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Learn about our Duchenne
Muscular Dystrophy research study.
Study Overview
What is the ULYSSES study about?
This study is being done to learn more about an investigational drug called givinostat. The study will test its efficacy in reducing muscle decline in people with Duchenne muscular dystrophy (DMD).
Learn more about the study by watching our video.
How does givinostat work?
DMD is caused by changes (mutations) in the DMD gene, which contains the instructions for cells to make a protein called dystrophin. Dystrophin is important for muscle health and helps prevent wear and tear on the muscles when you move. Mutations in the DMD gene cause the body to make less dystrophin. Without this protein, muscles can become damaged over time and replaced by scar tissue (fibrosis) and fat. This leads to muscle wasting and weakness.
Givinostat blocks enzymes called histone deacetylases (HDACs), which turn off gene activity and can affect the ability of muscle cells to repair themselves (regenerate). By blocking HDACs, givinostat may reduce muscle fibrosis and wasting and help muscle cells to regenerate.
Taking Part in the Study
Who can take part in this study?
About 138 boys will take part in the study at research sites all over the world.
Eligible participants must:
- Be male, aged 9 to 17 years (at the start of the study)
- Have a genetic diagnosis of Duchenne muscular dystrophy (DMD)
- Be non-ambulant (generally using a wheelchair for mobility)
- Be treated on a stable dose of systemic steroids for at least the past 6 months (such as prednisone, prednisolone, or deflazacort)
- Have not received any gene therapy treatments for DMD
- Have not had exposure to any dystrophin restoration product in the past 6 months (such as Ataluren, Viltepso, Exondys 51, Vyondys 53, Amondys 45)
Medical history and other criteria will be reviewed to determine eligibility.
How does the study work?
This study lasts about a year and a half and requires about 14 study-related site visits. The study is divided into 3 main parts:
(up to 1 month ±14 days)
(18 months)
If you qualify, you will enter the study treatment period and be assigned at random to one of two study treatments: the investigational drug (givinostat) or placebo (an inactive substance).
There is a 2 in 3 chance of getting the investigational drug and a 1 in 3 chance of getting placebo. You will take the study treatment twice each day for about 18 months. You and your parent/guardian will return to the study site about 10 times for additional study tests and procedures.
OPTIONAL LONG-TERM SAFETY STUDY:
If you complete the study treatment period, you will be invited to enrol in a long-term safety study in which you would take givinostat until the treatment becomes commercially available. During the long-term safety study, all participants will receive givinostat, even those who were previously taking placebo.
Why should I join the study?
DMD is a rare, progressive, debilitating, and life-threatening condition for which there is a critical need for novel therapies that are effective and well-tolerated in all people with DMD. Being in this clinical study may or may not help your DMD. However, we believe some benefits might include:
- Research opportunity for non-ambulant people with DMD as not many effective treatments are available
- Close monitoring of your health by a professional research team
- A chance to contribute to what we know about DMD
- A chance to help other people with DMD
Are there risks in the study?
There may be risks from taking the investigational drug. More information about the possible risks, side effects, discomforts, and inconveniences related to the study is available in the informed consent form and should be discussed with the study doctor.
Do I have to be in the study?
No. Being in a clinical study is completely voluntary. You do not have to join the study. Even if you join the study, you can stop being in the study at any time.
ABOUT CLINICAL STUDIES
What is a clinical research study?
A research study is designed to help us learn more about medical devices, drugs, or diagnostic tests. Health authorities use the results of research studies to decide if an investigational drug should be made available for use in a particular country.
How are my rights protected?
Every research study is reviewed by an Institutional Review Board (IRB) or Ethics Committee (EC), which helps ensure that the study is conducted safely and that the rights and privacy of study participants are protected. Research studies are conducted by experienced and trained medical professionals who monitor the health of participants throughout the study.