A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Public ClinicalTrials.gov record NCT05115110. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy
Study identification
- NCT ID
- NCT05115110
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Enrollment
- 259 participants
Conditions and interventions
Conditions
Interventions
- RO7204239 Drug
- Placebo Drug
- Risdiplam Drug
Drug
Eligibility (public fields only)
- Age range
- 2 Years to 25 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 1, 2022
- Primary completion
- Oct 27, 2026
- Completion
- Oct 27, 2026
- Last update posted
- Jun 10, 2026
2022 – 2026
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Nemours Children's Hospital | Orlando | Florida | 32827 | — |
| Boston Childrens Hospital | Boston | Massachusetts | 02115 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| Neurology & Neuromuscular Care Center | Flower Mound | Texas | 75028 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 31 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05115110, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 10, 2026 · Synced Jun 26, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05115110 live on ClinicalTrials.gov.