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

ClinicalTrials.gov ID: NCT05861999

Public ClinicalTrials.gov record NCT05861999. 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 20, 2026, 2:38 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase IV Open-Label 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

Study identification

NCT ID: NCT05861999
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: Aug 13, 2024
Primary completion: Mar 30, 2028
Completion: Mar 30, 2029
Last update posted: May 7, 2026

2024 – 2029

United States locations

U.S. sites: 10
U.S. states: 9
U.S. cities: 10

Facility	City	State	ZIP	Site status
University of Arkansas for Medical Sciences	Little Rock	Arkansas	72103	Recruiting
Valley Children's Hospital	Madera	California	93636	Recruiting
Stanford Univ Medical Center	Palo Alto	California	94304	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
Helen DeVos Children's Hospital at Spectrum Health	Grand Rapids	Michigan	49503	Recruiting
Children'S Hospital of Philadelphia	Philadelphia	Pennsylvania	19104	Recruiting
University of Texas Southwestern Medical Center	Dallas	Texas	75390	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 9 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT05861999, 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 7, 2026 · Synced May 20, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT05861999 live on ClinicalTrials.gov.

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