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New drug could help babies with SMA walk and sit normally

NCT ID NCT07221669

Disease control Sponsor: Biogen 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Aug 19, 2026 · Updated 7 times

Summary

This study tests a drug called salanersen in babies who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The drug helps the body make more of a protein that is missing in SMA, which is needed for muscles to work. Researchers want to see if starting treatment early can help babies reach normal milestones like sitting and walking, and prevent or reduce symptoms.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
salanersen (BIIB115)
What this could lead to
If successful, this could allow infants with SMA to reach normal motor milestones like sitting and walking, potentially preventing severe disability.
What could go wrong
This is an early-stage, small trial (30 infants) with no placebo group, so results may not be definitive. Long-term safety and efficacy are still unknown.

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

About 30 people

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

Started

Apr 2026

Expected to finish

May 2032

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

0 to 42 days

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.

Key Inclusion Criteria: -≤42 days of age at first dose of salanersen. * Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation. * Two or three copies of the survival motor neuron 2 (SMN2) gene. * Ulnar compound muscle action potential (CMAP) amplitude ≥2 millivolt (mV) at Screening and Day 1 predose. * Body weight ≥3rd percentile for age based on World Health Organization (WHO) Child Growth Standards at the time of informed consent. Key Exclusion Criteria: * Any clinical signs or symptoms at Screening or Day 1 predose that are, in the opinion of the Investigator, strongly suggestive of SMA. * Areflexia on neurologic examination at biceps, knee, or ankle at Screening or Day 1 Predose. * Hypoxemia (oxygen saturation \<96% awake or asleep without any supplemental oxygen or respiratory support, or for altitudes \>1000 meters (m), oxygen saturation of \<92% awake or asleep without any supplemental oxygen or respiratory support). * Diagnosis of neonatal respiratory distress syndrome necessitating surfactant replacement therapy or invasive ventilatory support. * Any reason, anatomical or otherwise (including hematology/coagulation laboratory results), that presents increased risk of complication from the LP procedures or safety assessments. * Any prior treatment with an approved SMA disease-modifying therapy (e.g., nusinersen, onasemnogene abeparvovec-xioi \[OA\], and/or risdiplam), a myostatin inhibitor therapy, or an investigational drug given for the treatment of SMA. Note: Other protocol-defined inclusion/exclusion criteria will apply.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

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

Muscular atrophy, spinal

How to take part

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

  1. The places running it

    11 sites in 5 countries. The list below names each one and where it is.

  2. 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 ↗

  3. 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

Locations

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Childrens Hospital of Fudan University_Shanghai

    RECRUITING

    Shanghai, 201122, China

  • Childrens Hospital of the Kings Daughter Norfolk

    RECRUITING

    Norfolk, Virginia, 23507, United States

  • Guangzhou Woman and Children's Medical Center_Guangzhou

    RECRUITING

    Guangzhou, 5106236, China

  • Hospital da Crianca de Brasilia

    RECRUITING

    Distrito Federal, 70684-831, Brazil

  • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90560-030, Brazil

  • Japan Institute for Health Security National Center for Global Health and Medicine

    RECRUITING

    Tokyo, 162-8655, Japan

  • Neurology Rare Disease Center

    RECRUITING

    Flower Mound, Texas, 75028, United States

  • PSEG Centro de Pesquisa Clinica

    RECRUITING

    Sai Paula, 4038002, Brazil

  • Sydney Children's Hospital_Randwick

    RECRUITING

    Randwick, New South Wales, 2031, Australia

  • The Third Affiliated Hospital of Zhengzhou University - North Campus

    RECRUITING

    Zhengzhou, Henan, 450052, China

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