OVERVIEW: PART A
OVERVIEW: PART B
The purpose of this study is to evaluate the safety and tolerability of the study drug, SRP-5051. The study is divided into two parts, Part A and Part B. The goal of Part A is to find the highest dose of the study drug that is considered safe and well tolerated. Once this dose is identified in Part A, all participants will then receive this dose in Part B.
Participant Eligibility Criteria
Your child may be eligible to participate in Momentum trial if he meets the following eligibility criteria:
- Ambulatory and non-ambulatory boys with DMD who are 7-21 years old (patients who enroll in Part A) or 4-21 years old (patients who enroll in Part B)
- Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amendable to exon 51-skipping treatment. Talk to your doctor if you are unsure.
- Has been on a stable dose of corticosteroids for at least 12 weeks, or has not received corticosteroids for at least 12 weeks prior to receiving SRP-5051
- Has stable lung (breathing) and heart function.
- Has not received treatment with any exon-51 skipping therapy within 12 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time
Additional requirements for participation will be reviewed with patients and their families during the screening process.
Frequently Asked Questions
What is an open label study?
Open-label means that the study doctor, nurses, caregivers, Sponsor, and you will all know that you are receiving study drug, and at what dose level.
What does multiple ascending dose mean?
Multiple means that all participants will receive doses of the study drug once every 4 weeks throughout the study. Ascending dose means that there are different study drug dose levels in Part A that increase from one dose group to the next. All participants will eventually move to the same dose level, the maximum tolerated dose, in Part B of the study.
How many boys will be enrolled in this study and where is it being run?
Approximately 45 participants are planned to be enrolled in this study, with clinical trial sites planned in the United States, Canada and Europe. Additional information on the location of clinical trial sites may be found on ClinicalTrials.gov.
How long is the study?
Participation in the study is expected to last approximately 70 weeks for participants who enroll in Part A. It may be shorter or longer depending on the time it takes to determine the highest tolerated dose. Duration of participation for participants who enroll in Part B of the study is expected to last approximately 32 weeks. Participants who complete the study may be eligible to participate in a long-term extension study.
Will I be paid for participating?
Participants will not be paid for participation in this study. However, reasonable travel costs associated with participation in the study will be prepaid or reimbursed by Sarepta in accordance with the approved study travel policy. Information will be provided by the study site.
What are some of the activities that will be required to take part in this study?
Enrolled participants will visit study sites for monthly dosing, functional assessments, medical testing, and 2-3 muscle biopsies over the course of the study. Two to three visits will require overnight stays at the study site. Participants will also undergo safety labs two weeks after each dose. Additional information will be provided by the study site.
Why do you need to collect biopsies?
This study will measure the change in the amount of dystrophin protein in muscle after treatment. Because we are testing to see if SRP-5051 increases levels of dystrophin in muscle through exon-skipping, the only way to show SRP-5051 is working as intended is to test muscle directly. This cannot be studied by testing blood or urine, for example. More information will be provided by the study site.
What risks are associated with this study?
As with all clinical studies, there can be risks associated with possible side effects of taking the study drug and with the standard medical tests carried out as part of the study at each visit. Information on the possible side effects that may be experienced in the study is available in the consent form and should be discussed with your study doctor.
What are some benefits to being a part of this clinical trial?
The potential benefits of SRP-5051 in patients with DMD are unknown. Even if you do not benefit from being in this study, we might learn something that could advance research and help others.
Exon Skipping for Duchenne
Duchenne is a rare, life-shortening genetic disorder that affects boys and causes their muscles to break down and lose strength over time. Duchenne is caused by specific errors (mutations) in the gene that codes for dystrophin. Dystrophin is a protein that plays a key role in the function of muscle cells and protects them from damage as muscles contract and relax. These mutations in the dystrophin gene lead to a lack of dystrophin protein in muscles. Without enough dystrophin, muscles gradually grow weaker until they can’t move at all, and eventually breathing and heart function are lost.
SRP-5051 is an investigational drug designed to treat Duchenne in patients who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51. It uses a technology called exon skipping.
About Exon Skipping
Exon-skipping technologies strive to address the underlying issue with Duchenne—a lack of the protein dystrophin. Many people with Duchenne have a genetic mutation in which one or more exons in the dystrophin gene are missing. This causes errors in the instructions for making dystrophin, leaving the body unable to produce the protein.
Exon skipping tells the body to hide an exon next to the missing piece so the whole section can be skipped over and the remaining exons can fit together. The goal of exon skipping is to allow the body to make a shorter form of the dystrophin protein.