Can a second treatment help kids with SMA who stalled after gene therapy?
NCT ID NCT05861999
First seen Jun 27, 2026 · Last updated Aug 12, 2026 · Updated 4 times
Summary
This study tests whether adding risdiplam, a daily oral medicine, can help children under 2 with spinal muscular atrophy (SMA) who stopped getting better or started declining after receiving gene therapy. The study will enroll 28 children and measure changes in motor skills over 72 weeks. The goal is to see if risdiplam can improve movement and function beyond what gene therapy alone achieved.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Risdiplam (a liquid medicine taken by mouth)
- What this could lead to
- If it works, this could offer a way to help children with SMA who have stopped improving after gene therapy regain some motor function.
- What could go wrong
- This is a small, early-phase study with only 28 children, so results may not apply to everyone. Risdiplam may cause side effects, and it is not known if it will reverse the plateau or decline.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
RECRUITINGBerlin, 13353, Germany
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Children'S Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's Healthcare of Atlanta Center for Advanced Pediatrics
RECRUITINGAtlanta, Georgia, 30329-2309, United States
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Children's Hospital of Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Children's Hospital of the King's Daughter
WITHDRAWNNorfolk, Virginia, 23510, United States
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Great Ormond Street Hospital For Children
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Helen DeVos Children's Hospital at Spectrum Health
RECRUITINGGrand Rapids, Michigan, 49503, United States
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Instytut Pomnik Centrum Zdrowia Dziecka
RECRUITINGWarsaw, 04-730, Poland
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Schneider Children's Medical Center of Israel
RECRUITINGPetah Tikva, 4920235, Israel
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Sidra Medicine
RECRUITINGAl Rayyan, Qatar
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Soroka Medical Center
RECRUITINGBeersheba, 8410101, Israel
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Sourasky MC, Dana-Dwek Children's Hospital
RECRUITINGTel Aviv, 6423906, Israel
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Stanford Univ Medical Center
RECRUITINGPalo Alto, California, 94304, United States
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UKGM Standort Gießen
RECRUITINGGiessen, 35392, Germany
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University of Arkansas for Medical Sciences
RECRUITINGLittle Rock, Arkansas, 72103, United States
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University of Florida Pediatrics
RECRUITINGGainesville, Florida, 32610, United States
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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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Uniwersyteckie Centrum Kliniczne
RECRUITINGGda?sk, 80-952, Poland
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Valley Children's Hospital
RECRUITINGMadera, California, 93636, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a spinal injection safely slow spinal muscular atrophy? a real-world study in korea seeks answers.
- New drug BIIB115 aims to build on gene therapy for spinal muscular atrophy
- New hope for SMA babies: boosting gene therapy with a Follow-Up drug
- Real-World data reveals treatment patterns for kids with SMA
- Massive data dive reveals how SMA drugs perform outside the lab