Clinical trial NCT03675126: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
Official title: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Muscular Dystrophy, Duchenne
Keywords: Duchenne muscular dystrophy
Exon Skipping
DMD
Exon 51
Ambulatory
Pediatric
Nonambulatory
Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
Duchenne
Lead sponsor: Sarepta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2018-09-18
Enrollment: 15
Sex: MALE
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.


Eligibility criteria: Inclusion Criteria:

• Has completed a study of SRP-5051 and continues to meet the Eligibility Criteria of Study 5051-102.

Exclusion Criteria:

* Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) since completing a study administering SRP-5051 and while participating in this study for any of the following: angiotensin converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), beta-blockers, potassium and steroids\*.
* Requires antiarrhythmic and/or diuretic therapy for heart failure.
* Use of any herbal medication/supplement containing aristolochic acid.
* Treatment with any experimental therapy since entering original study or any experimental gene therapy for the treatment of DMD at any time.
* Participation in an interventional clinical trial since completing original study.

Other inclusion/exclusion criteria apply.

\* The dose of steroids must remain constant except for modifications to accommodate changes in weight.

Primary outcomes:
measure: Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)
timeFrame: Up to approximately 135 weeks
Secondary outcomes:
measure: Plasma Concentration of SRP-5051
timeFrame: Day 1, Day 84, every 84 days after Day 84 (up to a maximum of approximately 135 weeks) (pre-dose, immediately prior to end of infusion, up to 4-6 hours post-dose)
Other outcomes:
Locations:
city: Hartford
country: United States
facility: Connecticut Children's Medical Center
state: Connecticut
city: Gulf Breeze
country: United States
facility: NW FL Clinical Research Group, LLC
state: Florida
city: Atlanta
country: United States
facility: Center for Integrative Rare Disease Research (CIRDR)
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
city: Dallas
country: United States
facility: Children's Medical Center Dallas
state: Texas
city: London
country: Canada
facility: London Health Sciences Centre
state: Ontario
Study officials:
affiliation: Sarepta Therapeutics, Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: