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New drug hopes to boost muscle power in babies with SMA

NCT ID NCT07047144

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Sep 15, 2026 · Updated 3 times

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.

Study facts

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

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 52 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2025

Expected to finish

Mar 2029

An estimate. End dates often move.

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

Up to 2 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: 1. Is \<2 years old at the time of the informed consent 2. Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth 3. Has confirmed diagnosis of 5q autosomal recessive SMA 4. Has confirmed presence of SMN2 gene copy(ies) 5. Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam) 6. Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit 7. Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score \<55 Exclusion Criteria: 1. Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric feeding tube, where most feeds are administered by this route 2. Major orthopedic issues such as severe scoliosis or severe contractures or interventional procedure, including spine or hip surgery, which is considered to have the potential to substantially limit the ability of the subject to be evaluated on any motor function outcome measures, within 6 months before Screening or anticipated during the study 3. Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from undergoing motor function outcome measures throughout the study.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    24 sites in 7 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • 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

More trials for these conditions

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