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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: Recruiting Phase 2 study of Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche.
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. According to ClinicalTrials.gov, the official US trial registry.
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 |
What NCT05808764 shows while recruiting
NCT05808764 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 75-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (87% lower).
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 lists 13 locations in 6 states (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
Similar trials for Muscular Atrophy, Spinal
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05808764's enrollment target sits among peer trials
10 16th of 15 the lowest of 15 other Muscular Atrophy, Spinal trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Muscular Atrophy, Spinal trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
ACTIVE NOT RECRUITING · Phase 2
-
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
COMPLETED · Phase 2
-
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
COMPLETED · Phase 2
-
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
RECRUITING · Phase 3
-
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
RECRUITING · Phase 4
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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
RECRUITING · Phase 4
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