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

NCT ID NCT05156320

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

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

188 people

The number who actually took part.

Started

Apr 2022

Finished

Dec 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Males and females 2 through 21 years old at Screening. * Documented diagnosis of 5q SMA. * Diagnosed with later-onset SMA (i.e., Type 2 and Type 3 SMA) before receiving an approved SMN upregulator therapy (i.e., either nusinersen or risdiplam). * Must be Nonambulatory at Screening. Nonambulatory patients must be able to sit independently (sits up straight with head erect for at least 10 seconds; does not use arms or hands to balance body or support position) per World Health Organization (WHO) motor milestones definition at Screening. * Receiving one background therapy for SMA (i.e., either nusinersen or risdiplam) for the time period specified below and anticipated to remain on that same treatment throughout the trial: 1. If receiving the SMN upregulator therapy nusinersen, must have completed at least 10 months of dosing (i.e., completed the loading regimen and at least 2 maintenance doses) before Screening; 2. If receiving the SMN upregulator therapy risdiplam, must have completed at least 6 months of dosing before Screening. * Motor Function Score (HFMSE) ≥10 and ≤45 at Screening. * Have no physical limitations that would prevent the patient from undergoing motor function outcome measures throughout the duration of the study. * Able to receive study drug infusions and provide blood samples through the use of a peripheral intravenous (IV) or a long-term IV access device that the patient has placed for reasons independent from the study throughout the duration of the study. * Able to adhere to the requirements of the protocol, including travel to the study center and completing all study procedures and study visits. * For patients who are expected to have reached reproductive maturity by the end of the study, adhere to study specific contraception requirements. Exclusion Criteria: * Received ZOLGENSMA® (onasemnogene abeparvovec-xioi) at any time and previous treatment with apitegromab. * Use of invasive ventilation and tracheostomy. * Use of chronic daytime non-invasive ventilatory support for \>16 hours daily in the 2 weeks prior to dosing, or anticipated to regularly receive such daytime ventilator support chronically over the duration of the study. * Any acute or co-morbid condition interfering with the well-being of the patient within 7 days of screening, including active systemic infection, the need for acute treatment or inpatient observation due to any reason. * Severe scoliosis and/or contractures at screening. Based on clinical judgement, any scoliosis or contractures present must be stable over the past 6 months, anticipated to be stable for the duration of the study and not prevent the patient from being evaluated on any functional outcome measures throughout the duration of the study. * Pregnant or breastfeeding. * Major orthopedic or other interventional procedure, including spine or hip surgery, considered to have the potential to substantially limit the ability of the patient to be evaluated on any functional outcome measures, within 6 months prior to Screening, or anticipated for the duration of the study. * Prior history of a hypersensitivity reaction to a monoclonal antibody (mAb) or recombinant protein bearing an Fc domain (such as a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab. * Treatment with investigational drugs within 3 months prior to Screening. * Use of therapies with potentially significant muscle effects (such as androgens, insulin-like growth factor, growth hormone, systemic beta-agonist, botulinum toxin, or muscle relaxants or muscle-enhancing supplements) or potentially significant neuromuscular effects (such as acetylcholinesterase inhibitors) within 60 days prior to screening. * Nutritional status not stable over the past 6 months and not anticipated to be stable throughout the duration of the study. * Patient has any other condition, which in the opinion of the Investigator may compromise safety or compliance, would preclude the patient from successful completion of the study, or interfere with the interpretation of the results.

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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

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

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