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A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
NCT05115110 · View on ClinicalTrials.gov ↗
Study Summary
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is an investigational anti-myostatin antibody that is designed to target myostatin. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength. RO7204239 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and clinical outcomes for people living with SMA. This trial will study the safety and efficacy of RO7204239 in combination with risdiplam in patients with spinal muscular atrophy (SMA). The trial has two parts; Part 1 is the dose-finding part in SMA patients that are either ambulant (aged 2-10 years) or non-ambulant (aged 5-10 years) within separate cohorts, and Part 2 is the pivotal part in SMA patients aged 2-25 years that are ambulant.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Risdiplam
- DRUG RO7204239
Study Locations (20)
Other
- UZ Gent — Ghent
- Chr de La Citadelle — Liège
- Clinical Hospital Centre Zagreb — Zagreb
- Kobe University Hospital — Hyōgo
- Kagoshima University Hospital — Kagoshima
- National Center for Global Health and Medicine — Tokyo
Lazio
- Ospedale Pediatrico Bambino Gesù — Rome
- Policlinico Agostino Gemelli — Rome
Lombardy
- Fondazione IRCCS Istituto Neurologico ?Carlo Besta? — Milan
- Asst Grande Ospedale Metropolitano Niguarda — Milan
Florida
- Nemours Children's Hospital — Orlando
Massachusetts
- Boston Childrens Hospital — Boston
New York
- Columbia University Medical Center — New York
Texas
- Neurology & Neuromuscular Care Center — Flower Mound
New South Wales
- Sydney Children's Hospital — Randwick
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 259 participants |
| Start Date | 2022-06-02 |
| Est. Completion | 2029-02-27 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05115110
The ClinicalTrials.gov registry entry for NCT05115110 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 259 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Hoffmann-La Roche, which has 758 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Spinal Muscular Atrophy (SMA) appearing as the primary indexed condition, and to 3 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05115110 reports 20 study locations spanning 13 distinct geographic areas — top geographies include Other, Lazio, Lombardy. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05115110 about?
NCT05115110 is a clinical study titled "A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy". Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is an i...
What is the current status of trial NCT05115110?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 259 participants. The study started on 2022-06-02. Estimated completion is 2029-02-27.
What conditions does trial NCT05115110 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy (SMA). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05115110?
The interventions under investigation include: Placebo (DRUG), Risdiplam (DRUG), RO7204239 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05115110?
This trial is sponsored by Hoffmann-La Roche, which has 758 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05115110 being conducted?
This trial has 20 study locations across Florida, Massachusetts, New York, Texas, New South Wales. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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