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NCT07221669 · ClinicalTrials.gov registry record · Phase 3
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 of Muscular Atrophy, Spinal, sponsored by Biogen.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 30
- Enrollment target
- 2
- Study locations
NCT07221669 is a Phase 3 study of Muscular Atrophy, Spinal that is actively recruiting participants, run by Biogen. The registered enrollment target is 30 participants, below the 74-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (59% lower). The trial reports 2 study locations across 2 states.
The verdict
NCT07221669, a Phase 3 study of Muscular Atrophy, Spinal, is actively recruiting participants, sponsored by Biogen.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 30 participants
- Enrollment target
- 2
- Study locations
Study 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. 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 b
Conditions Studied
Interventions
- DRUG Salanersen
Study Locations (2)
Texas
- Neurology Rare Disease Center - Flower Mound
Virginia
- Childrens Hospital of the Kings Daughter Norfolk - Norfolk
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2026-01-30 |
| Est. Completion | 2032-05-29 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07221669
The ClinicalTrials.gov registry entry for NCT07221669 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 30 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 74-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (59% lower). The listed sponsor is Biogen, which has 334 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Muscular Atrophy, Spinal appearing as the primary indexed condition, and to 1 intervention - of which Salanersen is the first listed.
NCT07221669 reports 2 study locations spanning 2 distinct geographic areas - top geographies include Texas, Virginia.
Frequently Asked Questions
What is clinical trial NCT07221669 about?
NCT07221669 is a clinical study titled "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)". 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. M...
What is the current status of trial NCT07221669?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 30 participants. The study started on 2026-01-30. Estimated completion is 2032-05-29.
What conditions does trial NCT07221669 study?
This clinical trial studies the following conditions: Muscular Atrophy, Spinal.
What interventions are being tested in trial NCT07221669?
The interventions under investigation include: Salanersen (DRUG).
Who is sponsoring clinical trial NCT07221669?
This trial is sponsored by Biogen, which has 334 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07221669 being conducted?
This trial has 2 study locations across Texas, Virginia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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