New hope for kids with SMA: experimental drug ARGX-119 enters Mid-Stage trial
NCT ID NCT07287982
First seen Jun 25, 2026 · Last updated Jul 22, 2026 · Updated 3 times
Summary
This study tests a new biologic drug called ARGX-119 in children aged 5 to 17 with spinal muscular atrophy (SMA). About 60 participants will receive either ARGX-119 or a placebo by IV infusion for 24 weeks, alongside their usual SMA therapy. The goal is to find the best dose and see if it safely improves muscle function and walking ability.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- ARGX-119 (a biologic drug given by IV infusion)
- What this could lead to
- If successful, ARGX-119 could become an additional treatment option to improve motor function in children with spinal muscular atrophy.
- What could go wrong
- This is a phase 2 trial with only 60 participants, so results may not apply to all. The drug is still experimental, and side effects or lack of benefit are possible.
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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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Boston Children's Hospital
NOT_YET_RECRUITINGBoston, Massachusetts, 02115, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital Philadelphia - Neurology
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Childrens Hospital of The Kings Daughters
NOT_YET_RECRUITINGNorfolk, Virginia, 23507, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Columbia University Herbert Irving Comprehensive Cancer Center
NOT_YET_RECRUITINGNew York, New York, 10032, United States
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Connecticut Children's Medical Center
RECRUITINGHartford, Connecticut, 06106, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neurology Rare Disease Center
RECRUITINGFlower Mound, Texas, 75028, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rady Childrens Hospital
NOT_YET_RECRUITINGSan Diego, California, 92123, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rare Disease Research FL LLC
RECRUITINGKissimmee, Florida, 34746, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rare Disease Research NC, LLC
RECRUITINGHillsborough, North Carolina, 27278, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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St. Jude Children's Research Hospital
NOT_YET_RECRUITINGMemphis, Tennessee, 38105, United States
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Stanford University Medical Center
NOT_YET_RECRUITINGStanford, California, 94305, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Texas Children's Hospital
NOT_YET_RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Curators of the University of Missouri on behalf of University of Missouri Health Care
NOT_YET_RECRUITINGColumbia, Missouri, 65212, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Johns Hopkins Hospital
NOT_YET_RECRUITINGBaltimore, Maryland, 21205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Iowa Stead Family Children's Hospital
NOT_YET_RECRUITINGIowa City, Iowa, 52242, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene therapy hope for SMA kids: early trial launches
- New knee device may help kids with SMA build leg strength
- New VR device aims to make exercise fun for kids with muscle weakness
- Wearable tech monitors SMA babies at home to pinpoint best time for extra treatment
- Summer camp aims to boost strength in kids with SMA
- Can a support group boost confidence for parents of kids with DMD or SMA?