A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Public ClinicalTrials.gov record NCT05808764. 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 Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Study identification
- NCT ID
- NCT05808764
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Enrollment
- 10 participants
Conditions and interventions
Conditions
Interventions
- Risdiplam Drug
Drug
Eligibility (public fields only)
- Age range
- Up to 19 Days
- 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
- Apr 25, 2024
- Primary completion
- Nov 29, 2026
- Completion
- Nov 29, 2026
- Last update posted
- May 3, 2026
2024 – 2026
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Ann and Robert H. Lurie Children Hospital of Chicago | Chicago | Illinois | 60611 | Recruiting |
| University Of Michigan | Ann Arbor | Michigan | 48109 | Recruiting |
| Clinic for Special Children. | Gordonville | Pennsylvania | 17529 | Recruiting |
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 10 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05808764, 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 May 3, 2026 · Synced May 19, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05808764 live on ClinicalTrials.gov.