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 motor function required for everyday movements
- Safety
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
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 motor function required for everyday movements 1 year after ELEVIDYS
- Safety
ADDITIONAL ASSESSMENTS
Additional assessments on everyday movements were gathered 3 years after treatment with ELEVIDYS. These exploratory results help researchers learn about ELEVIDYS over time. Although they suggest positive trends, more data are needed to confirm the result. Talk to your doctor to learn more.
Clinical trials looked at a wide range of different measurements
How much ELEVIDYS micro-dystrophin was produced in muscles
The impact on motor function needed for everyday movements
What side effects people experienced
Impact on muscle loss
Researchers use different comparisons to evaluate how well ELEVIDYS works
To understand the impact of ELEVIDYS, researchers compared the results from people who received ELEVIDYS in Study 3 (“EMBARK”) with 2 other groups who did not receive ELEVIDYS. All participants were on stable doses of steroids at the start of the trial, regardless of whether or not they received ELEVIDYS.
Placebo
These Study 3 (“EMBARK”) participants did not receive ELEVIDYS. Instead, they were given a placebo, which is a treatment with no real medicine in it. Results were measured at 1 year and compared with results in people who received ELEVIDYS.
External control
These individuals did not receive ELEVIDYS or participate in Study 3 (“EMBARK”) but had both Duchenne and similar characteristics (eg, age, physical ability) to those who did. Results were measured at 3 years and compared with results in people who received ELEVIDYS.
