Trial · NCT05115110
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
NCT05115110, Phase 2, Phase 3, Completed, Hoffmann-La Roche
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy. Trials, record NCT05115110. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05115110
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
- NCT number
- NCT05115110
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 97 participants, actual
- Lead sponsor
- Hoffmann-La Roche
- Conditions
- Spinal Muscular Atrophy (SMA)
- Interventions
- RO7204239, Placebo, Risdiplam
- First posted
- 2021-11-10
- Last updated
- 2026-11-04
Source ClinicalTrials.gov
Registered outcomes34
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 1 - Percentage of participants with adverse events (AEs) | Up to 4.5 years | |
| Primary | Part 1 - Incidence of relevant echocardiographic parameter z scores > 2 | Up to 4.5 years | |
| Primary | Part 1 - Serum concentration of RO7204239 | Through Week 96 | |
| Primary | Part 1 - Time to maximum serum concentration (Cmax) of RO7204239 | Through Week 96 | |
| Primary | Part 1 - Area under the curve (AUC) of RO7204239 | Through Week 96 | |
| Primary | Part 1 - Trough concentration (Ctrough) of RO7204239 | Through Week 96 | |
| Primary | Part 1 - Plasma concentration of risdiplam | Week 21 | |
| Primary | Part 1 - Plasma concentration of risdiplam metabolite (M1) | Week 21 | |
| Primary | Part 1 - Cmax of risdiplam | Week 21 | |
| Primary | Part 1 - AUC of risdiplam | Week 21 |
1 to 10 of 34Source ClinicalTrials.gov
See all 34 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05115110
GET https://conductscience.org/api/trials/v1/NCT05115110/registered?page=2