Muscular Atrophy, SpinalPhase 3STELLAR-1NCT07221669
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
A Phase 3 study testing Salanersen in people with Muscular Atrophy, Spinal, run by Biogen at 14 sites in 7 countries.
Trial summary
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through genetic testing but have not yet started showing signs or symptoms. Read more about this study
- Who is it for?
- Ages 0 to 42 days
- Conditions the study is for
- Muscular Atrophy, Spinal
- What is being tested?
- Salanersen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 14 sitesin 7 countries
- Study dates
- 28 Apr 2026to 29 May 2032
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 42 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- -≤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.
Who cannot join
- 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.
Trial locations and contacts
14 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
Global Biogen Clinical Trial Center contact
14 sites in 7 countries
Brazil4 sites, 4 recruiting
- Recruiting
Hospital da Crianca de Brasilia
Distrito Federal, 70684-831
55 800 002 2345
Janiana Monteiro
- Recruiting
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, 90560-030
55505359
Jonas Alex Morales Saute
- Recruiting
PSEG Centro de Pesquisa Clinica
Sai Paula, 4038002
55115572-4555
Paulo Victor Sgobbi de Souza
- Recruiting
Instituto Da Criança HC FMUSP
São Paulo, 05403-900
55 11 2661-8500
Edmar Zanoteli
United States3 sites, 3 recruiting
- Recruiting
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611
312-227-4637
Nancy Kuntz
- Recruiting
Neurology Rare Disease Center
Flower Mound, Texas, 75028
972-999-1011
Diana Castro
- Recruiting
Childrens Hospital of the Kings Daughter Norfolk
Norfolk, Virginia, 23507
757-668-6981, proud.research@chkd.org
Erika, erika.paradiso@chkd.org
China3 sites, 3 recruiting
- Recruiting
Guangzhou Woman and Children's Medical Center_Guangzhou
Guangzhou, 5106236
- Recruiting
Childrens Hospital of Fudan University_Shanghai
Shanghai, 201122
- Recruiting
The Third Affiliated Hospital of Zhengzhou University - North Campus
Zhengzhou, Henan, 450052
Australia1 site, 1 recruiting
- Recruiting
Sydney Children's Hospital_Randwick
Randwick, New South Wales, 2031
Chile1 site, 1 recruiting
- Recruiting
Clinica MEDS La Dehesa_Lo Barnechea
Lo Barnechea, 7691236
+5 (698) 234-7983
Claudia Castiglioni
India1 site, 1 recruiting
- Recruiting
Royal Institute of Child Neurosciences
Ahmedabad, Gujarat, 380054
+91-990996055
Siddharth Shah
Japan1 site, 1 recruiting
- Recruiting
Japan Institute for Health Security National Center for Global Health and Medicine
Tokyo, 162-8655
About this study
Official title: An 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
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen.
Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through genetic testing but have not yet started showing signs or symptoms. Most people with SMA have changes in a gene called survival motor neuron 1, also known as SMN1. These changes lower the amount of SMN protein in their bodies. Without enough of this protein, motor neurons and muscles cannot work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen works by helping the SMN2 gene to make more SMN protein.
Read more
In this study, participants will have either 2 SMN2 copies or 3 SMN2 copies. The higher the copy number, the less severe the participant's SMA is.
The main goal of this study is to see if starting salanersen before signs or symptoms appear can prevent signs or symptoms of SMA or make them less severe. Researchers will use different tests to learn if motor symptoms are changing, including the World Health Organization (WHO) motor milestones.
The main questions researchers want to answer in this study are:
- How many participants with 2 copies of the SMN2 gene can sit without support at 12 months?
- How many participants with 3 copies of the SMN2 gene can walk alone at 18 months?
Researchers will also learn more about:
- The effects on participants' motor symptoms and how many new movement milestones participants achieve.
- How many participants stay free of SMA symptoms
- How much salanersen gets into the fluid surrounding the brain and spinal cord.
- How much salanersen gets into the blood.
- How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug.
This study will be done as follows:
- First, participants will be screened to check if they can join the study. The screening period will be up to 28 days.
- This is an "open label" study. This is a study in which the participants, study doctor, and site staff know which study drug participants are receiving. In this study, all participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord.
- There will be 2 parts in this study. During Part 1, participants will receive 2 doses of salanersen, about 12 months apart from each other. Part 1 will last up to 25 months.
- During Part 2, participants will continue to receive salanersen. They will receive up to 3 doses, 12 months apart from each other. Part 2 will last up to 36 months.
- During Part 1, participants will have up to 11 clinic visits and up to 3 phone calls. During Part 2, participants will have up to 7 clinic visits and up to 12 phone calls.
Read the detailed description
For Part 1 of the study, the primary objective is to evaluate the clinical efficacy of salanersen in participants with genetically diagnosed SMA, and the secondary objective is to evaluate safety and tolerability, pharmacokinetics (PK), and effect on biomarkers after treatment with salanersen in participants with genetically diagnosed SMA.
For Part 2 of the study, the primary objective is to evaluate the long-term clinical efficacy of salanersen in participants with genetically diagnosed SMA, and the secondary objective is to evaluate the long-term safety and tolerability, PK, and effect on biomarkers after treatment with salanersen in participants with genetically diagnosed SMA.
- Conditions
- Muscular Atrophy, Spinal
- Being tested
- Salanersen
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SalanersenDrug
Also called BIIB115
Administered intrathecally
| Study group | Role | What they receive |
|---|---|---|
| Salanersen | 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. Receives: Drug: Salanersen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Percentage of Participants with 2 Survival Motor Neuron 2 (SMN2) Copies Sitting Without Support (for at Least 10 Seconds)
Measured: At Month 12
Part 1: Percentage of Participants with 3 SMN2 Copies Walking Alone (for at Least 5 Steps)
Measured: At Month 18
Part 2: Percentage of Participants Attaining and Maintaining World Health Organization (WHO) Motor Milestones
The WHO motor milestones will include six key developmental milestones: sitting without support, standing with assistance, hands-and-knees crawling, walking with assistance, standing alone, and walking alone.
Measured: Up to Day 1825
Secondary outcomes (16)
Part 1: Percentage of Participants Attaining World Health Organization (WHO) Motor Milestones
The WHO motor milestones will include six key developmental milestones: sitting without support, standing with assistance, hands-and-knees crawling, walking with assistance, standing alone, and walking alone.
Measured: Up to Day 730
Parts 1 and 2: Percentage of Participants Attaining Hammersmith Infant Neurological Examination Section 2 (HINE-2) Motor Milestones
Section 2 of the HINE is used to assess motor milestones and includes 8 motor milestone categories: voluntary grasp (0 to 3), ability to kick in supine position (0 to 4), head control (0 to 2), rolling (0 to 3), sitting (0 to 4), crawling (0 to 4), standing (0 to 3), and walking (0 to 3). Total HINE-2 score is the sum of points from each item and can range from 0 to 26, with higher scores depicting a better level of ability.
Measured: Up to Day 1825
Parts 1 and 2: Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale
The CHOP INTEND test is designed to evaluate the motor skills of participants with significant motor weakness. It includes 16 items (capturing neck, trunk, and proximal and distal limb strength) structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0-4. The total score ranges from 0-64, with higher scores depicting better motor function.
Measured: Up to Day 1825
Parts 1 and 2: Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitudes
CMAP is a well validated method for tracking disease progression in neuromuscular disorders such as SMA and amyotrophic lateral sclerosis and has been proposed as a potential biomarker of a therapeutic effect in SMA. CMAPs will be performed for the following nerve-muscle pairs: ulnar-abductor digiti minimi and peroneal-tibialis anterior.
Measured: Up to Day 1825
Part 1: Percentage of Participants who Remain Free of Clinically Manifested Spinal Muscular Atrophy (SMA)
Measured: Up to Day 730
Part 2: Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score
The HFMSE is a tool used to assess motor function in individuals with SMA. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all 33 items, with a maximum score of 66 with higher scores depicting better ability to perform activities.
Measured: Up to Day 1825
Part 2: Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE)
The HFMSE is a tool used to assess motor function in individuals with SMA. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all 33 items, with a maximum score of 66 with higher scores depicting better ability to perform activities.
Measured: Up to Day 1825
Part 2: Revised Upper Limb Module (RULM) Total Score
The RULM is developed to assess upper limb functional abilities of participants with SMA. This test consists of a total of 20 upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function.
Measured: Up to Day 1825
Part 2: Change From Baseline in Revised Upper Limb Module (RULM)
The RULM is developed to assess upper limb functional abilities of participants with SMA. This test consists of a total of 20 upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function.
Measured: Up to Day 1825
Part 2: Percentage of Participants who Develop Spinal Muscular Atrophy (SMA) Subtypes (Type 1, 2,3a and 3b) As Assessed by the Investigator
Measured: Up to Day 1825
Parts 1 and 2: Time to Death (Overall Survival)
Measured: Up to Day 1825
Parts 1 and 2: Time to Death or Permanent Ventilation
Permanent ventilation is defined as tracheostomy or ≥16 hours ventilation/day continuously for >21 days in the absence of an acute reversible event.
Measured: Up to Day 1825
Parts 1 and 2: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Up to Day 1825
Parts 1 and 2: Concentration of Salanersen in Cerebrospinal Fluid (CSF)
Measured: Up to Day 1460
Parts 1 and 2: Concentration of Salanersen in Serum
Measured: Up to Day 1825
Parts 1 and 2: Change From Baseline in Plasma Levels of Neurofilament Light Chain (NfL)
Measured: Up to Day 1825
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2025
- Study start28 Apr 2026
- Primary completion28 Nov 2028 (estimated)
- Study completion29 May 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07221669
- Sponsor's protocol ID
- 277SM302
- Other IDs
- 2025-523116-37, 2025
- Acronym
- STELLAR-1
- Status last verified
- Oct 2026
- First submitted
- 24 Oct 2025
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An 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
- ClinicalTrials.gov ID
- NCT07221669
- Acronym / sponsor ID
- STELLAR-1 / 277SM302
- Conditions
- Muscular Atrophy, Spinal
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 28 Apr 2026 (actual)
- Primary completion
- 28 Nov 2028 (estimated)
- Study completion
- 29 May 2032 (estimated)
- First posted
- 28 Oct 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen.
Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through genetic testing but have not yet started showing signs or symptoms. Most people with SMA have changes in a gene called survival motor neuron 1, also known as SMN1. These changes lower the amount of SMN protein in their bodies. Without enough of this protein, motor neurons and muscles cannot work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen works by helping the SMN2 gene to make more SMN protein.
In this study, participants will have either 2 SMN2 copies or 3 SMN2 copies. The higher the copy number, the less severe the participant's SMA is.
The main goal of this study is to see if starting salanersen before signs or symptoms appear can prevent signs or symptoms of SMA or make them less severe. Researchers will use different tests to learn if motor symptoms are changing, including the World Health Organization (WHO) motor milestones.
The main questions researchers want to answer in this study are:
- How many participants with 2 copies of the SMN2 gene can sit without support at 12 months?
- How many participants with 3 copies of the SMN2 gene can walk alone at 18 months?
Researchers will also learn more about:
- The effects on participants' motor symptoms and how many new movement milestones participants achieve.
- How many participants stay free of SMA symptoms
- How much salanersen gets into the fluid surrounding the brain and spinal cord.
- How much salanersen gets into the blood.
- How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug.
This study will be done as follows:
- First, participants will be screened to check if they can join the study. The screening period will be up to 28 days.
- This is an "open label" study. This is a study in which the participants, study doctor, and site staff know which study drug participants are receiving. In this study, all participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord.
- There will be 2 parts in this study. During Part 1, participants will receive 2 doses of salanersen, about 12 months apart from each other. Part 1 will last up to 25 months.
- During Part 2, participants will continue to receive salanersen. They will receive up to 3 doses, 12 months apart from each other. Part 2 will last up to 36 months.
- During Part 1, participants will have up to 11 clinic visits and up to 3 phone calls. During Part 2, participants will have up to 7 clinic visits and up to 12 phone calls.
Detailed description
For Part 1 of the study, the primary objective is to evaluate the clinical efficacy of salanersen in participants with genetically diagnosed SMA, and the secondary objective is to evaluate safety and tolerability, pharmacokinetics (PK), and effect on biomarkers after treatment with salanersen in participants with genetically diagnosed SMA.
For Part 2 of the study, the primary objective is to evaluate the long-term clinical efficacy of salanersen in participants with genetically diagnosed SMA, and the secondary objective is to evaluate the long-term safety and tolerability, PK, and effect on biomarkers after treatment with salanersen in participants with genetically diagnosed SMA.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Salanersen | Experimental | Drug: SalanersenIn 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. |
Who may qualify
- Ages
- 0 to 42 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- -≤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 (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SalanersenAlso: BIIB115Administered intrathecally | Drug | Salanersen |
Primary outcome
Part 1: Percentage of Participants with 2 Survival Motor Neuron 2 (SMN2) Copies Sitting Without Support (for at Least 10 Seconds)
Time frame: At Month 12
Part 1: Percentage of Participants with 3 SMN2 Copies Walking Alone (for at Least 5 Steps)
Time frame: At Month 18
Part 2: Percentage of Participants Attaining and Maintaining World Health Organization (WHO) Motor Milestones
Time frame: Up to Day 1825
The WHO motor milestones will include six key developmental milestones: sitting without support, standing with assistance, hands-and-knees crawling, walking with assistance, standing alone, and walking alone.
16 secondary outcomes
Part 1: Percentage of Participants Attaining World Health Organization (WHO) Motor Milestones
Time frame: Up to Day 730
Parts 1 and 2: Percentage of Participants Attaining Hammersmith Infant Neurological Examination Section 2 (HINE-2) Motor Milestones
Time frame: Up to Day 1825
Parts 1 and 2: Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale
Time frame: Up to Day 1825
Parts 1 and 2: Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitudes
Time frame: Up to Day 1825
Part 1: Percentage of Participants who Remain Free of Clinically Manifested Spinal Muscular Atrophy (SMA)
Time frame: Up to Day 730
Part 2: Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score
Time frame: Up to Day 1825
Part 2: Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE)
Time frame: Up to Day 1825
Part 2: Revised Upper Limb Module (RULM) Total Score
Time frame: Up to Day 1825
Part 2: Change From Baseline in Revised Upper Limb Module (RULM)
Time frame: Up to Day 1825
Part 2: Percentage of Participants who Develop Spinal Muscular Atrophy (SMA) Subtypes (Type 1, 2,3a and 3b) As Assessed by the Investigator
Time frame: Up to Day 1825
Parts 1 and 2: Time to Death (Overall Survival)
Time frame: Up to Day 1825
Parts 1 and 2: Time to Death or Permanent Ventilation
Time frame: Up to Day 1825
Parts 1 and 2: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to Day 1825
Parts 1 and 2: Concentration of Salanersen in Cerebrospinal Fluid (CSF)
Time frame: Up to Day 1460
Parts 1 and 2: Concentration of Salanersen in Serum
Time frame: Up to Day 1825
Parts 1 and 2: Change From Baseline in Plasma Levels of Neurofilament Light Chain (NfL)
Time frame: Up to Day 1825
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2026.
- Connect to a Patient NavigatorLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Global Biogen Clinical Trial Center contact
Medical Director study director
Biogen
14 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Salanersen in people with Muscular Atrophy, Spinal, run by Biogen at 14 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in Brazil, United States, China, Australia, Chile and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact US Biogen Clinical Trial Center (866-633-4636, clinicaltrials@biogen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part 1: Percentage of Participants with 2 Survival Motor Neuron 2 (SMN2) Copies Sitting Without Support (for at Least 10 Seconds) (time frame: At Month 12). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2026.