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NCT05808764 · ClinicalTrials.gov registry record · Phase 2
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
A Phase 2 study of Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 10
- Enrollment target
- 13
- Study locations
NCT05808764 is a Phase 2 study of Muscular Atrophy, Spinal that is actively recruiting participants, run by Hoffmann-La Roche. The registered enrollment target is 10 participants, below the 75-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (87% lower). The trial reports 13 study locations across 6 states.
The verdict
NCT05808764, a Phase 2 study of Muscular Atrophy, Spinal, is actively recruiting participants, sponsored by Hoffmann-La Roche.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 10 participants
- Enrollment target
- 13
- Study locations
Study 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 Studied
Interventions
- DRUG Risdiplam
Study Locations (13)
Other
- Hopital Universitaire des Enfants Reine Fabiola - Brussels
- CHR Citadelle - Liège
- Universitatsklinikum Essen - Essen
- UMC Utrecht - Utrecht
- OUS (Oslo University Hospital), Rikshospitalet - Oslo
- Uniwersyteckie Centrum Kliniczne - Gdansk
- Instytut Pomnik - Centrum Zdrowia Dziecka - Warsaw
Emilia-Romagna
- Fondazione Serena Onlus - CENTRO CLINICO NEMO - Milano
- Fondazione Policlinico Univeristario A. Gemelli - ROMA
Illinois
- Ann and Robert H. Lurie Children Hospital of Chicago - Chicago
Michigan
- University Of Michigan - Ann Arbor
Pennsylvania
- Clinic for Special Children. - Gordonville
Ontario
- Children'S Hospital of Eastern Ontario - Ottawa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2024-04-26 |
| Est. Completion | 2026-11-30 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05808764
The ClinicalTrials.gov registry entry for NCT05808764 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 75-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (87% lower). The listed sponsor is Hoffmann-La Roche, which has 909 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Muscular Atrophy, Spinal appearing as the primary indexed condition, and to 1 intervention - of which Risdiplam is the first listed.
NCT05808764 reports 13 study locations spanning 6 distinct geographic areas - top geographies include Other, Emilia-Romagna, Illinois.
Frequently Asked Questions
What is clinical trial NCT05808764 about?
NCT05808764 is a clinical study titled "A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy". 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.
What is the current status of trial NCT05808764?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 10 participants. The study started on 2024-04-26. Estimated completion is 2026-11-30.
What conditions does trial NCT05808764 study?
This clinical trial studies the following conditions: Muscular Atrophy, Spinal.
What interventions are being tested in trial NCT05808764?
The interventions under investigation include: Risdiplam (DRUG).
Who is sponsoring clinical trial NCT05808764?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05808764 being conducted?
This trial has 13 study locations across Illinois, Michigan, Pennsylvania, Ontario, Emilia-Romagna. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Related
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