New drug may boost motor skills in kids with SMA after gene therapy

NCT ID NCT05861986

Recruiting now Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 05, 2026 · Updated 4 times

Summary

This study is testing whether the drug risdiplam can improve motor skills in children under 2 with spinal muscular atrophy (SMA) who have already received gene therapy. The 28 participants will take risdiplam by mouth, and researchers will measure changes in their gross motor skills over 72 weeks. The goal is to see if adding this medication after gene therapy provides extra benefit.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: risdiplam
What this could lead to: If it works, this could show that adding risdiplam after gene therapy helps young children with SMA gain better motor skills.
What could go wrong: This is a small, early-phase study with only 28 participants, so results may not apply to all children. Risks include side effects from the drug.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MUSCULAR ATROPHY, SPINAL

Contacts and locations

Locations

Ann and Robert H. Lurie Children Hospital of Chicago
NOT_YET_RECRUITING

Chicago, Illinois, 60611, United States
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
RECRUITING

Berlin, 13353, Germany
Children'S Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Children's Healthcare of Atlanta Center for Advanced Pediatrics
RECRUITING

Atlanta, Georgia, 30329-2309, United States
Children's Hospital of Colorado
RECRUITING

Aurora, Colorado, 80045, United States
Children's Hospital of the King's Daughter
WITHDRAWN

Norfolk, Virginia, 23510, United States
Columbia University Medical Center
RECRUITING

New York, New York, 10032, United States
Cook Children's Jane and John Justin Neurosciences Center
RECRUITING

Fort Worth, Texas, 76104, United States
Great Ormond Street Hospital For Children
RECRUITING

London, WC1N 3JH, United Kingdom
Helen DeVos Children's Hospital at Spectrum Health
RECRUITING

Grand Rapids, Michigan, 49503, United States
Instytut Pomnik Centrum Zdrowia Dziecka
RECRUITING

Warsaw, 04-730, Poland
The University of Texas Southwestern Medical Center at Dallas
RECRUITING

Dallas, Texas, 75390, United States
UKGM Standort Gießen
RECRUITING

Giessen, 35392, Germany
University of Arkansas for Medical Sciences
RECRUITING

Little Rock, Arkansas, 72103, United States
University of Florida Pediatrics
RECRUITING

Gainesville, Florida, 32610, United States
Uniwersyteckie Centrum Kliniczne
RECRUITING

Uniwersyteckie Centrum Kliniczne, 80-952, Poland

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