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Study design

A detailed look at the clinical study

SAPPHIRE was the phase 3 clinical trial used for the FDA (US Food and Drug Administration) approval of ISEMBYLD. Here’s who participated and what the study measured.

Review the Efficacy & Safety Data

Gender

Gender breakdown of SAPPHIRE study participants: 50% male
50% female

Study length

Study length: 52 weeks
188 people participated in the SAPPHIRE study. Age range: Primary Study Group 2 to 12 years old, average age 8; Exploratory Study Group 13 to 21 years old, average age 16. Study participant breakdown: 156 in the primary and 32 in the exploratory
188 people participated in the SAPPHIRE study. Age range: Primary Study Group 2 to 12 years old, average age 8; Exploratory Study Group 13 to 21 years old, average age 16. Study participant breakdown: 156 in the primary and 32 in the exploratory
Gender breakdown of SAPPHIRE study participants: 50% male and 50% female. Study length: 52 weeks

THE PARTICIPANTS’ SMA HISTORY

When did they begin seeing SMA symptoms?

On average, participants aged 2-21 years had an onset of SMA symptoms when they were 1 year old.

What type of SMA did they have?

Participants had type 2 and type 3.

What treatments were they receiving, and for how long?

# OF PEOPLETREATMENTDURATION
139 of 188 peopleSpinraza®* (nusinersen)5-6 years average
49 of 188 peopleEvrysdi®* (risdiplam)3 years average

What was their level of mobility?

100% of participants were non-ambulatory (unable to walk independently without aids or orthotics over 10 steps).

Average HFMSE Scores at the Start of the Study

Average HFMSE scores at the start of the SAPPHIRE study: approximately 26 for ISEMBYLD group ages 2-12, approximately 21 for ages 13-21; approximately 28 for placebo group ages 2-12, approximately 23 for ages 13-21

aPlacebo is a substance that has no therapeutic effect.
HFMSE=Hammersmith Functional Motor Scale–Expanded.

THE PROCESS

Over the 52 weeks of the study:

Participants were treated every

4

weeks

Motor function was assessed approximately every

8

weeks

How was the study set up?

Participants continued on their SMN2-targeted treatment while they were treated with ISEMBYLD or a placebo (ie, a substance with no therapeutic effect) every 4 weeks over the course of a year. Motor function was assessed approximately every 8 weeks. At the end of the study, motor function changes were compared between the group receiving ISEMBYLD with their SMN2-targeted treatment and the group receiving SMN2-targeted treatment alone.

How was motor function measured in the study?

The Hammersmith Functional Motor Scale–Expanded (HFMSE) is one of several tests that assesses motor function abilities in your daily life. It was the primary scale used to measure whether ISEMBYLD improved motor function after 52 weeks of treatment. Functional activities tested include lying down, head lifting, rolling, sitting up, and standing.

See what ISEMBYLD could mean for you.

EXPLORE THE FINDINGS