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New drug hopes to boost muscle power in babies with SMA

NCT ID NCT07047144

Recruiting now Disease control Sponsor: Scholar Rock, Inc. Source: ClinicalTrials.gov ↗

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.

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.

Neuromuscular Manifestations proximal spinal muscular atrophy Spinal Muscular Atrophies of Childhood spinal muscular atrophy spinal muscular atrophy, type II spinal muscular atrophy, type III

As listed by the trial registrant

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

ANTI-MYOSTATIN NEUROMUSCULAR MANIFESTATIONS SMA SPINAL MUSCULAR ATROPHY SPINAL MUSCULAR ATROPHY TYPE 2 SPINAL MUSCULAR ATROPHY TYPE 3

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Atrium Health Wake Forest Baptist

    RECRUITING

    Winston-Salem, North Carolina, 27157, United States

  • CHR Citadelle

    RECRUITING

    Liège, 4000, Belgium

  • Centro Clinico NeMO Milano - Fondazione Serena Onlus

    RECRUITING

    Milan, 20162, Italy

  • Children's Healthcare of Atlanta

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Orange County (CHOC)

    RECRUITING

    Orange, California, 92868, United States

  • Cook Children's Medical Center

    RECRUITING

    Fort Worth, Texas, 76104, United States

  • Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta

    RECRUITING

    Milan, 20133, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    RECRUITING

    Rome, 00168, Italy

  • Helen DeVos Children's Hospital at Spectrum Health

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

  • Hopital Trousseau - I-Motion

    RECRUITING

    Paris, 75012, France

  • Hospital Sant Joan de Déu Barcelona

    RECRUITING

    Barcelona, 08950, Spain

  • Hospital Universitari i Politecnico La Fecnic La Fe

    RECRUITING

    Valencia, 46026, Spain

  • Le Bonheur Children's Hospital

    NOT_YET_RECRUITING

    Memphis, Tennessee, 38103, United States

  • Leeds General Infirmary Children's Research Department

    RECRUITING

    Leeds, West Yorkshire, LS1 3EX, United Kingdom

  • Neurology Rare Disease Center

    RECRUITING

    Flower Mound, Texas, 75028, United States

  • Phoenix Children's Hospital

    RECRUITING

    Phoenix, Arizona, 85016, United States

  • Stanford Neuroscience Health Center (SNHC)

    RECRUITING

    Palo Alto, California, 94304, United States

    Contact Email: •••••@•••••

  • Texas Children's Hospital (TCH)-Clinical Care Center (CCC)

    RECRUITING

    Houston, Texas, 77030, United States

  • The Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • UZ Gent

    RECRUITING

    Ghent, East-Flanders, 9000, Belgium

  • UZ Leuven - Campus Gasthuisberg

    RECRUITING

    Leuven, 3000, Belgium

  • Universitair Medisch Centrum Utrecht

    RECRUITING

    Utrecht, 3584 CX, Netherlands

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75207, United States

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