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Spinal injection drug shows promise in stopping SMA before symptoms start

NCT ID NCT02386553

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a drug called nusinersen (Spinraza) in 25 infants who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The drug is given through a spinal injection to help delay or prevent the need for breathing support or early death. The goal was to see if early treatment can keep these babies healthier longer.

What this could mean

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

Active substance
Nusinersen (Spinraza), a drug given via spinal injection
What this could lead to
If it works, this could help infants with SMA grow up without needing breathing support or dying early.
What could go wrong
This is a small, early-phase study with only 25 infants, so results may not apply to all. The drug requires repeated spinal injections, which carry risks like infection or headache.

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

25 people

The number who actually took part.

Started

May 2015

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

0 to 6 weeks

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: * Age ≤ 6 weeks at first dose. * Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation. * Genetic documentation of 2 or 3 copies of survival motor neuron 2 (SMN2). * Ulnar compound muscle action potential (CMAP) ≥ 1 mV at Baseline. * Gestational age of 37 to 42 weeks for singleton births; gestational age of 34 to 42 weeks for twins. * Meet additional study related criteria. Key Exclusion Criteria: * Hypoxemia (oxygen saturation \<96% awake or asleep without any supplemental oxygen or respiratory support). * Any clinical signs or symptoms at Screening or immediately prior to the first dosing (Day 1) that are, in the opinion of the Investigator, strongly suggestive of SMA. * Clinically significant abnormalities in hematology or clinical chemistry parameters. * Treatment with an investigational drug given for the treatment of SMA biological agent, or device. Any history of gene therapy, prior antisense oligonucleotide (ASO) treatment, or cell transplantation. * Meet additional study related criteria. Note: Other protocol defined Inclusion/Exclusion criteria may 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.

Spinal muscular atrophy

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611-2605, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Columbia University

    New York, New York, 10032, United States

  • David Geffen School of Medicine

    Los Angeles, California, 90095, United States

  • Fondazione Serena Onlus - Centro Clinico Nemo

    Milan, 20162, Italy

  • Hacettepe University Medical Faculty

    Ankara, 6230, Turkey (Türkiye)

  • Hamad General Hospital

    Doha, 3050, Qatar

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 807, Taiwan

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • National Taiwan University Hospital

    Taipei, 100, Taiwan

  • Nemours Children's Hospital, Orlando

    Orlando, Florida, 32827, United States

  • Ospedale Pediatrico Bambino Gesù

    Rome, Lazio, 165, Italy

  • Queensland Children's Hospital

    South Brisbane, Queensland, 4101, Australia

  • Royal Children's Hospital

    Parkville, Victoria, 3052, Australia

  • Seattle Children's Research Institute

    Seattle, Washington, 98101, United States

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • The Johns Hopkins Hospital

    Baltimore, Maryland, 21205, United States

  • Universitaetsklinikum Freiburg

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • University of California Davis Health System

    Sacramento, California, 95817, United States

  • University of Utah

    Salt Lake City, Utah, 84112, United States

  • Yeditepe University Medical School Hospital

    Istanbul, 31755, Turkey (Türkiye)

More trials for these conditions

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