STELLAR-2: A New SMA Research Study for Infants Treated with Zolgensm

Learning that your child may have spinal muscular atrophy (SMA) can bring many questions. If you are expecting a child, or caring for a newborn, who has been diagnosed with SMA, it may help to know that a clinical study called STELLAR-2 could be a possibility to explore.

Our research team at the Neurology Rare Disease Center (NRDC) can begin screening families who meet the study criteria. Here is a simple guide to what the study is, who may qualify, and what taking part would involve.

A quick look at SMA

SMA is a rare, genetic disease that affects motor neurons, the nerves that control muscle movement. It weakens or destroys these nerves, which can lead to problems with moving, breathing and swallowing. SMA affects each person differently: some notice changes slowly, while others notice them more quickly.

In most people with SMA, changes in a gene called SMN1 mean the body makes less SMN protein. A similar gene, SMN2, also makes this protein, but usually not enough to make up the difference.

What is the STELLAR-2 study?

STELLAR-2 is a clinical study looking at how safe a study drug called salanersen is, and what effect it has, in infants and children with SMA. Treatment options already exist for SMA, but they may not work for everyone, which is why research continues.

Salanersen is designed to help the SMN2 gene make more SMN protein. It is being tested in another study in older children who already received Zolgensma. In STELLAR-2, researchers want to learn whether adding salanersen soon after Zolgensma, in children treated very early, may support outcomes later in childhood.

Salanersen is an investigational drug. It is not approved by regulatory authorities.

Who may qualify?

If your child meets these criteria, we can begin screening and enroll within 6 months of Zolgensma:

• A genetic diagnosis of SMA.

• Two copies of the SMN2 gene.

• Zolgensma at 42 days of life or earlier.

• No symptoms of SMA before receiving Zolgensma. Children who develop symptoms after their Zolgensma dose may still be allowed to enroll.

• Screening within six months of the Zolgensma dose.

There are other requirements as well, and our study team will explain them all.

What does taking part involve?

Your child would receive one injection a year. It is called an intrathecal lumbar puncture: a thin needle delivers the study drug into the fluid around the spine.

Children are assigned to one of two groups. One group receives five injections of the study drug. The other receives one sham procedure, followed by four injections of the study drug. In a sham procedure, the doctor gives a small needle prick on the skin of the lower back. The needle does not go deeper and no study drug is given. A sham helps researchers compare the two groups fairly. You and most of the study team will not know which group your child is in.

What does the journey look like?

The study lasts a little over five years:

• Screening: up to 2 clinic visits, within 6 months of the Zolgensma dose.

• Treatment and follow-up: 18 clinic visits and 16 phone calls over about 60 months. Visits become less frequent over time.

Along the way, the team will do physical and neurological exams, blood and urine tests, vital signs, electrocardiograms (ECGs), and growth and movement assessments. Your child's health is monitored alongside their other doctors, and travel reimbursement is available for your family.

It is always your choice

Taking part in a clinical study is voluntary. Our team will explain the potential risks, benefits and possible side effects before you decide. All clinical studies are designed to protect the rights, safety and well-being of participants, follow strict scientific and ethical principles, and are reviewed by an institutional review board (IRB).

Reach out early

Because the study must enroll children within six months of their Zolgensma dose, we ask families to reach out as soon as possible. We know this is a big decision and that it can take time, so our team is happy to answer every question.

Contact our research team: research@neuromdcenter.com

STELLAR-2 is a clinical study of an investigational drug that is not approved by regulatory authorities. Participation is voluntary, eligibility depends on the study requirements, and no specific outcome is guaranteed.