An Extension of Study SRP-5051 for Duchenne Muscular Dystrophy
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Age: 12 years or older
Gender:
Male
Healthy Volunteers: No
Keywords: Duchenne Muscular Dystrophy, DMD
Type: Drug study, Phase [/1/,/ /2/]
Target:
5 Participants
Description
This research study is for participants who completed Study 5051-101. It offers these participants the opportunity to receive additional doses of experimental SRP-5051 (the study drug). Participants' blood and urine samples will be collected to determine if repeat monthly doses of the study drug are safe. We will use blood tests to determine how the body processes repeat doses of the study drug (pharmacokinetics). This research study will also collect information about upper limb, lower limb, and lung function.

SRP-5051 is not approved by the U.S. Food and Drug Administration (FDA), or any other similar governmental agency abroad. This means that SRP-5051 is investigational (experimental) and can only be used in research studies like this one.

DMD is caused by a mutation (a change) in the gene (the part of cells that give the body instructions on how to grow and work) that makes dystrophin (a protein). Dystrophin is important for protecting muscles from stress and damage during activity. If a person has DMD, his body is not able to make enough working dystrophin to protect his muscles.

Some DMD participants are missing certain parts (exons) of the dystrophin gene. Their bodies can’t make full-length dystrophin protein. For these participants with deletion mutations, “skipping” over the missing area may allow the body to produce a shortened, but still working, form of the dystrophin protein. SRP-5051 is designed to skip exon 51.

This study will test SRP-5051 in males with DMD who have deletions that may be treated by skipping exon 51.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

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.
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study doctor will discuss the risks associated with taking part in this research study.
Compensation
You will not be paid to take part in this research study.
Resources
Schedule
Study duration and period
You will be in this study for about 60 weeks (a little over a year). You will need to visit the research site at least monthly for scheduled study visits.
Recruitment period
From Nov. 5, 2018
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Contact
Elizabeth Guy
Research Topic
Conditions:
  • Muscular Dystrophy
  • Duchenne