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Results measured in multiple clinical trials

The ELEVIDYS clinical trial program included more than 200 ambulatory participants with a range of genetic mutations across multiple trials.

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Study 1
Forty-one ambulatory participants
Forty-one ambulatory participants

Study 1 was a 2-part, placebo-controlled trial of 41 ambulatory people aged 4 to 7 years. In Part 1, which lasted 48 weeks, the participants were randomly split into 2 groups: 1 that received ELEVIDYS and 1 that received a placebo (a substance with no active medicine). 

Part 2 of the trial, which also lasted 48 weeks, reversed the groups, so those who had received ELEVIDYS in Part 1 now received a placebo, and vice versa. No one knew who was in each group.

MAIN GOALS WERE TO MEASURE

  • ELEVIDYS micro-dystrophin
  • Impact on muscle function
  • Safety
Study 2
Forty ambulatory participants
Forty ambulatory participants

Study 2, also called ENDEAVOR, is an open-label trial, which means everyone knew they were receiving ELEVIDYS. This trial included 40 ambulatory participants aged 3 to 12 years.

MAIN GOALS WERE TO MEASURE

  • ELEVIDYS micro-dystrophin
  • Safety

Muscle function was not a main goal of this study.

Study 3
One hundred twenty five ambulatory participants
One hundred twenty five ambulatory participants


Study 3, also called EMBARK, is a 2-part, placebo-controlled trial of 125 ambulatory people aged 4 to 7 years. In Part 1, which lasted 52 weeks, the participants were randomly split into 2 groups: 1 that received ELEVIDYS and 1 that received a placebo.

Part 2 of the trial, which also lasted 52 weeks, reversed the groups, so those who had received ELEVIDYS in Part 1 now received a placebo, and vice versa. No one knew who was in each group. Results from Part 1 are shared on this website.

Main goals WERE TO MEASURE

  • Impact on muscle function
  • Safety
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Results measured

ELEVIDYS micro-dystrophin

If ELEVIDYS micro-dystrophin was produced in skeletal muscles

Muscle function

If ELEVIDYS micro-dystrophin worked properly, measured through impact on muscle function 

Safety

Safety, or what side effects people experienced after ELEVIDYS

Multiple trials continue to gather information about the long-term impact of ELEVIDYS.

DID YOU KNOW?

Two ELEVIDYS trials included a placebo group: Study 1 and Study 3. Participants in the placebo group were treated exactly the same as the ELEVIDYS-treated participants, except they did not receive ELEVIDYS during their infusion. Comparing results from these 2 groups helps researchers understand the impact of treatment. These individuals did have the opportunity to receive ELEVIDYS later in the trial

DID YOU KNOW?

Two ELEVIDYS trials included a placebo group: Study 1 and Study 3. Participants in the placebo group were treated exactly the same as the ELEVIDYS-treated participants, except they did not receive ELEVIDYS during their infusion. Comparing results from these 2 groups helps researchers understand the impact of treatment. These individuals did have the opportunity to receive ELEVIDYS later in the trial

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Next up: Learn about eligibility

Now that you’ve explored the clinical trials, find out more about who may be eligible to receive ELEVIDYS.

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What is ELEVIDYS?

ELEVIDYS is a prescription gene therapy used to treat ambulatory individuals at least 4 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.

ELEVIDYS is not recommended for individuals with:

  • Preexisting liver problems or liver infection because of the high risk of rapid serious liver injury and rapid liver failure
  • Recent vaccination (within 4 weeks of ELEVIDYS treatment)
  • Current or recent infections (within 4 weeks of ELEVIDYS treatment)
     

Please see full Prescribing Information, including Boxed Warning, and Medication Guide.

Important Safety Information

What is the most important information to know about ELEVIDYS?
Rapid Serious Liver Injury and Rapid Liver Failure

  • ELEVIDYS can increase certain liver lab test levels and cause rapid serious liver injury, rapid liver failure, and death. Patients with preexisting liver problems may be at higher risk.
  • Complication of blood clots in the blood vessel in the abdomen that helps carry blood from the intestines to the liver has happened.
  • Patients will receive oral corticosteroid medication before and after ELEVIDYS infusion and will need weekly blood tests to monitor liver function for 3 months or longer after treatment.
  • For at least 2 months following ELEVIDYS infusion, stay close to a healthcare facility that the doctor recommends.
  • Contact a doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish or if the patient misses a dose of corticosteroid or vomits it up.

Serious Infection

  • Because patients will be taking corticosteroids as part of ELEVIDYS treatment, this may lower the ability of their immune system to fight infections and make it easier to get an infection. Getting an infection (like a cold, flu, stomach flu, ear infection, chest infection) before or after ELEVIDYS infusion could lead to more serious health problems, including death.
  • Contact a doctor right away if you notice any signs of infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Vaccinations should be completed at least 4 weeks before starting the corticosteroids that are part of the ELEVIDYS treatment.
  • ELEVIDYS should not be given to patients with an infection.

Inflammation of the Heart Muscle

  • Serious and life-threatening inflammation of the heart muscle has happened following ELEVIDYS infusion.
  • Patients will need weekly blood tests for a heart protein that can detect damage to the heart muscle cells for the first month after treatment.
  • Contact a doctor right away if the patient begins to experience chest pain and/or trouble breathing or shortness of breath.

Infusion-related Reactions

  • Infusion-related reactions, including hypersensitivity and serious allergic reactions (anaphylaxis), have happened during and after ELEVIDYS infusion.
  • Contact a doctor right away if you notice: fast heart rate, fast breathing, swollen lips, shortness of breath, nostrils widening, hives, red and blotchy skin, itchy or inflamed lips, rash, vomiting, nausea, chills, and fever.
  • Your doctor will monitor you during and at least 3 hours after ELEVIDYS infusion. If an infusion-related reaction occurs, your doctor may slow or stop the ELEVIDYS infusion and provide additional medical treatment as needed.

Immune Response Affecting Muscles (Immune-mediated Myositis)

  • Immune response affecting muscles, including serious and life-threatening reactions, has happened in patients about 1 month after receiving ELEVIDYS infusion.
  • Contact a doctor immediately if the patient experiences any unexplained increased muscle pain, tenderness, or weakness, including trouble swallowing, breathing, or speaking.

Antibodies to ELEVIDYS

  • Patients need to have blood tests to ensure that they do not have antibodies that may prevent them from being able to receive ELEVIDYS. High levels of antibodies may keep the medicine from working as intended.
  • Treatment with ELEVIDYS is not recommended for patients who have high antibodies to the vector, the part of gene therapy used to deliver ELEVIDYS.

You should discuss the potential benefits and risks of ELEVIDYS with your doctor.

Who should not receive ELEVIDYS?
Individuals with a certain type of genetic mutation, called a deletion, involving any portion of or the entire exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.

Are there any considerations for vaccination schedules and ELEVIDYS?
Patient vaccinations should be up to date with current immunization guidelines. Vaccinations should be completed at least 4 weeks before starting corticosteroids that are part of the ELEVIDYS treatment.

Are there any precautions that need to be considered when handling a patient’s bodily waste?
Vector shedding of ELEVIDYS occurs primarily through body waste. Patients and caregivers should use proper hand hygiene, such as hand washing, when coming into direct contact with patient body waste. Place potentially contaminated materials that may have the patient’s bodily fluids/waste in a sealable bag and dispose into regular trash. Precautions should be followed for 1 month after ELEVIDYS infusion.

What are the most common side effects of ELEVIDYS?
The most common side effects that occurred in patients treated with ELEVIDYS were vomiting, nausea, liver injury, fever, lower number of platelets (a kind of blood cell that helps you stop bleeding), and higher levels of heart protein that can detect damage to muscle cells in the heart. 

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away. 

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

Please see full Prescribing Information, including Boxed Warning, and Medication Guide.