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Completed Phase 2 Interventional Results available

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

ClinicalTrials.gov ID: NCT02908685

Public ClinicalTrials.gov record NCT02908685. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Jul 22, 2026, 11:47 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients

Study identification

NCT ID
NCT02908685
Recruitment status
Completed
Study type
Interventional
Phase
Phase 2
Lead sponsor
Hoffmann-La Roche
Industry
Enrollment
231 participants

Conditions and interventions

Interventions

  • Placebo Drug
  • Risdiplam Drug

Drug

Eligibility (public fields only)

Age range
2 Years to 25 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Oct 18, 2016
Primary completion
Sep 5, 2019
Completion
Oct 1, 2023
Last update posted
Apr 23, 2024

2016 – 2023

United States locations

U.S. sites
2
U.S. states
2
U.S. cities
2
Facility City State ZIP Site status
Stanford University Medical Center Palo Alto California 94304
Columbia University Medical Center; The Neurological Institute of New York New York New York 10032

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 43 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT02908685, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Apr 23, 2024 · Synced Jul 22, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT02908685 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →