New Muscle-Boosting drug shows promise for SMA patients in major trial

NCT ID NCT05156320

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 trial tested apitegromab, a drug that blocks a muscle-limiting protein, in 188 nonambulatory children and young adults with later-onset spinal muscular atrophy (SMA types 2 and 3). All participants were already taking standard SMA therapies (nusinersen or risdiplam). The study measured changes in motor function over time. Results could support apitegromab as an add-on therapy to improve muscle strength and daily movement.

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 mean a new add-on treatment to help people with later-onset SMA gain more muscle strength and movement.
What could go wrong: This is a Phase 3 trial, but results are not yet published. Adding another drug may increase side effects, and not everyone may benefit.

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

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Atrophy muscular atrophy neuromuscular disease 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 ATROPHY MUSCULAR ATROPHY MUSCULAR ATROPHY, SPINAL NEUROMUSCULAR DISEASES NEUROMUSCULAR MANIFESTATIONS SMA SPINAL MUSCULAR ATROPHY SPINAL MUSCULAR ATROPHY TYPE 2 SPINAL MUSCULAR ATROPHY TYPE 3

Contacts and locations

Show contact details

Locations

A.O.U Policlinico G. Martino

Messina, 98125, Italy
ASST Grande Ospedale Metropolitano Niguarda

Milan, 20162, Italy
Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
CHU Lille - Hôpital Jeanne de Flandre

Lille, 59037, France
CHU Toulouse - Hopital des Enfants

Toulouse, 31059, France
Centro Clinico Nemo Pediatrico Policlinico A. Gemelli-Università Cattolica Sacro Cuore

Roma, 00168, Italy
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States
Children's Medical Center Dallas

Dallas, Texas, 75235, United States
Children's of Alabama

Birmingham, Alabama, 35233, United States
Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Chr de La Citadelle

Liège, 4000, Belgium
Columbia University, SMA Clinical Research Center

New York, New York, 10032, United States
Dr. von Haunersches Kinderspital

Munich, 80337, Germany
Foundation I.R.C.C.S. Carlo Besta Neurological Institute

Milan, 20133, Italy
Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States
Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom
Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States
Hospital Sant Joan de Déu

Barcelona, Spain
Hospital Universitario y Politécnico La Fe

Valencia, Spain
Hôpital Armand Trousseau, I-Motion

Paris, 75012, France
IRCCS Istituto Giannina Gaslini

Genoa, Italy
Instytut Pomnik - Centrum Zdrowia Dziecka: CZD Warszawa

Warsaw, 04-736, Poland
Leeds Teaching Hospitals NHS Trust

Leeds, LS1 3EX, United Kingdom
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Nemours Children's Hospital

Orlando, Florida, 32827, United States
Oregon Health & Sciences University

Portland, Oregon, 97239, United States
Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States
Rady's Children's Hospital/UCSD

San Diego, California, 92123, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Stanford University Medical Center

Palo Alto, California, 94304, United States
The Johns Hopkins University

Baltimore, Maryland, 21287, United States
UZ Gent

Ghent, 9000, Belgium
UZ Leuven

Leuven, 3000, Belgium
Universitaetsklinikum Freiburg

Freiburg im Breisgau, 79106, Germany
Universitair Medisch Centrum Utrecht

Utrecht, 3584, Netherlands
University of Iowa

Iowa City, Iowa, 52242, United States
University of Kansas Medical Center

Kansas City, Kansas, 66160, United States
University of Oxford

Oxford, OX3 0ER, United Kingdom
University of Utah

Salt Lake City, Utah, 84112, United States
University of Wisconsin School of Medicine and Public Health

Madison, Wisconsin, 53705, United States
Universitätsklinikum Bonn

Bonn, 53127, Germany
Universitätsklinikum Essen

Essen, 45147, Germany
Uniwersyteckie Centrum Kliniczne w Gdańsku

Gdansk, 80-001, Poland
Uniwersytet Medyczny im. Karola Marcinkowskiego w Poznaniu

Poznan, 61-701, Poland
Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, 27157, United States
Washington University School of Medicine in St. Louis

St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.