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NCT02913482 · ClinicalTrials.gov registry record · Phase 2
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
A Phase 2 study, sponsored by Hoffmann-La Roche.
- Completed
- Registry status
- Phase 2
- Development phase
- 62
- Enrollment target
NCT02913482: Completed Phase 2 study, sponsored by Hoffmann-La Roche.
NCT02913482 is a Phase 2 study that has completed, run by Hoffmann-La Roche. The registered enrollment target is 62 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (53% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02913482, a Phase 2 study, has completed, sponsored by Hoffmann-La Roche.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 62 participants
- Enrollment target
Study Summary
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate Risdiplam (RO7034067) for 24-months at the dose selected in Part 1.
Primary Outcome
All safety, tolerability, PK and PD data available up to the clinical cut-off date of 5 January 2018, plus data that became available prior to the database snapshot on 6 February 2018 were included in the Internal Monitoring Committee (IMC) review. The IMC was responsible for selecting the dose for Part 2 of the study (pivotal dose). An external Independent Data Monitoring Committee (iDMC) reviewed data from Part 1 and confirmed the dose-selection decision of the IMC. The dose for Part 2 selecte
Interventions
- DRUG Risdiplam
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 62 participants |
| Start Date | 2016-12-23 |
| Est. Completion | 2023-12-22 |
| Phase | Phase 2 |
What the finished NCT02913482 record still lists
NCT02913482 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 62 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (53% lower).
The record links to 0 conditions, and to 1 intervention - of which Risdiplam is the first listed.
NCT02913482 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02913482 about?
NCT02913482 is a clinical study titled "Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy". Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a con...
What is the current status of trial NCT02913482?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 62 participants. The study started on 2016-12-23. Estimated completion is 2023-12-22.
What interventions are being tested in trial NCT02913482?
The interventions under investigation include: Risdiplam (DRUG).
Who is sponsoring clinical trial NCT02913482?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02913482's enrollment target sits among peer trials
62 1016th of 2000 higher than 975 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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