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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
NCT02908685 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02908685
The ClinicalTrials.gov registry entry for NCT02908685 describes a study currently listed as completed. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 231 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Hoffmann-La Roche, which has 758 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02908685 reports 20 study locations spanning 8 distinct geographic areas — top geographies include Other, Lazio, California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT02908685?
The interventions under investigation include: Placebo (DRUG), Risdiplam (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT02908685?
This trial is sponsored by Hoffmann-La Roche, which has 758 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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