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Clinical trials for SMA

Zolgensma (onasemnogene abeparvovec)

OAV101

ANB-004

SMN1 gene insertion

Evrysdi (risdiplam)

Spinraza (nusinersen)

BIIB115

SMN2 gene enhancement

Medtronic Vectris Leads

Spinal cord stimulation

Apitegromab (SRK015)

Taldefgrobep alfa

GYM329

NMD-670

SMN independent

Clinical trials for SMA by therapeutic strategy

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A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have already been treated with onasemnogene abeparvovec (OA) after being diagnosed with SMA.

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.

OA works by replacing the missing or abnormal SMN1 gene. Sometimes, OA treatment may not work as well as expected. As a result, researchers are exploring whether giving another drug after OA could lead to better outcomes for people with SMA.

In this study, participants will have 2 SMN2 copies. The higher the copy number, the less severe the participant's SMA is. They will also have received treatment with OA by the time they were 42 days old and before showing any symptoms of SMA.

The main goal of the study is to learn more about the safety of giving salanersen to babies after OA treatment. Researchers will also learn more about whether salanersen can help make SMA symptoms less serious.

The main question researchers want to answer in this study is:

• How many participants have adverse events and serious adverse events after treatment?

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 neurofilament protein is found in the blood after treatment.
* How much salanersen gets into the fluid surrounding the brain and spinal cord.
* How much salanersen gets into the blood. Researchers will use different tests to learn if motor symptoms are changing, including the World Health Organization (WHO) motor milestones and Hammersmith Infant Neurological Examination (HINE) Section 2 motor milestones.

The study will be done in 2 parts. Part A will last 1 year while Part B will last up to 4 years.

The 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 6 months. Participants must have received OA treatment before the age of 42 days and started screening within 6 months of the OA dose.
* Participants will be assigned to 1 of 2 treatment groups by chance. This is a "double blind" study which means neither the participants, study doctor, nor site staff will know which treatment group the participants are assigned to.
* In this study, salanersen will be given as an intrathecal injection, which is an injection into the fluid surrounding the spine. This is done by a procedure called a lumbar puncture (LP) which involves inserting a needle into the lower back into the space around the spinal cord.
* During Part A, one group will receive 80 milligrams (mg) of salanersen while another group receives a sham (fake) procedure. This means that a small needle prick will be done, but no injection will be given.
* For each participant, the first visit of Part A will be 6 months after they receive OA treatment.
* Part A will have up to 6 clinic visits and 2 phone calls and last up to 1 year.
* During Part B, both groups of participants will receive 80 mg of salanersen once a year.
* Part B will have up to 12 clinic visits and 14 phone calls and last up to 4 years.
* In total, participants will be in the study for up to 5 and a half years.

Biogen

A Phase 3, Randomized, Double-Blinded Study to Evaluate the Safety and Efficacy of Salanersen (BIIB115) After Onasemnogene Abeparvovec Treatment in Infants With Genetically Diagnosed Spinal Muscular Atrophy

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. 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.

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

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam

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 individuals with SMA who are between the ages of 15 and 60 years old. In most people living with SMA, changes to or a lack of a gene called survival motor neuron 1 (SMN1) - often referred to as gene mutations or variants - affect how this gene works. As a result, their bodies produce less SMN protein. Without enough of this protein, motor neurons and muscles cannot work properly. There is a similar gene called SMN2 that produces SMN protein, but it usually does not produce enough SMN protein on its own to make up for the changes in the SMN1 gene. Salanersen is a drug designed to help the SMN2 gene to make more working SMN protein.

In this study, there will be 2 groups of participants: a group who has never received treatment for SMA before joining this study, and a group who has been treated with risdiplam, an approved drug for SMA . Those participants must not have received any other SMA treatments before and will need to stop their risdiplam treatment for the duration of the study.

The main goal of this study is to learn more about how salanersen affects the participants' motor function. Researchers will use different tests and questionnaires to learn if motor function is changing over the study duration.

The main question researchers want to answer in this study is:

• For the group who has never been treated for SMA, how much do scores on the HFMSE movement test change at 12 months compared to the beginning of the study? The Hammersmith Functional Motor Scale - Expanded (HFMSE) has 33 activities that are scored which include sitting, lying down, walking, jumping, and more.

Researchers will also learn more about:

* The effects on participants' motor function and how well their nerves and muscles function.
* The effects on participants' overall sense of change and how they perform daily activities.
* 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.
* How much salanersen gets into the fluid surrounding the brain and spinal cord.
* How much salanersen gets into the blood.

This study will be done as follows:

* First, participants will be screened to check if they can join the study. The screening period may be up to 4 weeks.
* This is an "open-label" study. This is a study in which the participants, study doctor, and site staff will know that participants are receiving salanersen.
* All participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord.
* Participants will receive salanersen once every year for a total of 5 times throughout the study.
* Including screening, participants will have 17 study visits and 9 telephone calls during this study, which will last up to 61 months in total.

An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of Salanersen (BIIB115) in Participants Aged 15-60 Years With Spinal Muscular Atrophy Who Are Either Treatment-Naïve or Have Previously Been Treated With Risdiplam

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A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

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 Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam

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