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NCT02908685 · ClinicalTrials.gov registry record · Phase 2
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
A Phase 2 study of Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche.
- Completed
- Registry status
- Phase 2
- Development phase
- 231
- Enrollment target
- 20
- Study locations
NCT02908685: Completed Phase 2 study of Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche.
NCT02908685 is a Phase 2 study of Muscular Atrophy, Spinal that has completed, run by Hoffmann-La Roche. The registered enrollment target is 231 participants, above the 60-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (285% higher). The trial reports 20 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02908685, a Phase 2 study of Muscular Atrophy, Spinal, has completed, sponsored by Hoffmann-La Roche.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 231 participants
- Enrollment target
- 20
- Study locations
Study Summary
Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1) of Risdiplam for 12 weeks and a confirmatory part (Part 2) of Risdiplam for 24 months.
Primary Outcome
The Internal Monitoring Committee (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 selected by the IMC was a dose that: 1.Was judged to be safe and well-tolerated, based on all available safety data from Part 1 and as confirmed by the iDMC; 2. Resulted in an exposure at steady-state below the exposure cap
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Risdiplam
Study Locations (20)
Other
- UZ Gent - Ghent
- UZ Leuven Gasthuisberg - Leuven
- Chr de La Citadelle - Liège
- Peking University First Hospital - Beijing
- Children's Hospital of Fudan University - Shanghai
- Clinical Medical Center Zagreb; University Hospital Rebro Department of Paediatrics - Zagreb
- Hopital Femme Mere Enfant; Medecine Physique et Readaptation Pediatrique ? L?ESCALE - Bron
- Hopital Roger Salengro - Lille
- CHU de Nantes - Hotel Dieu - Nantes
- Hôpital Necker-Enfants Malades; Service de neuropédiatrie - Paris
- Hopital Armand Trousseau - Paris
- Universitätsklinikum Freiburg; Klinik für Neuropädiatrie und Muskelerkrankungen - Freiburg im Breisgau
Lazio
- IRCCS Ospedale Pediatrico Bambino Gesù; U.O. Malattie Neuromuscolari e Neurodegenerative - Rome
- Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile - Rome
California
- Stanford University Medical Center - Palo Alto
New York
- Columbia University Medical Center; The Neurological Institute of New York - New York
Rio de Janeiro
- Instituto de Puericultura E Pediatria Martagão Gesteira - Rio de Janeiro
Alberta
- Alberta Children's Hospital Division of Pediatric Neurology - Calgary
Ontario
- London Health Sciences Centre; Children's Hospital; Pediatrics - London
Quebec
- McGill University Health Centre - Glen Site - Montreal
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 231 participants |
| Start Date | 2016-10-19 |
| Est. Completion | 2023-10-02 |
| Phase | Phase 2 |
What the finished NCT02908685 record still lists
NCT02908685 is an interventional study that assigns participants to a tested intervention. The registered 231 participants enrollment target is mid-sized for trials with a published cap, above the 60-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (285% higher).
The record links to 1 condition, with Muscular Atrophy, Spinal appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT02908685 names 20 study sites across 8 states, led by Other, Lazio, California.
Frequently Asked Questions
What is clinical trial NCT02908685 about?
NCT02908685 is a clinical study titled "A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants". Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1...
What is the current status of trial NCT02908685?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 231 participants. The study started on 2016-10-19. Estimated completion is 2023-10-02.
What conditions does trial NCT02908685 study?
This clinical trial studies the following conditions: Muscular Atrophy, Spinal.
What interventions are being tested in trial NCT02908685?
The interventions under investigation include: Placebo (DRUG), Risdiplam (DRUG).
Who is sponsoring clinical trial NCT02908685?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02908685 being conducted?
This trial has 20 study locations across California, New York, Rio de Janeiro, Alberta, Ontario. 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 NCT02908685's enrollment target sits among peer trials
231 2nd of 15 the highest 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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An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
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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 as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
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