Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

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 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.

View on ClinicalTrials.gov ↗

View your shortlist →

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.

Conditions Studied

Muscular Atrophy, Spinal

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

FieldValue
Enrollment Target 28 participants
Start Date 2024-05-30
Est. Completion 2029-03-31
Phase Phase 4

Sponsor

Hoffmann-La Roche

909 total trials

Interested in This Trial?

Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT05861986

The ClinicalTrials.gov registry entry for NCT05861986 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.

NCT05861986 reports 16 study locations spanning 11 distinct geographic areas - top geographies include 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.

Explore more on PlainTrial

Muscular Atrophy, Spinal

All registered trials for this condition

Arkansas

All trials with a study site in Arkansas

Recruiting now

Find trials open to new participants near you

Rankings

Top sponsors, conditions, and phases

Read our methodology - how this data is sourced, computed, and verified.

Related

Browse all → | Rankings →
Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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.