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SHIELD DMD study overview

What is SHIELD DMD?

SHIELD DMD is a global clinical study recruiting participants to test a potential new treatment for young people living with Duchenne Muscular Dystrophy (“Duchenne”).

Around 50 young individuals will take part in SHIELD DMD. The study will look at a study medicine called satralizumab (also known as RO5333787) and if it is safe and works well in young people living with Duchenne to prevent bones breaking and improve muscle strength.

About Duchenne Muscular Dystrophy (DMD)

Duchenne is a genetic condition that makes your muscles get weaker and weaker. Over time, weakened muscles are replaced with fat tissue causing problems with walking, breathing, and heart function. Individuals with Duchenne will eventually need to use a wheelchair.
When muscles are not strong enough to support the bones, they cannot grow properly and become fragile. As muscles and bones become weaker, it is easier to break bones. The femur, which is the longest bone in the legs, is the most commonly broken bone in individuals with Duchenne. Current treatments for Duchenne (steroids) often have unwanted effects that affect the bones as well. They can increase the number of fractures, especially fractures in the bones of the spine, or vertebrae. People living with Duchenne typically lose the ability to walk before they reach the age of 13. If these fractures can be prevented or minimized, it could also help individuals with Duchenne walk for a longer time and have a better quality of life.
Right now, there aren't any approved treatments for Duchenne that stop or prevent bones from breaking. Physical therapy can help with the symptoms but there is an urgent need to have new treatments to prevent this.

About the study medicine (satralizumab)

People living with Duchenne have an abnormal inflammatory response due to the constant damage to the muscles. Interleukin-6 (IL-6) is one of the proteins involved in this process. Abnormal activity of IL-6 causes constant inflammation, which weakens muscles and causes bones to become fragile. The study medicine, satralizumab, blocks the activity of the IL-6 receptor (IL-6R) and may be able to help individuals with Duchenne have healthier muscles and bones.

Satralizumab is already approved by healthcare authorities for a different disease called Neuromyelitis Optica Spectrum Disorder (NMOSD). It has been shown to be safe for both adults and children.
Satralizumab is given as a subcutaneous (under the skin) injection into the stomach or thigh every 4 weeks

Can your child join SHIELD DMD?

Your child may be eligible to join the SHIELD DMD study if they:

  • Are at least 8 years old and under 16
  • Have a confirmed medical diagnosis of Duchenne by genetic testing
  • Have been receiving corticosteroids for at least 12 months with a stable dose for at least 12 weeks before the start of the study
  • Additional criteria may apply based on your child’s ability to walk (ambulatory vs non-ambulatory)

If your child meets the study criteria and you are both interested in taking part in SHIELD DMD, you will be asked to read and sign an Informed Consent Form. An assent form for your child will also be collected where appropriate.

Choosing to take part in a clinical study is an important and personal decision. It is important to discuss study participation with your family and primary care physician to make sure it is right for you.

Informed Consent Form or Assent Form

Signing the Informed Consent Form and/or the Assent Form means you understand what the study involves, and you agree to take part. Please note, you can change your mind at any time.

What happens if your child joins the study?

1. Screening (up to 3 months)

After signing your Informed Consent Form and/or the Assent Form, the screening period will begin. The study doctor will do tests to confirm eligibility. These will include:

  • Review of your child's background information, medical history, and current medications
  • Measuring how long it takes your child to get up from the floor when lying on his back (if your child can walk)
  • Vital signs measurements including body temperature, blood pressure, breathing rate, and pulse
  • Physical examination
  • Weight measurement
  • Height measurement (if able to walk) or measurement of the length from your child’s elbow to wrist (if not able to walk)
  • Taking blood samples
  • Electrocardiograms (ECG) to measure the heart’s electrical activity
  • Echocardiogram to look at the heart and nearby blood vessels
  • X-rays of your child's left hand and spine
  • Body scan to measure total lean body mass, fat mass, and bone mineral density
2. Treatment (2 years)
If your child qualifies, they will receive the study medicine every 4 weeks for 2 years (plus an extra dose at Week 2). The first 4 doses (at Weeks 1, 2, 4 and 8) must be given at the study clinic. After that, you can give your child the injections at home or they can inject themselves if they are at least 12 years and feel comfortable with it.
SHIELD DMD is an open-label study - this means that all participants will receive the study medicine

How often are clinic visits?

There are 10 in-person study clinic visits in the treatment period, plus the initial screening visit and the final safety follow-up. They happen every 2-26 weeks depending on how far along your child is in the journey. There will also be monthly phone calls from the study clinic to check in on your child’s health.

How long do visits take?

Most visits will last approximately 4 hours, but a few may last up to 8 hours.

What happens during visits

The tests will be similar to screening and depending on the visit may also include:

  • Movement and strength tests
  • Questions regarding discomfort levels
  • Checking the skin where the study medicine was injected
3. Safety visit (3 months)
There will be one final in-person visit about 3 months after the last dose of study medicine. At this visit, your child will have tests to check his health and well-being after stopping treatment.
The total length of the study is about 2 and a half years. If you and your child choose to leave the study early there will be a final visit within 4 weeks of the last dose of study medicine. The tests done at this visit will be similar to those done at screening and in the treatment period.

What are your obligations during the study?

  • Keep and attend study appointments and complete all study tests
  • Inform the study team about any symptoms or side effects, changes in medications, other doctor’s or nurse’s appointments, or hospital admissions
  • Agree to not participate in any other clinical study during the time you will both be in the SHIELD DMD study
  • Tell the study team if you change your mind about participating in the study
  • Tell your child’s other doctors that they are taking part in this study
  • Ensure that your child is carrying a study identification card at all times during the study

While receiving the study medicine, you and your child will be required to:

  • Take the study medicine as instructed
  • Transport and store the study medicine as instructed
  • Use the study medicine only with your child who is participating in the study
  • Keep the study medicine in a safe place, away from other children not participating in this study

What are the benefits?

There is no guarantee that your child will receive any benefits from this study. Their health may or may not improve. Information from this study will help doctors learn more about the study medicine, and the treatment of individuals with Duchenne. It may also benefit other patients with Duchenne or a similar condition in the future.

What are the risks?

There are potential risks associated with any clinical study. Your child may have side effects from the study medicine or tests in this study. Side effects can vary from mild to serious and may vary from person to person.

Side effects associated with the study medicine include but are not limited to the following:

Very common(happens in more than 10% of patients)

  • Headache
  • Joint pain
  • Injection reactions (swelling, itching, rash)
  • Decreased white blood cell count

Common(happens in 1-10% of patients)

  • Muscle stiffness
  • Difficulty sleeping or falling asleep
  • Swelling of hands, feet or lower leg
  • Rash or itching
  • Migraine
  • Hay fever
  • Decreased level of a blood protein needed for blood clotting
  • Increased blood level of a liver pigment, often a sign of liver problems
More information is available in the Informed Consent Form and Assent Form. Your child’s health is important to us and will be monitored throughout the study.

Are there any costs?

You will not be charged for the study medicine while your child is participating in this study. All tests for the SHIELD DMD study that are not part of your child’s regular medical care will be provided to you at no charge.

Note, some health plans will not pay for medications or procedures for people participating in clinical studies. Your doctor can find out what your health plan will pay for.

You may be reimbursed for meals or any reasonable travel expenses that incur while taking part in this study. If your child is injured because he participates in this study, you/your child will be entitled to receive compensation per national legislation. The study doctor will explain how you can obtain a copy of these guidelines.

I'm excited to take part! What happens next?