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NCT03779334 · ClinicalTrials.gov registry record · Phase 2
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
A Phase 2 study of Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche.
- Active
- Registry status
- Phase 2
- Development phase
- 26
- Enrollment target
- 7
- Study locations
NCT03779334 is a Phase 2 study of Muscular Atrophy, Spinal that is active but no longer recruiting, run by Hoffmann-La Roche. The registered enrollment target is 26 participants, below the 74-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (65% lower). The trial reports 7 study locations across 5 states.
The verdict
NCT03779334, a Phase 2 study of Muscular Atrophy, Spinal, is active but no longer recruiting, sponsored by Hoffmann-La Roche.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 26 participants
- Enrollment target
- 7
- Study locations
Study Summary
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
Conditions Studied
Interventions
- DRUG Risdiplam
Study Locations (7)
Other
- Chr de La Citadelle - Liège
- Szpital Gdanskiego Uniwersytetu Medycznego - Gda?sk
- Kaohsiung Medical University Chung-Ho Hospital - Kaohsiung City
Florida
- Nemours Children's Hospital - Orlando
New South Wales
- Sydney Children's Hospital - Randwick
São Paulo
- Hospital das Clinicas - FMUSP_X - São Paulo
Moscow Oblast
- Russian Children Neuromuscular Center of Veltischev - Moscow
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2019-08-07 |
| Est. Completion | 2027-02-28 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03779334
The ClinicalTrials.gov registry entry for NCT03779334 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 26 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 74-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (65% lower). The listed sponsor is Hoffmann-La Roche, which has 909 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Muscular Atrophy, Spinal appearing as the primary indexed condition, and to 1 intervention - of which Risdiplam is the first listed.
NCT03779334 reports 7 study locations spanning 5 distinct geographic areas - top geographies include Other, Florida, New South Wales.
Frequently Asked Questions
What is clinical trial NCT03779334 about?
NCT03779334 is a clinical study titled "A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy". A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
What is the current status of trial NCT03779334?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 26 participants. The study started on 2019-08-07. Estimated completion is 2027-02-28.
What conditions does trial NCT03779334 study?
This clinical trial studies the following conditions: Muscular Atrophy, Spinal.
What interventions are being tested in trial NCT03779334?
The interventions under investigation include: Risdiplam (DRUG).
Who is sponsoring clinical trial NCT03779334?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03779334 being conducted?
This trial has 7 study locations across Florida, New South Wales, São Paulo, Moscow Oblast. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Read our methodology - how this data is sourced, computed, and verified.
Related
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