Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric patients with Spinal Muscular Atrophy

This study is investigating a new medicine, Risdiplam, for individuals with Spinal Muscular Atrophy (SMA). We aim to understand its safety and if it causes any unwanted side effects when taken by adults and children. We will also carefully check how the medicine moves through the body and what effects it has. This is an early-stage study (Phase II) to gather important information about the treatment. Participants will be closely monitored for any changes in their health, and doctors will perform various checks including physical exams, eye tests, and neurological assessments. The main goal is to make sure the medicine is well-tolerated and to learn more about how it works.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

F. Hoffmann-La Roche AG

Enrolment target

110

Start

07 Dec 2023

Spinal Muscular Atrophy (SMA)

What is this study about?

This study is about a new potential medicine called Risdiplam (also known as RO7034067) for people who have Spinal Muscular Atrophy (SMA). SMA is a condition that affects nerves and muscles, making them weaker over time.

In this study, we want to learn more about how Risdiplam works in the body and if it's safe for people to take. We'll be looking carefully at any side effects people might experience, how well their body handles the medicine, and how much of the medicine is in their blood over time. This is an 'open-label' study, which means everyone involved, including the study doctors and participants, will know that the study medicine is being given.

This kind of study, called a Phase II trial, is an important step in developing new medicines. It helps us understand if the medicine has a good balance of showing helpful signs while also being safe enough to test in larger groups of people. Our main focus is always on the safety and well-being of the participants, so we'll be carrying out many different health checks throughout the study.

Key takeaways

This study is testing a new SMA medicine called Risdiplam.
It aims to find out if Risdiplam is safe and how it works in the body.
Both adults and children with SMA can participate.
Participants will have regular health checks and close monitoring.
Your safety and well-being are the top priority throughout the study.

Who may be eligible?

This study is looking for adults aged 18 years and older who have been diagnosed with Spinal Muscular Atrophy (SMA). Children with SMA can also take part.

Both men and women are welcome to join the study.

Important note: The study team will review your medical history and current health condition to decide if this study is suitable for you. This is to ensure your safety and that the study is a good fit for your individual circumstances.

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, or are you a child with SMA?
Have you been diagnosed with Spinal Muscular Atrophy (SMA)?
Are you able to travel to study visits?
Are you willing to have regular medical checks?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be given the study medicine, Risdiplam. You will have regular visits to the clinic where doctors and nurses will monitor your health very closely. These visits will include various assessments such as physical examinations, blood tests, heart checks (ECGs), eye tests, and neurological exams to check your nerve and muscle function. We will also ask about your general well-being and any symptoms. The study team will tell you the exact schedule of visits and assessments, and the total duration of your participation, which will include taking the medication and follow-up checks.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit of joining this study is that you would receive a new medicine, Risdiplam, which might help with your SMA, although this is not guaranteed as it is still being investigated. We are also trying to learn more about SMA itself, and your participation helps us understand the condition better. Potential risks include experiencing side effects from the study medicine, or discomfort from the tests and procedures involved. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

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

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Unverified
Netherlands
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Unverified
Italy
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Unverified
France
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Unverified
Poland
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Unverified
Belgium

Common questions

What is Spinal Muscular Atrophy (SMA)?

SMA is a genetic condition that causes muscle weakness and wasting over time, because it affects the nerves that control movement.

What does 'open-label' mean?

It means that everyone involved, including the patients and the study doctors, will know that you are receiving the study medicine, Risdiplam.

What is Risdiplam?

Risdiplam is the name of the new medicine being tested in this study for people with SMA.

Will I have to pay to be in the study?

No, you will not have to pay for the study medicine or the assessments related to the study.

Can children join this study?

Yes, both adults and children with SMA can take part in this study.

How to find out more

Official trial record

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