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NCT05861999 · ClinicalTrials.gov registry record · Phase 4
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
A Phase 4 study of Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche.
- Recruiting
- Registry status
- Phase 4
- Development phase
- 28
- Enrollment target
- 19
- Study locations
NCT05861999 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 19 study locations across 10 states.
The verdict
NCT05861999, 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
- 19
- Study locations
Study Summary
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
Conditions Studied
Interventions
- DRUG Risdiplam
Study Locations (19)
Other
- Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie - Berlin
- UKGM Standort Gießen - Giessen
- Soroka Medical Center - Beersheba
- Schneider Children's Medical Center of Israel - Petah Tikva
- Sourasky MC, Dana-Dwek Children's Hospital - Tel Aviv
- Uniwersyteckie Centrum Kliniczne - Gdansk
- Instytut Pomnik Centrum Zdrowia Dziecka - Warsaw
- Sidra Medicine - Al Rayyan
- Great Ormond Street Hospital For Children - London
California
- Valley Children's Hospital - Madera
- Stanford Univ Medical Center - Palo Alto
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
Michigan
- Helen DeVos Children's Hospital at Spectrum Health - Grand Rapids
Pennsylvania
- Children'S Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 28 participants |
| Start Date | 2024-08-14 |
| Est. Completion | 2029-03-31 |
| Phase | Phase 4 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05861999
The ClinicalTrials.gov registry entry for NCT05861999 describes a study currently listed as recruiting, categorized as Phase 4. The registered enrollment target is 28 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 74-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (62% 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.
NCT05861999 reports 19 study locations spanning 10 distinct geographic areas - top geographies include Other, California, Arkansas.
Frequently Asked Questions
What is clinical trial NCT05861999 about?
NCT05861999 is a clinical study titled "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". This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be e...
What is the current status of trial NCT05861999?
This trial is currently recruiting. It is a Phase 4 study. The enrollment target is 28 participants. The study started on 2024-08-14. Estimated completion is 2029-03-31.
What conditions does trial NCT05861999 study?
This clinical trial studies the following conditions: Muscular Atrophy, Spinal.
What interventions are being tested in trial NCT05861999?
The interventions under investigation include: Risdiplam (DRUG).
Who is sponsoring clinical trial NCT05861999?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05861999 being conducted?
This trial has 19 study locations across Arkansas, California, Colorado, Florida, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.