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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: Active Phase 2 study of Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche.
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. According to ClinicalTrials.gov, the official US trial registry.
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).
Primary Outcome
The Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item ach
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 |
What the registry record for NCT03779334 still lists
NCT03779334 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 26 participants, a relatively small participant target, below the 74-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (65% lower).
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 lists 7 locations in 5 states (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
Similar trials for Muscular Atrophy, Spinal
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03779334's enrollment target sits among peer trials
26 12th of 15 higher than 3 of 15 other Muscular Atrophy, Spinal trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Muscular Atrophy, Spinal 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
-
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
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-
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
COMPLETED · Phase 2
-
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
COMPLETED · Phase 2
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A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
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-
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
RECRUITING · Phase 4
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