New drug hopes to boost muscle power in babies with SMA
NCT ID NCT07047144
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a drug called apitegromab in children under 2 years old who have spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The drug works by blocking a protein that limits muscle growth. Researchers want to see if adding apitegromab to standard SMA treatments helps babies reach motor milestones like sitting or crawling. The trial involves 52 participants and will measure safety, drug levels, and motor function over 48 weeks.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
apitegromab (a lab-made antibody that blocks myostatin, a protein that limits muscle growth)
What this could lead to
If it works, this could offer a new way to strengthen muscles in infants with SMA, potentially improving their ability to sit, crawl, or walk.
What could go wrong
This is an early Phase 2 study with only 52 participants, so results may not apply to all children. The drug is given alongside existing treatments, and its long-term safety and benefits are not yet known.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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Atrium Health Wake Forest Baptist
RECRUITINGWinston-Salem, North Carolina, 27157, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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CHR Citadelle
RECRUITINGLiège, 4000, Belgium
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Centro Clinico NeMO Milano - Fondazione Serena Onlus
RECRUITINGMilan, 20162, Italy
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Children's Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Orange County (CHOC)
RECRUITINGOrange, California, 92868, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cook Children's Medical Center
RECRUITINGFort Worth, Texas, 76104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta
RECRUITINGMilan, 20133, Italy
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
RECRUITINGRome, 00168, Italy
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Helen DeVos Children's Hospital at Spectrum Health
RECRUITINGGrand Rapids, Michigan, 49503, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hopital Trousseau - I-Motion
RECRUITINGParis, 75012, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Sant Joan de Déu Barcelona
RECRUITINGBarcelona, 08950, Spain
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Hospital Universitari i Politecnico La Fecnic La Fe
RECRUITINGValencia, 46026, Spain
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Le Bonheur Children's Hospital
NOT_YET_RECRUITINGMemphis, Tennessee, 38103, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Leeds General Infirmary Children's Research Department
RECRUITINGLeeds, West Yorkshire, LS1 3EX, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neurology Rare Disease Center
RECRUITINGFlower Mound, Texas, 75028, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Phoenix Children's Hospital
RECRUITINGPhoenix, Arizona, 85016, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Stanford Neuroscience Health Center (SNHC)
RECRUITINGPalo Alto, California, 94304, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Texas Children's Hospital (TCH)-Clinical Care Center (CCC)
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UZ Gent
RECRUITINGGhent, East-Flanders, 9000, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UZ Leuven - Campus Gasthuisberg
RECRUITINGLeuven, 3000, Belgium
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Universitair Medisch Centrum Utrecht
RECRUITINGUtrecht, 3584 CX, Netherlands
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75207, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••