Recruiting

STELLAR-1Salanersen for Presymptomatic Infants with Spinal Muscular Atrophy

Study Aim

This study aims to evaluate the effectiveness and safety of Salanersen treatment in presymptomatic infants diagnosed with Spinal Muscular Atrophy.

What is being tested

Salanersen

Drug

Who is being recruted

Central Nervous System Diseases+4

+ Muscular Atrophy, Spinal

+ Nervous System Diseases

+12 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 3

Interventional

Study Start: January 2026

See protocol details

Summary

Principal SponsorBiogen

Study ContactUS Biogen Clinical Trial CenterMore contacts

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: January 30, 2026

Actual date on which the first participant was enrolled.

This clinical trial aims to test a new treatment called salanersen for infants who have been genetically diagnosed with Spinal Muscular Atrophy (SMA) but have not yet shown symptoms. The study is particularly focused on infants who have not received any previous treatments for SMA. The goal is to see how effective salanersen is in improving the health of these infants and to understand its safety for long-term use. Spinal Muscular Atrophy is a serious genetic disorder that affects muscle strength, and finding effective treatments is crucial to improve the quality of life for those affected. Participants in the study will receive salanersen through a process called intrathecal delivery, which involves administering the medication directly into the spinal fluid. The research will involve two parts: the first part focuses on how well the medicine works and its immediate safety, while the second part looks at the long-term effects and safety of the treatment. Researchers will also study how the body processes the drug and its impact on certain biological markers. The study does not provide specific information on risks, but evaluating the safety is a key component of the research.

Official TitleAn Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular Atrophy

NCT07221669

Principal SponsorBiogen

Study ContactUS Biogen Clinical Trial CenterMore contacts

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

30 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Range of ages for which participants are eligible to join.

Healthy volunteers allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Central Nervous System DiseasesMuscular Atrophy, SpinalNervous System DiseasesNeuromuscular DiseasesSpinal Cord DiseasesMotor Neuron DiseaseNeurodegenerative Diseases

Criteria

5 inclusion criteria required to participate

Ulnar compound muscle action potential (CMAP) amplitude ≥2 millivolt (mV) at Screening and Day 1 predose.

≤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.

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7 exclusion criteria prevent from participating

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.

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Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

In Part 1 of the study, participants will receive two doses of salanersen, 80 milligrams (mg) by intrathecal (IT) lumbar puncture (LP), approximately 12 months apart. Participants who complete Part 1 of the study will receive three doses of salanersen, 80 mg by IT LP, approximately 12 months apart, in Part 2 of the study.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 2 locations

Recruiting

Neurology Rare Disease Center

Flower Mound, United StatesOpen Neurology Rare Disease Center in Google Maps

Recruiting

Childrens Hospital of the Kings Daughter Norfolk

Norfolk, United States

Recruiting

2 Study Centers