A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
This study, called ASCEND, is for teenagers and adults aged 15 to 50 living with Spinal Muscular Atrophy (SMA). It's designed for those who can't walk on their own and have already tried another SMA medicine called risdiplam. The main aim is to see if a higher dose of nusinersen, given through injections into the spine, improves muscle strength and movement, particularly in the arms and hands. Researchers will use a special test called the Revised Upper Limb Module to track these changes. They will also carefully monitor participants for any side effects and other changes in health to understand how safe the higher dose is. The study involves regular injections and check-ups over about 4.5 years.
At a glance
What is this study about?
This study, called ASCEND, is looking into a new way to treat Spinal Muscular Atrophy (SMA) in teenagers and adults. SMA is a condition that affects your muscles, making them weaker over time. This particular study is for people who can't walk on their own and have already been taking a different SMA medicine called risdiplam. The aim is to see if a higher dose of another medicine, nusinersen, can help improve their muscle strength and ability to move.
The main thing researchers want to find out is whether this higher dose of nusinersen helps with movement, especially in the arms and hands. They will use a specific test called the Revised Upper Limb Module to measure how well people can do tasks that use their arms, shoulders, elbows, wrists, and hands. This test helps track any changes in these abilities over time. They also want to understand if this higher dose is safe and whether participants experience any side effects.
Taking part in this study means you would receive nusinersen injections into the fluid around your spinal cord in your lower back. This would happen regularly over several years. Researchers would also keep a close eye on your health with various tests. The hope is that this research will provide valuable information to help improve treatments for people living with SMA.
Key takeaways
- It's for teenagers and adults (15-50 years old) with SMA.
- Participants must have previously taken risdiplam and cannot walk independently.
- Investigates a higher dose of nusinersen for muscle and movement improvement.
- Medicine is given via spinal injections (lumbar puncture).
- Study involves regular doses and check-ups over approximately 4.5 years.
- Your health and safety will be closely monitored throughout.
Who may be eligible?
This study is looking for teenagers and adults with a specific type of SMA (5q SMA), confirmed by genetic tests. You need to be between 15 and 50 years old and weigh more than 20 kg. Importantly, you must have started experiencing SMA symptoms after 6 months of age, and you shouldn't be able to walk independently.
To join, you must have already taken risdiplam. If you haven't taken nusinersen before, you need to have been on risdiplam for at least 6 months. If you have taken nusinersen in the past, you must have stopped it for over 16 months and been on risdiplam for at least 12 months before joining the study. You also need to be able to do certain arm and hand movement tests within a specific score range.
There are also some reasons why you wouldn't be able to join. These include having a major illness recently, an untreated infection, having a shunt for brain fluid drainage, or needing permanent breathing support. Having certain brain or spine-related medical conditions like bacterial meningitis or hydrocephalus would also exclude you. The study doctors will carefully check if any ongoing health issues might interfere with the study assessments or your safety.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 15 and 50 years old?
- Have you been diagnosed with 5q Spinal Muscular Atrophy (SMA), confirmed by genetic tests?
- Do you get around without walking independently?
- Have you been taking risdiplam for a certain period? (At least 6 months if never had nusinersen, or 12 months if you stopped nusinersen over 16 months ago)
- Are you willing to stop risdiplam if you join the study?
- Do you have good general health without recent major illnesses or untreated infections?
What does participation involve?
If you decide to take part, the first step is a screening process to check if you meet all the study requirements. If you do, you'll enter the main treatment period. You'll receive two initial, slightly higher 'loading' doses of nusinersen, given two weeks apart. After that, you'll get regular 'maintenance' doses every four months. All these doses are given as injections into the fluid around your spinal cord in your lower back – a procedure called a lumbar puncture.
The main treatment period lasts about two years, and there will be a follow-up visit four months after your last dose. If you complete this period, you might have the option to continue receiving the maintenance doses for another two years in an extended phase of the study, with another follow-up visit. In total, you would have up to 18 visits to the clinic over about 4.5 years if you complete both parts of the study. Researchers will also call you after each dose to check in on how you're doing.
Potential risks and benefits
Locations (22)
- Barrow Neurological InstituteVerified postcodePhoenix, United States
- Stanford Neuroscience Health CenterVerified postcodePalo Alto, United States
- Rare Disease Research, LLCVerified postcodeAtlanta, United States
- Boston Children's HospitalVerified postcodeBoston, United States
- Memorial HealthcareVerified postcodeOwosso, United States
- Columbia UniversityVerified postcodeNew York, United States
- Wake Forest University - School of Medicine - CentralVerified postcodeWinston-Salem, United States
- University of PennsylvaniaVerified postcodePhiladelphia, United States
- Neurology Rare Disease CenterVerified postcodeDenton, United States
- The University of Texas Health Science Center at HoustonVerified postcodeHouston, United States
- University of Washington Medical CenterVerified postcodeSeattle, United States
- Universitaetsklinikum HeidelbergVerified postcodeHeidelberg, Germany
Common questions
What is the main goal of this study?
The main goal is to see if a higher dose of nusinersen can improve muscle and movement abilities, especially in the arms and hands, for teenagers and adults with SMA already taking risdiplam.
How is the medicine given?
The medicine, nusinersen, is given through an injection into the fluid around your spinal cord in your lower back (a lumbar puncture).
How long will the study last?
If you complete both the main treatment period and the optional extended period, you would be in the study for about 4.5 years.
Who can join this study?
This study is for teenagers and adults aged 15-50 with a specific type of SMA who can't walk and have previously received risdiplam. There are also other health requirements.
Will I have to stop my current SMA medicine?
Yes, if you join this study, you will need to stop taking risdiplam.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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