Active not recruitingPHASE2, PHASE3INTERVENTIONAL

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

This study explores a new treatment combination for Spinal Muscular Atrophy (SMA), a condition that affects muscle movement and strength. Researchers are testing two medicines together: RO7204239 and risdiplam. Risdiplam helps the body make more of a protein essential for nerve cells that control muscles. RO7204239 is designed to help muscles grow stronger. The study wants to find out if this combination is safe and effective in improving muscle function in people with SMA. It will involve different age groups, from 2 to 25 years old, including those who can walk and those who cannot.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

Hoffmann-La Roche

Enrolment target

259

Start

02 Jun 2022

Estimated completion

27 Feb 2029

Spinal Muscular Atrophy (SMA)

What is this study about?

Spinal Muscular Atrophy (SMA) is a condition that affects how well your muscles work. This happens because certain nerve cells, called motor neurons, don't work properly, leading to muscle weakness and problems with movement. Currently, risdiplam is a medicine that helps your body produce more of an important protein that these motor neurons need to survive, which can help improve muscle function.

This study is looking at an additional medicine, RO7204239, to see if combining it with risdiplam can lead to even better results. RO7204239 works in a different way; it aims to help your muscles grow bigger and stronger. Imagine it like this: risdiplam helps keep your muscle-controlling nerves healthy, and RO7204239 helps the muscles themselves become more powerful. By using both together, the hope is to further improve movement and overall well-being for people with SMA.

The study will happen in two main parts. The first part will help researchers figure out the right amount of the new medicine (RO7204239) to give. The second part will then test this combination in a larger group of people with SMA, aged between 2 and 25 years, who can walk. The main goal is to check how safe this new combination is and how well it works to help patients.

Key takeaways

This study explores a new combination treatment for Spinal Muscular Atrophy (SMA).
It combines risdiplam (already used for SMA) with a new medicine called RO7204239.
RO7204239 is designed to help muscles grow and get stronger.
The study aims to check how safe and effective this new combination is.
It includes participants aged 2 to 25 with SMA, both those who can walk and those who cannot.
Careful monitoring and assessments will be part of taking part.

Who may be eligible?

To join this study, people must have a confirmed genetic diagnosis of Spinal Muscular Atrophy (SMA) and show symptoms of the condition. The study is open to both boys and girls.

For the first part of the study, depending on the group, participants can be between 2 and 10 years old. For the second part, participants can be between 2 and 25 years old and must be able to walk unassisted (without crutches, walkers, or any help) for about 10 meters in 30 seconds or less. A specific group in the first part will include young people aged 5-10 who are not able to walk but can sit up on their own for a short time.

People who have received other SMA treatments before might be able to join, but there are specific waiting periods mentioned for each previous treatment. For example, if you've had gene therapy (like onasemnogene abeparvovec), you would need to wait at least 90 days. If you are currently taking risdiplam, you might be able to switch to the study's version of the medicine.

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 I have a confirmed genetic diagnosis of SMA?
Do I currently have symptoms of SMA?
Am I between 2 and 25 years old?
If I'm aged 2-25, can I walk 10 metres in 30 seconds or less without help? (This is important for the main part of the study)
If I'm aged 5-10 and cannot walk, can I sit independently for a short time?
Have I had other SMA treatments? If so, am I aware there might be a waiting time before I can join?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve regular visits to the clinic for assessments and to receive the study medicines. You would either receive RO7204239 combined with risdiplam or a placebo (a dummy medicine) combined with risdiplam, depending on the study group you are in. Doctors and nurses will monitor your health closely, perform examinations, and take blood samples. They will also assess your muscle strength and movement abilities using specific tests. The exact number of visits and the total length of your participation would be discussed in detail by the study team.

Potential risks and benefits

There are potential benefits to joining a clinical trial, such as potentially gaining access to a new treatment that might improve your condition. However, there are also risks, as new medicines can have side effects that are not yet fully known. The study team will continuously monitor your health for any side effects. You will be given full information about all known and potential risks before you decide to take part. Remember, joining this study is completely voluntary, and you are free to withdraw at any time without affecting your usual medical care.

Locations (35)

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

Nemours Children's Hospital
Verified postcode
Orlando, United States
Boston Childrens Hospital
Verified postcode
Boston, United States
Columbia University Medical Center
Verified postcode
New York, United States
Neurology & Neuromuscular Care Center
Verified postcode
Flower Mound, United States
Sydney Children's Hospital
Verified postcode
Randwick, Australia
UZ Gent
Verified postcode
Ghent, Belgium
Chr de La Citadelle
Verified postcode
Liège, Belgium
British Columbia Children's Hospital
Verified postcode
Vancouver, Canada
The Hospital for Sick Children
Verified postcode
Toronto, Canada
McGill University Health Centre - Glen Site
Verified postcode
Montreal, Canada
Clinical Hospital Centre Zagreb
Verified postcode
Zagreb, Croatia
Ospedale Pediatrico Bambino Gesù
Verified postcode
Rome, Italy

Common questions

What is SMA?

SMA stands for Spinal Muscular Atrophy. It's a condition that affects nerve cells that control muscles, leading to muscle weakness and problems with movement.

What are these medicines, RO7204239 and risdiplam?

Risdiplam helps your body make a protein important for nerves that control muscles. RO7204239 is a new medicine designed to help muscles grow stronger. This study is testing them together.

Who can join this study?

The study is for people with SMA, aged 2-25 years, who have a confirmed genetic diagnosis. Specific age and walking abilities apply to different parts of the study.

Will I get the new medicine?

Participants will either receive RO7204239 combined with risdiplam or a placebo (a dummy medicine) combined with risdiplam. This helps researchers compare the effects.

Can I stop participating if I want to?

Yes, you can leave the study at any time, for any reason, without it affecting your medical care.