RecruitingPhase 4NCT05861999

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 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

Sponsor

Hoffmann-La Roche

Enrollment

28 participants

Start Date

Aug 14, 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 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.

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 19 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(19)

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Valley Children's Hospital

Madera, California, United States

Stanford Univ Medical Center

Palo Alto, California, 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

Helen DeVos Children's Hospital at Spectrum Health

Grand Rapids, Michigan, United States

Children'S Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Texas Southwestern Medical Center

Dallas, 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

Soroka Medical Center

Beersheba, Israel

Schneider Children's Medical Center of Israel

Petah Tikva, Israel

Sourasky MC, Dana-Dwek Children's Hospital

Tel Aviv, Israel

Uniwersyteckie Centrum Kliniczne

Gda?sk, Poland

Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, Poland

Sidra Medicine

Al Rayyan, Qatar

Great Ormond Street Hospital For Children

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT05861999

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