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NCT05861986 · ClinicalTrials.gov registry record · Phase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
A Phase 4 study of Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche.
- Recruiting
- Registry status
- Phase 4
- Development phase
- 28
- Enrollment target
- 16
- Study locations
NCT05861986: Recruiting Phase 4 study of Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche.
NCT05861986 is a Phase 4 study of Muscular Atrophy, Spinal that is actively recruiting participants, run by Hoffmann-La Roche. The registered enrollment target is 28 participants, below the 74-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (62% lower). The trial reports 16 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05861986, a Phase 4 study of Muscular Atrophy, Spinal, is actively recruiting participants, sponsored by Hoffmann-La Roche.
- RECRUITING
- Registry status
- Phase 4
- Development phase
- 28 participants
- Enrollment target
- 16
- Study locations
Study Summary
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
Primary Outcome
The BSID-III is a standardized assessment commonly used to evaluate developmental functioning of infants and young children between 1 month and 42 months of age. The gross motor scale measures the movement of the limbs and torso. Items assess static positioning (e.g., sitting, standing); dynamic movement, including locomotion and coordination; balance; and motor planning. The gross motor scale consists of 72 items scored at 0 (unable to perform) or 1 (criteria for item achieved). A higher raw sc
Conditions Studied
Interventions
- DRUG Risdiplam
Study Locations (16)
Other
- Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie - Berlin
- UKGM Standort Gießen - Giessen
- Uniwersyteckie Centrum Kliniczne - Uniwersyteckie Centrum Kliniczne
- Instytut Pomnik Centrum Zdrowia Dziecka - Warsaw
- Great Ormond Street Hospital For Children - London
Texas
- The University of Texas Southwestern Medical Center at Dallas - Dallas
- Cook Children's Jane and John Justin Neurosciences Center - Fort Worth
Arkansas
- University of Arkansas for Medical Sciences - Little Rock
Colorado
- Children's Hospital of Colorado - Aurora
Florida
- University of Florida Pediatrics - Gainesville
Georgia
- Children's Healthcare of Atlanta Center for Advanced Pediatrics - Atlanta
Illinois
- Ann and Robert H. Lurie Children Hospital of Chicago - Chicago
Michigan
- Helen DeVos Children's Hospital at Spectrum Health - Grand Rapids
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 28 participants |
| Start Date | 2024-05-30 |
| Est. Completion | 2029-03-31 |
| Phase | Phase 4 |
What NCT05861986 shows while recruiting
NCT05861986 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 28 participants, a relatively small participant target, below the 74-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (62% 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.
NCT05861986 lists 16 locations in 11 states (Other, Texas, Arkansas).
Frequently Asked Questions
What is clinical trial NCT05861986 about?
NCT05861986 is a clinical study titled "A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy". This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants...
What is the current status of trial NCT05861986?
This trial is currently recruiting. It is a Phase 4 study. The enrollment target is 28 participants. The study started on 2024-05-30. Estimated completion is 2029-03-31.
What conditions does trial NCT05861986 study?
This clinical trial studies the following conditions: Muscular Atrophy, Spinal.
What interventions are being tested in trial NCT05861986?
The interventions under investigation include: Risdiplam (DRUG).
Who is sponsoring clinical trial NCT05861986?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05861986 being conducted?
This trial has 16 study locations across Arkansas, Colorado, Florida, Georgia, Illinois. 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 NCT05861986's enrollment target sits among peer trials
28 10th of 15 higher than 5 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 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
-
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
RECRUITING · 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 to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
RECRUITING
-
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
RECRUITING · Phase 1
-
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
ACTIVE NOT RECRUITING · Phase 2
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