An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051



Status:Recruiting
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:12 - Any
Updated:1/25/2019
Start Date:December 19, 2018
End Date:November 1, 2020
Contact:Medical information
Email:clinicaltrials@sarepta.com
Phone:+1 888 727 3782

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


Inclusion Criteria:

• Has completed Study 5051-101 (NCT03375255) and continues to meet the Safety Eligibility
Criteria of Study 5051-101 (NCT03375255).

Exclusion Criteria:

- Initiation or change of dosing (except for modifications to accommodate changes in
weight) since entering Study 5051-101 (NCT03375255) 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.
We found this trial at
4
sites
3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
Principal Investigator: Jeffrey Statland, MD
Phone: 913-945-9928
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Principal Investigator: Hoda Abdel-Hamid, MD
Phone: 412-692-5176
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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Atlanta, Georgia 30318
Principal Investigator: Han C Phan, MD
Phone: 678-883-6897
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Atlanta, GA
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Gulf Breeze, Florida 32561
Principal Investigator: Weldon Mauney, MD
Phone: 850-934-1299
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Gulf Breeze, FL
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