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NCT03032172 · ClinicalTrials.gov registry record · Phase 2
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
A Phase 2 study of Spinal Muscular Atrophy, sponsored by Hoffmann-La Roche.
- Completed
- Registry status
- Phase 2
- Development phase
- 174
- Enrollment target
- 20
- Study locations
NCT03032172: Completed Phase 2 study of Spinal Muscular Atrophy, sponsored by Hoffmann-La Roche.
NCT03032172 is a Phase 2 study of Spinal Muscular Atrophy that has completed, run by Hoffmann-La Roche. The registered enrollment target is 174 participants, above the 101-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (72% higher). The trial reports 20 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03032172, a Phase 2 study of Spinal Muscular Atrophy, has completed, sponsored by Hoffmann-La Roche.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 174 participants
- Enrollment target
- 20
- Study locations
Study Summary
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.
Primary Outcome
An AE was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product, regardless of causal attribution. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
Conditions Studied
Interventions
- DRUG Risdiplam
Study Locations (20)
Other
- UZ Gent - Ghent
- UZ Leuven Gasthuisberg - Leuven
- Hopital Femme Mere Enfant - Bron
- Hopital Roger Salengro - Lille
- CHRU de Montpellier, Hopital Gui de Chauliac - Montpellier
- Hôpital Necker-Enfants Malades - Paris
- Hopital des Enfants - Toulouse
- Universitätsklinikum Essen - Essen
- Universitätsklinikum Freiburg - Freiburg im Breisgau
- UMC Utrecht - Utrecht
- Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie - Warsaw
Lazio
- IRCCS Ospedale Pediatrico Bambino Gesù - Rome
- Policlinico Agostino Gemelli - Rome
California
- Stanford University Medical Center - Palo Alto
Florida
- Nemours Children's Hospital - Orlando
Massachusetts
- Boston Childrens Hospital - Boston
New York
- Columbia University Medical Center - New York
Liguria
- IRCCS Istituto Giannina Gaslini - Genoa
Lombardy
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Milan
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 174 participants |
| Start Date | 2017-03-03 |
| Est. Completion | 2025-02-07 |
| Phase | Phase 2 |
What the finished NCT03032172 record still lists
NCT03032172 is an interventional study that assigns participants to a tested intervention. The registered 174 participants enrollment target is mid-sized for trials with a published cap, above the 101-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (72% higher).
The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 1 intervention - of which Risdiplam is the first listed.
NCT03032172 names 20 study sites across 9 states, led by Other, Lazio, California.
Frequently Asked Questions
What is clinical trial NCT03032172 about?
NCT03032172 is a clinical study titled "A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy". This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO...
What is the current status of trial NCT03032172?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 174 participants. The study started on 2017-03-03. Estimated completion is 2025-02-07.
What conditions does trial NCT03032172 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy.
What interventions are being tested in trial NCT03032172?
The interventions under investigation include: Risdiplam (DRUG).
Who is sponsoring clinical trial NCT03032172?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03032172 being conducted?
This trial has 20 study locations across California, Florida, Massachusetts, New York, Lazio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Spinal Muscular Atrophy
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 NCT03032172's enrollment target sits among peer trials
174 6th of 22 higher than 17 of 22 other Spinal Muscular Atrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Spinal Muscular Atrophy 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.
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