RecruitingPhase 4NCT05861986

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 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

Sponsor

Hoffmann-La Roche

Enrollment

28 participants

Start Date

May 30, 2024

Study Type

INTERVENTIONAL

Conditions

Muscular Atrophy, Spinal

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.

Eligibility

Min Age: 3 MonthsMax Age: 24 Months

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Risdiplam for people with muscular atrophy, spinal. The study is currently recruiting participants at 16 locations. People eligible for this study include aged 3 Months to 24 Months.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGRisdiplam

Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.

Locations(16)

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Children's Hospital of Colorado

Aurora, Colorado, United States

University of Florida Pediatrics

Gainesville, Florida, United States

Children's Healthcare of Atlanta Center for Advanced Pediatrics

Atlanta, Georgia, United States

Ann and Robert H. Lurie Children Hospital of Chicago

Chicago, Illinois, United States

Helen DeVos Children's Hospital at Spectrum Health

Grand Rapids, Michigan, United States

Columbia University Medical Center

New York, New York, United States

Children'S Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

The University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, United States

Cook Children's Jane and John Justin Neurosciences Center

Fort Worth, Texas, United States

Children's Hospital of the King's Daughter

Norfolk, Virginia, United States

Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie

Berlin, Germany

UKGM Standort Gießen

Giessen, Germany

Uniwersyteckie Centrum Kliniczne

Uniwersyteckie Centrum Kliniczne, Poland

Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, Poland

Great Ormond Street Hospital For Children

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT05861986

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