Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Longitudinal study of clinical outcome measures in adult patients with Spinal Muscular Atrophy (SMA) treated with Risdiplam.

This important study is looking into how a medicine called Risdiplam, also known as Evrysdi, affects adults who have a condition called Spinal Muscular Atrophy (SMA) type 2 or 3. The main goal is to find out if this medicine helps improve how people feel and function in their daily lives over a period of two years. Researchers will also be checking the medicine's safety and if it causes any side effects. This type of study, called a Phase III trial, is a crucial step to confirm how well a new treatment works and ensures it's safe for wider use. It's designed to bring us closer to understanding the best ways to manage SMA.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

UZ Leuven

Enrolment target

Start

28 Nov 2022

Spinal Muscular Atrophy (SMA)

What is this study about?

This study is about a medicine called Risdiplam, which you might know as Evrysdi. It’s for adults who have a condition called Spinal Muscular Atrophy (SMA), specifically types 2 or 3, or who have up to 4 copies of a gene called SMN2. SMA is a genetic condition that affects the nerves that control muscle movement.

The main aim of this research is to understand how Risdiplam affects the daily lives and physical abilities of these adult patients over a two-year period. Researchers will be looking at things like how strong muscles are, how well people can move, and other measures of their overall health and function. They'll compare these measures at the beginning of the study to how they are after 24 months of treatment.

Alongside checking how people's abilities change, the study will also carefully look at how safe Risdiplam is and if people can take it without too many problems. This includes checking for any side effects and looking at certain markers in the blood that can tell us about nerve health. This kind of study is crucial because it helps doctors and patients understand what to expect from treatments for SMA.

Key takeaways

The study examines Risdiplam (Evrysdi) for adults with SMA Type 2 or 3.
It aims to see if the medicine improves daily life and physical function over two years.
Safety and side effects of Risdiplam will also be carefully checked.
Participation requires regular clinic visits and taking the study medication.
You can stop participating in the study at any time.

Who may be eligible?

To be part of this study, you need to be an adult, 18 years old or older. There isn't an upper age limit, so people of all adult ages are welcome to be considered.

Both men and women can take part in this research. The study is specifically for adults who have been diagnosed with Spinal Muscular Atrophy (SMA) of type 2 or type 3. It also includes people who have a certain genetic make-up related to SMA, specifically those with up to 4 copies of the SMN2 gene.

Basically, if you're an adult living with SMA Type 2 or 3, or you meet the SMN2 gene copy criteria, you might be able to join. The research team will carefully check to make sure the study is a good fit for you and that it's safe for you to participate.

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 of age or older?
Do you have a diagnosis of Spinal Muscular Atrophy (SMA) Type 2 or Type 3?
Do you have up to 4 copies of the SMN2 gene?
Are you able to commit to taking the medicine and attending regular check-ups for two years?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be taking Risdiplam (Evrysdi) as a liquid medicine. The study will last for a total of two years, during which time you'll have regular visits to the clinic. These visits will involve various tests to see how you're doing, such as checking your muscle strength, movement, and overall physical abilities. You might also have blood tests to look at certain markers. All these assessments help the doctors understand how the medicine is working and ensure your safety. The research team will explain everything in detail, including how often you'll need to come in and what each visit will involve.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit of this study is that Risdiplam might improve your daily life and physical abilities. It also contributes valuable information that could help others with SMA in the future. However, like all medicines, Risdiplam can have side effects, and some might be serious. The study team will monitor you closely for any unwanted effects. You'll always have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
Belgium

Common questions

What condition is this study for?

This study is for adults with Spinal Muscular Atrophy (SMA) type 2 or 3, or those with up to 4 copies of the SMN2 gene.

What is the medicine being tested called?

The medicine being tested is called Risdiplam, also known as Evrysdi.

How long will the study last if I take part?

If you take part, the study will last for a total of two years.

What will doctors be looking for in this study?

Doctors will be looking to see if Risdiplam improves your daily life and physical abilities, and checking its safety.

Can I stop participating in the study if I change my mind?

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