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An overview of ELEVIDYS clinical trials

The ELEVIDYS clinical trial program included more than 200 participants across multiple trials.

Age range of three to twenty years old
3 to 20 years old*
Ambulatory and non-ambulatory
Ambulatory and non-ambulatory†
Range of genetic mutations
A range of genetic mutations‡
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* ELEVIDYS is approved for individuals at least 4 years old with Duchenne. 
† Safety information in non-ambulatory people is limited to 8 clinical trial participants. 
‡ ELEVIDYS cannot be used in anyone with a deletion in exon 8 and/or exon 9 in the dystrophin gene.

 

 

 

There were 3 main trials

Each trial included different people and had specific goals

Study 1
Forty-one ambulatory participants. Four to seven years old. ELEVIDYS versus placebo
Forty-one ambulatory participants, Four to seven years old, ELEVIDYS versus placebo


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-eight participants, Three to twenty years old.
48 participants, Three to twenty years old, ELEVIDYS

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
  • 8 non-ambulatory participants aged 10 to 20 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, ELEVIDYS micro-dystrophin, ELEVIDYS versus placebo


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.

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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 (delandistrogene moxeparvovec-rokl)?

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 approved under accelerated approval for non-ambulatory patients at least 4 years old with DMD who have a confirmed mutation in the DMD gene. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. ELEVIDYS treatment increased the marker, ELEVIDYS micro-dystrophin in skeletal muscle. Verification of a clinical benefit may be needed for ELEVIDYS to continue to be approved for non-ambulatory patients with DMD.

Important Safety Information

Who should not receive ELEVIDYS?
Individuals with certain types of mutations, any deletion in exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.

What is the most important information to know about ELEVIDYS?
Infusion-related reactions, including hypersensitivity and serious allergic reactions (anaphylaxis), have occurred during and after ELEVIDYS infusion. Symptoms may include 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. Contact a healthcare provider immediately if infusion-related symptoms occur. 

ELEVIDYS can increase certain liver enzyme levels and cause acute serious liver injury. Patients will receive oral corticosteroid medication before and after infusion with ELEVIDYS and will undergo weekly blood tests to monitor liver enzyme levels for 3 months after treatment. Contact a healthcare provider 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. 

Administration of ELEVIDYS may be delayed in patients who have acute liver disease until the condition is resolved or under control. Patients with preexisting liver impairment, chronic liver infection, or acute liver disease may be at higher risk of acute serious liver injury.

Immune-mediated myositis (an immune response affecting muscles) was observed in patients with a deletion mutation in the DMD gene that is contraindicated. Patients with certain mutation deletions (in exons 1 to 17 and/or exons 59 to 71) may be at risk for a severe immune-mediated myositis reaction. Caregivers should contact a healthcare provider immediately if the patient experiences any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, breathing, or speaking, as these may be symptoms of myositis. 

Myocarditis (inflammation of the heart) has been observed within days following ELEVIDYS infusion. The patient’s doctor will conduct weekly blood tests for the first month after treatment to evaluate troponin-I (a cardiac protein that can detect damage to muscle cells in the heart). Caregivers should contact a healthcare provider immediately if the patient begins to experience chest pain and/or shortness of breath. More frequent monitoring may be required if the patient has cardiac symptoms. 

Patients need to have blood tests to ensure that they do not have antibodies that may prevent them from being able to receive ELEVIDYS, as introducing the gene therapy could increase the risk of a severe allergic reaction or prevent desired therapeutic levels. Treatment with ELEVIDYS is not recommended for patients who have high antibodies to the vector, the part of gene therapy used to deliver ELEVIDYS. 

Due to the need to follow a corticosteroid regimen, an infection (such as cold, flu, gastroenteritis [stomach flu], otitis media [ear infection], bronchiolitis [respiratory infection], etc) before or after ELEVIDYS infusion could lead to more serious complications. Caregivers should contact a healthcare provider immediately if they see any symptoms suggestive of infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.

Are there any considerations for vaccination schedules and ELEVIDYS?
Patient vaccinations should be up to date with current immunization guidelines. Vaccinations should be received at least 4 weeks prior to starting the corticosteroid regimen that is required before receiving ELEVIDYS.

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 possible or likely side effects of ELEVIDYS?
The most common side effects that occurred in patients treated with ELEVIDYS were vomiting, nausea, liver injury, fever, and decreased platelet counts. 

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 for ELEVIDYS.

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