Ongoing, recruitingTherapeutic use (Phase IV)Interventional

A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - RESPOND

This study, called RESPOND, is looking at a medicine called Spinraza (nusinersen) for people living with Spinal Muscular Atrophy (SMA). Specifically, it's for patients who have already received a different treatment called Zolgensma (onasemnogene abeparvovec). The main goal is to understand if adding Spinraza can further improve movement and motor skills, like head control, sitting, and walking. Researchers will also be checking for any side effects or changes in health that might occur. This is a "Phase 4" study, meaning the medicine is already approved, and researchers are gathering more information about its use in a specific group of patients over about two years.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic use (Phase IV)

Sponsor

Biogen Idec Research Limited

Enrolment target

Start

01 Jul 2024

Spinal Muscular Atrophy

What is this study about?

This research study, named RESPOND, is investigating a medicine called Spinraza (nusinersen) for individuals who have Spinal Muscular Atrophy (SMA). SMA is a condition that affects nerve cells in the spinal cord, leading to muscle weakness and problems with movement. Spinraza is a treatment designed to help these nerve cells work better.

What makes this particular study important is that it focuses on patients with SMA who have already received another treatment called Zolgensma (onasemnogene abeparvovec). Zolgensma is a gene therapy, and researchers want to see if using Spinraza afterwards can provide additional benefits. The main thing they will be looking at is improvements in motor skills, such as head control, sitting, and standing, by using a special scoring system called the Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score.

This is a "Phase 4" study, which means Spinraza is already a licensed medicine. However, doctors are still gathering more information about how it works in different situations and patient groups, like those who have previously taken Zolgensma. The study will also be carefully monitoring for any side effects or changes in health, making sure the treatment is as safe and effective as possible for these patients.

Key takeaways

The study is about Spinraza for people with SMA.
It's for those who have already received Zolgensma.
The main goal is to see if Spinraza improves movement.
It's a two-year study tracking health and progress.
All ages and genders are welcome to potentially participate.

Who may be eligible?

This study is open to patients of all ages, including infants, children, and adults, who have been diagnosed with Spinal Muscular Atrophy (SMA).

To be considered for this study, participants must have previously received another SMA treatment called Zolgensma (onasemnogene abeparvovec).

Both males and females are welcome to take part in this research. The medical team will check other specific health details to ensure the study is right for each person.

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.

Do you have Spinal Muscular Atrophy (SMA)?
Have you previously received the SMA treatment called Zolgensma?
Are you able to attend regular study appointments for about two years?
Are you comfortable with receiving injections into your spine and having other medical tests?

Answer every question to see your result.

What does participation involve?

If you join this study, you would receive doses of Spinraza (12 mg) as a solution injected into your spine by a doctor. This treatment will be given on a schedule, and you would have study visits over about two years (up to 778 days). During these visits, doctors and nurses would perform various health checks and tests. These include physical examinations to assess your movement and motor skills, blood tests, and possibly heart rhythm tests (ECGs). They would also ask about any changes in your health or any side effects you might experience. Throughout your time in the study, doctors will closely monitor your progress and well-being.

Potential risks and benefits

Participating in research studies can offer the potential benefit of receiving close medical care and a treatment that might help improve your condition. However, Spinraza, like any medication, can have potential risks and side effects, and the study team will carefully explain these to you. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Spain
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Unverified
Germany
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Unverified
Italy

Common questions

What is Spinal Muscular Atrophy (SMA)?

SMA is a genetic condition that affects the nerves responsible for muscle movement, leading to muscle weakness over time.

What is Spinraza (nusinersen)?

Spinraza is a medicine that helps improve the way the body makes a protein needed for healthy nerve cells, which can help people with SMA.

Why are they studying Spinraza in people who already had Zolgensma?

Researchers want to see if using Spinraza can provide additional benefits for movement and well-being in patients with SMA who have already been treated with Zolgensma.

What kind of tests will be done during the study?

You might have physical exams, tests to measure your movement skills, blood tests, and heart rhythm checks (ECGs).

How long does the study last?

The study will involve regular visits and check-ups for about two years.

How to find out more

Official trial record

Always speak to your GP or specialist before deciding to take part in a study.