A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

ClinicalTrials.gov ID: NCT05861986

Public ClinicalTrials.gov record NCT05861986. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced May 8, 2026, 10:45 PM EDT

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Official title

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy

Study identification

NCT ID: NCT05861986
Recruitment status: Recruiting
Study type: Interventional
Phase: Phase 4
Lead sponsor: Hoffmann-La Roche; Industry
Enrollment: 28 participants

Conditions and interventions

Conditions

Muscular Atrophy, Spinal

Interventions

Risdiplam Drug

Drug

Eligibility (public fields only)

Age range: 3 Months to 24 Months
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: May 29, 2024
Primary completion: Mar 30, 2028
Completion: Mar 30, 2029
Last update posted: May 3, 2026

2024 – 2029

United States locations

U.S. sites: 11
U.S. states: 10
U.S. cities: 11

Facility	City	State	ZIP	Site status
University of Arkansas for Medical Sciences	Little Rock	Arkansas	72103	Recruiting
Children's Hospital of Colorado	Aurora	Colorado	80045	Recruiting
University of Florida Pediatrics	Gainesville	Florida	32610	Recruiting
Children's Healthcare of Atlanta Center for Advanced Pediatrics	Atlanta	Georgia	30329-2309	Recruiting
Ann and Robert H. Lurie Children Hospital of Chicago	Chicago	Illinois	60611	Not yet recruiting
Helen DeVos Children's Hospital at Spectrum Health	Grand Rapids	Michigan	49503	Recruiting
Columbia University Medical Center	New York	New York	10032	Recruiting
Children'S Hospital of Philadelphia	Philadelphia	Pennsylvania	19104	Recruiting
The University of Texas Southwestern Medical Center at Dallas	Dallas	Texas	75390	Recruiting
Cook Children's Jane and John Justin Neurosciences Center	Fort Worth	Texas	76104	Recruiting
Children's Hospital of the King's Daughter	Norfolk	Virginia	23510	Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT05861986, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted May 3, 2026 · Synced May 8, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT05861986 live on ClinicalTrials.gov.

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