Clinical trial NCT01540409: Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
Official title: Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy (DMD)
Keywords: DMD, Duchenne, Eteplirsen, dystrophy, dystrophin, exon 51
Lead sponsor: Sarepta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2012-02-28
Enrollment: 12
Sex: MALE
Minimum age (years): 7
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28 week eteplirsen study: Study 4658-us-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.

Detailed description: This is an open label, multiple dose extension study to assess the ongoing efficacy, safety, and tolerability of weekly intravenous (IV) infusions of eteplirsen in DMD subjects who have successfully completed Study 4658-us 201.

Subjects will have the opportunity to enroll in this study during the last visit of Study 4658-us-201 (Week 28). Eligible subjects will receive once weekly IV infusions of eteplirsen (50 or 30 mg/kg) for an additional 212 weeks. Subjects will receive the same dose of eteplirsen they received in Study 4658-us-201. Subjects will thereafter continue to receive once weekly IV infusions of eteplirsen for up to an additional 72 week period (through week 284). If commercial eteplirsen becomes available during this additional 72 week period, participation in the study will be discontinued as subjects transition to commercial eteplirsen.

Safety, efficacy, pharmacokinetic (PK), and biomarker assessments will be performed at scheduled visits; adverse events (AEs) and concomitant medications and therapies will be continuously monitored.

If review of data from this open label study suggests that continued treatment with eteplirsen is warranted, this study may be extended by protocol amendment or subjects who successfully complete this study may have the opportunity to participate in a separate follow on, open label eteplirsen study.

Eligibility criteria: Inclusion Criteria:

A subject must meet all of the following criteria to be eligible for this study.

1. The subject and/or their parent/legal guardian are willing and able to provide signed informed consent.
2. The subject has successfully completed 28 weeks of treatment in Study 4658-US-201.
3. The subject has a parent(s) or legal guardian(s) who is able to understand and comply with all of the study procedure requirements.

Exclusion Criteria:

A subject who meets any of the following criteria will be excluded from this study.

1. The subject has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the subject or make it unlikely that the course of treatment or follow-up would be completed or impair the assessment of study results.

Primary outcomes:
measure: Change From Baseline in the 6 Minute Walk Test (6MWT) at Week 240
timeFrame: Parent Baseline and Week 240
measure: Change From Baseline in the Percentage of Dystrophin Positive Fibers (PDPF) at Week 48
timeFrame: Parent Baseline and Week 48
Secondary outcomes:
Other outcomes:
Locations:
city: Long Beach
country: United States
facility: Miller Children's Hospital
state: California
city: Gainesville
country: United States
facility: University of Florida Clinical Research Center
state: Florida
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University Medical School
state: Missouri
city: Liverpool
country: United States
facility: Summerwood Pediatrics/Infusacare Medical Services
state: New York
city: Charlotte
country: United States
facility: Levine Children's Hospital
state: North Carolina
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Norfolk
country: United States
facility: Children's Specialty Group, Pediatric Neurology
state: Virginia
city: Osceola
country: United States
facility: Osceola Medical Center
state: Wisconsin
Study officials:
affiliation: Sarepta Therapeutics, Inc.
name: Medical Director
role: STUDY_DIRECTOR
References:
citation: Mendell JR, Goemans N, Lowes LP, Alfano LN, Berry K, Shao J, Kaye EM, Mercuri E; Eteplirsen Study Group and Telethon Foundation DMD Italian Network. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann Neurol. 2016 Feb;79(2):257-71. doi: 10.1002/ana.24555. Epub 2016 Jan 8.