A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD
Spinal muscular atrophy (SMA) is a rare genetic condition that causes muscles to weaken over time. Nusinersen, also known as Spinraza, is a medicine that helps the body produce a protein needed for healthy nerves and muscles. This trial, called ONWARD, is an extension study. It is designed for people with SMA who have already taken part in a previous clinical trial testing higher doses of nusinersen (28 mg or 50 mg). Standard treatment uses a lower dose, so doctors want to find out if higher amounts give better long-term support. Over roughly five years, researchers will monitor safety, muscle strength, and breathing. They will check heart health, blood clotting, and kidney function. Taking part helps doctors understand whether higher doses are safe and effective for long-term care.
At a glance
What is this study about?
Spinal muscular atrophy (SMA) is a condition that affects nerve cells in the spinal cord. Without enough of a specific protein, these nerves break down, leading to muscle weakness that can make moving, sitting, and breathing difficult. Nusinersen (Spinraza) was developed to help the body make more of this essential protein.
Currently, nusinersen is approved at a standard dose. However, doctors believe that higher doses might provide greater benefits for some people. The ONWARD study is a long-term follow-up study looking at higher doses, specifically 28 mg and 50 mg. It welcomes people who have already received nusinersen in an earlier trial to see how they do over an extended period.
The main goal of the trial is to check patient safety over up to five years (around 1,921 days). The research team will track how well the body tolerates the higher dose by monitoring growth, heart rhythms, and blood and urine samples. They will also measure physical movement and see if participants need help with breathing.
This study matters because it could help doctors understand if giving more medicine safely improves physical ability and quality of life for people living with SMA. The findings may shape how the condition is treated in the future.
Key takeaways
- The ONWARD study is testing higher doses of nusinersen (Spinraza) for spinal muscular atrophy.
- It is an extension study for people who already took part in a previous nusinersen trial.
- The trial will follow participants for around five years to check long-term safety.
- Doctors will closely monitor heart health, blood clotting, kidneys, and muscle movement.
- Taking part is voluntary, and you can withdraw at any time.
Who may be eligible?
This trial is specifically for people diagnosed with spinal muscular atrophy who have already taken part in an earlier trial testing nusinersen. Because it is an extension study, it is not open to people who are completely new to the medicine or who have not been in a previous trial run by the study sponsor.
According to study records, participants are generally aged 18 and older, and the trial is open to all genders.
To take part, participants must also be able to undergo routine health checks and receive injections into the spinal fluid. Your study doctor will review your medical history to make sure continuing in this extension trial is safe for you.
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.
- I have a confirmed diagnosis of spinal muscular atrophy (SMA).
- I have already taken part in a previous clinical trial for nusinersen.
- I am aged 18 or older.
- I am able to attend regular hospital visits for up to five years.
- I am willing and able to have injections into the spinal fluid (lumbar punctures).
What does participation involve?
Taking part in this study lasts for up to five years (around 1,921 days). The study medicine is given through a lumbar puncture, which is a small injection into the fluid around the spine. Participants receive scheduled doses throughout the study period.
During clinic visits, you will have regular health checks. These include blood tests to check how your blood clots, urine tests to check your kidneys, and heart traces (ECGs) to check your heart rhythm. The study team will also measure your height and weight, test your muscle movement, and ask questions about your daily breathing.
You will have regular appointments so the medical team can closely follow your progress and answer any questions you or your family might have.
Potential risks and benefits
Locations (3)
- —UnverifiedItaly
- —UnverifiedSpain
- —UnverifiedGermany
Common questions
What is an extension study?
An extension study allows people who finished an earlier clinical trial to continue receiving the study medicine. It helps researchers gather long-term safety and health information over several years.
How is the medicine given?
Nusinersen is given through an injection into the lower back, into the fluid surrounding the spinal cord. This is known as a lumbar puncture.
Why test higher doses?
Doctors want to find out if giving a higher dose than the standard approved amount can protect nerves better and provide greater improvements in movement and breathing.
Will taking part cost me anything?
Clinical trials generally provide the study medicine and related tests free of charge. Many trials also help cover travel expenses to and from the hospital.
What happens if I change my mind?
You are free to leave the trial at any time. Your decision will not change the standard of medical care you receive from the NHS.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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