Active not recruitingNAINTERVENTIONAL

Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA®

This study aims to understand how a treatment called Spinraza (also known as nusinersen) affects movement in adults living with Spinal Muscular Atrophy Type II. Spinraza is a medication approved to treat SMA. The main goal is to observe changes in muscle function over time using a special series of tests. This research is important because it helps doctors and patients understand the long-term effects of Spinraza on adults with this specific type of SMA. It focuses on adult patients who use a wheelchair and helps gather more information about how this treatment works in a real-world setting. The study will carefully monitor their progress to learn more about improving daily life for these patients.

At a glance

Status

Active not recruiting

Phase

Sponsor

Centre Hospitalier Universitaire de Nice

Enrolment target

Start

25 Feb 2020

Estimated completion

31 Dec 2025

Spinal Muscular Atrophy

What is this study about?

This study is looking into how a medicine called Spinraza helps adults with a specific type of muscle weakness called Spinal Muscular Atrophy Type II (SMA Type II). Spinraza (the proper name is nusinersen) was the first medicine of its kind approved to treat SMA in both children and adults. It's given by an injection into the fluid around the spinal cord.

The main aim of this research is to keep an eye on how well Spinraza works over time for adult patients with SMA Type II. Researchers will use a special set of movement tests, called the Motor Function Measure-32 (MFM-32), to check for any changes in muscle function. By regularly carrying out these tests, the study hopes to learn more about how Spinraza affects the ability to move and do everyday activities for these patients.

Understanding how Spinraza helps adults with SMA Type II is really important. It can provide valuable information for doctors and patients about what to expect from the treatment in the long run. This information can help improve care and quality of life for people living with SMA Type II.

Key takeaways

This study monitors how Spinraza affects movement in adults with SMA Type II.
It's for adults aged 18+ who use a wheelchair and are already on Spinraza.
The study uses special tests to track changes in muscle function over time.
Your Spinraza treatment will continue as usual.
The goal is to learn more about the long-term benefits of Spinraza for these patients.
You can withdraw from the study at any time.

Who may be eligible?

This study is specifically for adults aged 18 or older who have Spinal Muscular Atrophy Type II. You must already be using a wheelchair. It's important that your SMA Type II was diagnosed through a genetic test proving a specific change in your SMN1 gene. You should have developed SMA symptoms after 6 months of age and never have been able to walk, but you could sit up without help.

To be considered, you'll also need a certain score (19 or higher) on a muscle function test (MFM-32). Doctors will also need to check that giving you the Spinraza injection into your spine is safe and possible by doing a special scan. If you're a woman who could become pregnant, you'll need to have a negative pregnancy test and agree to use reliable contraception throughout the study and for 6 months afterwards.

There are also some reasons why you couldn't take part. These include having a high risk of bleeding, kidney problems, or certain brain conditions. You also can't be pregnant or breastfeeding. You shouldn't have received any other experimental SMA treatments or gene therapy for SMA in the month before the study starts. Also, if you are an adult under someone else's legal guardianship, you wouldn't be able to join this study.

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 18 years old or older?
Do you have a genetically confirmed diagnosis of SMA Type II?
Do you currently use a wheelchair?
Could you sit without support, but never walked?
Have you *not* received any experimental SMA treatments or gene therapy recently?
If you are a woman who could become pregnant, are you able to use contraception?

Answer every question to see your result.

What does participation involve?

This study aims to monitor your progress with Spinraza. You will receive regular injections of Spinraza into your spinal fluid, as you would as part of your standard treatment. Throughout the study, you will have regular medical check-ups and assessments of your movement and muscle function, which will involve specific tests like the Motor Function Measure-32. You will also have blood and urine tests before each injection. The total duration of your participation will depend on the ongoing treatment plan and monitoring schedule.

Potential risks and benefits

The potential benefit of taking part in this study is contributing to a better understanding of how Spinraza works in adults with SMA Type II, which can help yourself and others in the future. As Spinraza is a prescribed treatment, you would continue to receive the known benefits of the medication. Potential risks are generally associated with the Spinraza treatment itself, such as side effects from the injection into the spine, which your doctor will discuss with you. Before joining, you will be given all the information you need, and you always have the right to withdraw from the study at any time without affecting your medical care.

Locations (7)

CHU de Lyon
Verified postcode
Lyon, France
CHRU de Lille
Verified postcode
Lille, France
CHU de Montpellier
Verified postcode
Montpellier, France
CHU de Toulouse
Verified postcode
Toulouse, France
Hopital de la Timone - APHM
Verified postcode
Marseille, France
CHU de Nice
Verified postcode
Nice, France
APHP
Verified postcode
Paris, France

Common questions

What is SMA Type II?

Spinal Muscular Atrophy (SMA) Type II is a genetic condition that causes muscles to become weaker over time, affecting movement and strength. People with SMA Type II can usually sit without support but never learn to walk.

What is Spinraza?

Spinraza (nusinersen) is a medicine used to treat Spinal Muscular Atrophy. It is given as an injection into the fluid around the spinal cord to help the body produce a protein needed for nerve and muscle health.

Will I get a new or different treatment?

No, this study is about monitoring patients who are already being treated with Spinraza. You will continue to receive Spinraza as your regular treatment.

How often will I need to visit the hospital?

You will have regular visits for your Spinraza injections, medical check-ups, and muscle function assessments. Your medical team will provide a detailed schedule.

Is this a trial where I might get a placebo?

No, this is a monitoring study for people already receiving Spinraza. Everyone in this study will continue to receive Spinraza treatment.