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Study on the Safety and Efficacy of RO7204239 and Risdiplam for Patients with Spinal Muscular Atrophy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Spinal Muscular Atrophy (SMA), a genetic disorder that affects the muscles used for movement. The study is testing a combination of two treatments: RO7204239 and Risdiplam. RO7204239 is a type of medication known as a monoclonal antibody, which is designed to target specific proteins in the body. Risdiplam is an oral solution that helps increase the production of a protein important for muscle function.

The purpose of this study is to evaluate the safety and effectiveness of these treatments when used together. Participants in the study will receive either the combination of RO7204239 and Risdiplam or a placebo, which is a substance with no active medication. The study will be conducted in two parts. In the first part, the focus will be on understanding how the body processes these medications and their safety. The second part will assess how well the combination works in improving muscle function in people with Spinal Muscular Atrophy.

Throughout the study, participants will undergo various assessments to monitor their health and the effects of the treatment. These assessments may include regular check-ups, blood tests, and imaging tests like MRI to evaluate muscle changes. The study aims to provide valuable information on the potential benefits of combining RO7204239 and Risdiplam for individuals living with Spinal Muscular Atrophy.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on age and genetic diagnosis of spinal muscular atrophy (SMA).

    Participants must have received previous SMA therapies at least 90 days prior to joining.

  2. Step 2

    Part 1: initial treatment phase

    Participants receive a combination of RO7204239 and risdiplam.

    RO7204239 is administered as a subcutaneous injection, while risdiplam is taken orally as a solution.

    The focus is on evaluating safety, how the body processes the drugs, and the immune response.

  3. Step 3

    Monitoring and assessments

    Regular monitoring includes checking vital signs, physical exams, and laboratory tests.

    Participants undergo assessments such as ECGs and echocardiograms to track heart health.

    Blood samples are taken to measure drug levels and immune response.

  4. Step 4

    Part 2: extended treatment phase

    Participants continue receiving the combination treatment for an extended period.

    The focus shifts to evaluating the effectiveness of the treatment in improving motor function and muscle strength.

  5. Step 5

    Final evaluations

    At the end of the study, final assessments are conducted to measure changes in motor function and muscle mass.

    The study aims to determine the overall impact of the treatment on SMA symptoms.

Who can join the trial?

8 criteria

  • Participants must be between the ages of 5 and 10 years for Part 1 Cohorts A, B, and D, between 2 and 4 years for Part 1 Cohort C, and between 2 and 25 years for Part 2.
  • Participants must have a confirmed genetic diagnosis of **5q-autosomal recessive Spinal Muscular Atrophy (SMA)**, which is a specific type of genetic condition.
  • Participants who have received previous treatments for SMA can join if:
    • They received **onasemnogene abeparvovec** at least 90 days before the screening. They should have stopped taking steroids before starting **risdiplam** and have normal liver function, blood clotting, platelet levels, and heart muscle enzyme levels 90 days after the treatment or at least 1 month after stopping steroids, whichever is later.
    • They received their last dose of **nusinersen** at least 90 days before the screening.
    • They switch from **risdiplam** to the study medication provided by the research site.
    • For Part 1 Cohorts A, B, and C and Part 2, participants must be **ambulant**, meaning they can walk or run without help from devices like canes or walkers, and can complete a 10-meter walk or run in 30 seconds or less.
    • For Part 1 Cohort D, participants must be able to sit without using their arms for support, as measured by specific tests.
    • For Part 1 Cohort D, participants must be able to lift a plastic cup with a 200g weight to their mouth, using both hands if needed, as measured by a specific test.

Who cannot join the trial?

9 criteria

  • Patients who have any other serious health conditions that could interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent surgery or are planning to have surgery during the study period.
  • Patients who have allergies to any of the study medications.
  • Patients who are unable to follow the study procedures or attend the required visits.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who have received certain treatments for Spinal Muscular Atrophy (SMA) recently.
  • Patients who have any condition that, in the opinion of the study doctor, makes them unsuitable for the study.
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Investigated drugs

  • RO7204239

    is an investigational medication being studied for its potential to help patients with spinal muscular atrophy. It is being tested to see how safe it is, how well it is tolerated by patients, and how it behaves in the body when used together with another medication called risdiplam. Researchers are also looking at how the body’s immune system responds to this medication and how it affects the disease.

  • Risdiplam

    is a medication that is already used to treat spinal muscular atrophy. In this study, it is being combined with RO7204239 to see if the combination is more effective than using risdiplam alone. The study aims to understand how risdiplam works in the body when used with RO7204239 and to evaluate the overall effectiveness of this combination in treating the disease.

What is already known about the treatment

  • Risdiplam

    Risdiplam is administered orally and is currently being studied in combination with another medication for the treatment of Spinal Muscular Atrophy (SMA). It is an approved medication in the medical literature for SMA, known for its ability to increase the production of a protein essential for muscle function. Risdiplam works by modifying the splicing of a specific gene to increase the levels of the survival motor neuron (SMN) protein. It is classified as a small molecule splicing modifier.

  • RO7204239

    RO7204239 is administered in combination with risdiplam and is currently under investigation in clinical trials for its safety, tolerability, and efficacy in treating Spinal Muscular Atrophy (SMA). The medication is still in the experimental phase and is not yet widely recognized in medical literature. It is being studied for its potential to enhance the effects of risdiplam, although its exact mechanism of action at the molecular level is still being explored. RO7204239 is classified as an investigational drug.

Investigated diseases

Spinal Muscular Atrophy (SMA) – Spinal Muscular Atrophy is a genetic disorder characterized by weakness and wasting of the muscles used for movement. It is caused by a loss of specialized nerve cells, called motor neurons, in the spinal cord and the part of the brain connected to the spinal cord. This leads to progressive muscle weakness and atrophy, primarily affecting the muscles closest to the center of the body. The severity of the disease can vary, with some individuals experiencing significant physical limitations. SMA is typically classified into different types based on the age of onset and the highest physical milestone achieved. Over time, individuals with SMA may experience difficulties with activities such as walking, sitting, and even breathing.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2023-506761-65-00Protocol codeBN42644Estimated enrolment156 patientsSponsorF. Hoffmann-La Roche AG

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