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Tests treatment safety and results for Muscular Atrophy, Spinal
Official title 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)
ClinicalTrials.gov ID: NCT07221669
What this study is testing
What is Salanersen?
Salanersen is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for muscular atrophy, spinal.
Also referred to as BIIB115.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 42
You may be able to join if
- Key -≤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
You likely can't join if
- 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 \ 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...
- Any prior treatment with an approved SMA disease-modifying therapy (e.g., nusinersen, onasemnogene abeparvovec-xioi [OA], and/or risdiplam), a...
The study team makes the final eligibility decision.
Where it's taking place
- Chicago, Illinois, United States
- Flower Mound, Texas, United States
- Norfolk, Virginia, United States
- Shanghai, China
- Tokyo, Japan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 days to 42 days. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Chicago, Illinois, United States; Flower Mound, Texas, United States; Norfolk, Virginia, United States; Shanghai, China; Tokyo, Japan. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.