Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Public ClinicalTrials.gov record NCT02913482. 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.
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, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy
Study identification
- NCT ID
- NCT02913482
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Enrollment
- 62 participants
Conditions and interventions
Conditions
Interventions
- Risdiplam Drug
Drug
Eligibility (public fields only)
- Age range
- 1 Month to 7 Months
- 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
- Dec 22, 2016
- Primary completion
- Nov 13, 2019
- Completion
- Dec 21, 2023
- Last update posted
- Aug 6, 2024
2016 – 2023
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Stanford University Medical Center | Palo Alto | California | 94304 | — |
| Ann and Robert H. Lurie Children Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Boston Childrens Hospital | Boston | Massachusetts | 02115 | — |
| 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 28 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02913482, 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 Aug 6, 2024 · Synced May 16, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02913482 live on ClinicalTrials.gov.