A Study of the Safety, Tolerability, and Pharmacokinetics (drug activity) of Experimental 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, genes
Type: Drug study, Phase 1
Target:
5 Participants
Investigator:
Description
The purpose of this study is to find the highest dose of SRP-5051 (study drug), that can be tolerated without causing very severe side effects. This is done by starting at a dose lower than the one that does not cause side effects in animals. This is the first time this has been tested in people. Participants are given the study drug and are watched very closely to see what side effects they have and to make sure the side effects are not severe. If the side effects are not severe, then new participants will be given a higher dose of SRP-5051. Participants joining this study later on will get higher doses of SRP-5051 than participants who join earlier. This will continue until a dose is found that causes severe side effects or the highest dose planned for this study is reached. Doses higher than that will not be given.

SRP-5051 is not approved by the U.S. Food and Drug Administration (FDA). 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 instructions telling our bodies 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 or “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 SRP5051 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 a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping treatment
  • Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration with continued dosing of oral corticosteroids while participating in the study*, or has not received corticosteroids for at least 12 weeks prior to study drug administration and will not initiate dosing of oral corticosteroids while participating in the study

Exclusion criteria:

  • Has a left ventricular ejection fraction (LVEF) less than (<) 40 percent (%) based on an echocardiogram (ECHO) performed within 3 months prior to Screening or at the Screening visit
  • Has a QT interval corrected with Fridericia's method (QTcF) >= 450 millisecond (msec) on the Screening electrocardiogram (ECG)
  • Initiation or change of dosing (except for modifications to accommodate changes in weight) within 12 weeks prior to Screening and while participating in the study for any of the following: angiotensin-converting enzyme (ACE) inhibitors, angiotensin-receptor blocking agents (ARBs), beta-blockers, or potassium
  • Requires antiarrhythmic and/or diuretic therapy for heart failure
  • Forced vital capacity (FVC) <40% of predicted value within 3 months of Screening or at the Screening visit
  • Known kidney disease or had an acute kidney injury within 6 months prior to Screening
  • Treatment with eteplirsen or drisapersen within 6 months prior to Screening, or any experimental gene therapy for the treatment of DMD at any time
  • Use of any herbal medication/supplement containing aristolochic acid 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 study. Reasonable travel expenses may be covered for you. This may include mileage, parking, meal allowance, and overnight stays.
Resources
Schedule
Study duration and period
Your participation in this research study will last about 14 weeks: • 2 weeks in the Screening Period, • 4 weeks in the Treatment and Observation Period, and 8 weeks in the Safety Follow-up Period. During this time, you will need to go to the site 5 times for scheduled study visits. One of these visits requires at least 2 consecutive overnight stays at the study site.
Recruitment period
From July 9, 2018
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Contact
Elizabeth Guy
Research Topic
Conditions:
  • Muscular Dystrophy
  • Duchenne