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Aktiv men inte rekryterandePHASE2, PHASE3INTERVENTIONELL

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.

Överblick

Status
Aktiv men inte rekryterande
Fas
PHASE2, PHASE3
Sponsor
Hoffmann-La Roche
Inskrivningsmål
97
Starta
02 Jun 2022
Beräknad slutförande
28 Oct 2026
Spinal Muscular Atrophy (SMA)

Vad handlar denna studie om?

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.

Viktiga punkter

  • 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.

Vem kan vara berättigad?

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.

Kan denna studie passa dig?

Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.

  1. Do I have a confirmed genetic diagnosis of SMA?
  2. Do I currently have symptoms of SMA?
  3. Am I between 2 and 25 years old?
  4. 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)
  5. If I'm aged 5-10 and cannot walk, can I sit independently for a short time?
  6. Have I had other SMA treatments? If so, am I aware there might be a waiting time before I can join?
Svara på varje fråga för att se ditt resultat.

Vad innebär deltagande?

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.

Potentiella risker och fördelar

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.

Platser (30)

  • Boston Childrens Hospital
    Verifierat postnummer
    Boston, USA
  • Columbia University Medical Center
    Verifierat postnummer
    New York, USA
  • Sydney Barnsjukhus
    Verifierat postnummer
    Randwick, Australien
  • UZ Gent
    Verifierat postnummer
    Gent, Belgien
  • Chr de La Citadelle
    Verifierat postnummer
    Liège, Belgien
  • British Columbia Barnsjukhus
    Verifierat postnummer
    Vancouver, Kanada
  • Sjukhuset för sjuka barn
    Verifierat postnummer
    Toronto, Kanada
  • McGill University Health Centre - Glen Site
    Verifierat postnummer
    Montreal, Kanada
  • Clinical Hospital Centre Zagreb
    Verifierat postnummer
    Zagreb, Kroatien
  • Policlinico Agostino Gemelli
    Verifierat postnummer
    Rome, Italy
  • IRCCS Istituto Giannina Gaslini
    Verifierat postnummer
    Genoa, Italy
  • Fondazione IRCCS Istituto Neurologico ?Carlo Besta?
    Verifierat postnummer
    Milano, Italien

Vanliga frågor

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.

Vem kan delta i denna studie?

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.

Kommer jag att få den nya medicinen?

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

Kan jag sluta delta om jag vill?

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

Hur du får reda på mer

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