) Reported as TEAEs
timeFrame: Baseline up to 96 weeks
measure: Number of Participants With Abnormalities in Echocardiograms (ECHO) Reported as TEAEs
timeFrame: Baseline up to 96 weeks
Secondary outcomes:
measure: Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96
timeFrame: Baseline, Week 48 and 96
measure: Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96
timeFrame: Baseline, Week 48 and 96
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Neuromuscular Research Center of Arizona
state: Arizona
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: Stanford
country: United States
facility: Stanford University Medical Center
state: California
city: Gainesville
country: United States
facility: University of Florida, Shands Hospital
state: Florida
city: Atlanta
country: United States
facility: Rare Disease Research Center
state: Georgia
city: Atlanta
country: United States
facility: Children's Hospital of Atlanta
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa Children's Hospital
state: Iowa
city: Saint Louis
country: United States
facility: St. Louis Children's Hospital
state: Missouri
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Shriners Hospital for Children
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
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: