Salanersen for Spinal Muscular Atrophy
(SOLAR Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen.
Specifically, researchers will learn more about how salanersen works in individuals with SMA who are between the ages of 15 and 60 years old. In most people living with SMA, changes to or a lack of a gene called survival motor neuron 1 (SMN1) - often referred to as gene mutations or variants - affect how this gene works. As a result, their bodies produce less SMN protein. Without enough of this protein, motor neurons and muscles cannot work properly. There is a similar gene called SMN2 that produces SMN protein, but it usually does not produce enough SMN protein on its own to make up for the changes in the SMN1 gene. Salanersen is a drug designed to help the SMN2 gene to make more working SMN protein.
In this study, there will be 2 groups of participants: a group who has never received treatment for SMA before joining this study, and a group who has been treated with risdiplam, an approved drug for SMA . Those participants must not have received any other SMA treatments before and will need to stop their risdiplam treatment for the duration of the study.
The main goal of this study is to learn more about how salanersen affects the participants' motor function. Researchers will use different tests and questionnaires to learn if motor function is changing over the study duration.
The main question researchers want to answer in this study is:
• For the group who has never been treated for SMA, how much do scores on the HFMSE movement test change at 12 months compared to the beginning of the study? The Hammersmith Functional Motor Scale - Expanded (HFMSE) has 33 activities that are scored which include sitting, lying down, walking, jumping, and more.
Researchers will also learn more about:
* The effects on participants' motor function and how well their nerves and muscles function.
* The effects on participants' overall sense of change and how they perform daily activities.
* How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug.
* How much salanersen gets into the fluid surrounding the brain and spinal cord.
* How much salanersen gets into the blood.
This study will be done as follows:
* First, participants will be screened to check if they can join the study. The screening period may be up to 4 weeks.
* This is an "open-label" study. This is a study in which the participants, study doctor, and site staff will know that participants are receiving salanersen.
* All participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord.
* Participants will receive salanersen once every year for a total of 5 times throughout the study.
* Including screening, participants will have 17 study visits and 9 telephone calls during this study, which will last up to 61 months in total.
Who Is on the Research Team?
Medical Director
Principal Investigator
Biogen
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive salanersen through intrathecal injection once every year for a total of 5 doses
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Salanersen
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Treatment-naïve participants will receive salanersen 80 milligrams (mg) by intrathecal (IT) lumbar puncture (LP) every 12 months for a total of five doses.
Risdiplam-treated participants will receive salanersen 80 mg by IT LP every 12 months for a total of five doses.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Biogen
Lead Sponsor
Daniel Quirk
Biogen
Chief Medical Officer
MD
Christopher A. Viehbacher
Biogen
Chief Executive Officer since 2022
Graduated from Queen's University, Kingston, Ontario, Canada
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