Trials / Recruiting
RecruitingNCT05808764
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
- Status
- Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- Sponsor
- Hoffmann-La Roche · Industry
- Sex
- All
- Age
- 19 Days
- Healthy volunteers
- Not accepted
Summary
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Risdiplam | Participants will receive 0.15 mg/kg risdiplam orally once daily for 28 days. |
Timeline
- Start date
- 2024-04-26
- Primary completion
- 2026-11-30
- Completion
- 2026-11-30
- First posted
- 2023-04-11
- Last updated
- 2026-04-03
Locations
13 sites across 8 countries: United States, Belgium, Canada, Germany, Italy, Netherlands, Norway, Poland
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05808764. Inclusion in this directory is not an endorsement.