EN-US
STELLAR-2
A clinical study for children with SMA
STELLAR-2 is a clinical study for infants diagnosed with spinal muscular atrophy (SMA) who have received treatment with onasemnogene abeparvovec, also called ZOLGENSMA®.
This study is evaluating an investigational drug called salanersen to understand its safety and potential benefits when added after ZOLGENSMA® treatment.
Participation is voluntary. Travel and reimbursement support is available.
About the STELLAR-2 study
Evaluating an investigational drug for SMA
The STELLAR-2 Study is evaluating the effect of a study drug and how safe it is for the treatment of spinal muscular atrophy (SMA) in infants and children. Although there are treatment options available for individuals with SMA, they may not be effective for everyone. This is why it is important for clinical research to continue to explore different therapies.
If you are interested in having your child join the STELLAR-2 Study, they must:
- Have a genetic diagnosis of SMA
- Have two copies of the SMN2 gene
- Receive ZOLGENSMA® no more than 42 days after birth
- Study screening must begin within six months of the dose - Not experience any symptoms of SMA before receiving ZOLGENSMA®
- Participants who experience symptoms after their ZOLGENSMA® dose may still be allowed to enroll
There are more requirements to join the study, which will be explained by the study team.
What causes SMA?
SMA is a rare, genetic disease that affects motor neurons. These are the nerves that control muscle movement. SMA weakens or destroys motor neurons, causing muscles to waste away. Over time, this can lead to problems with moving, breathing, and swallowing. SMA affects each person differently––some may notice motor function changes slowly, while others may see them more quickly.
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. There is another gene called SMN2 gene 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.
What is salanersen?
The study drug being evaluated in the STELLAR-2 Study is called salanersen.
In people with SMA, the SMN1 gene is either missing or changed leading to a lower amount of overall SMN protein in the body. Without enough of this protein, motor neurons and muscles lose their ability to work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen is designed to help the SMN2 gene to make more SMN protein.
Salanersen is being tested in an ongoing phase 1 clinical study for participants ages six months to 12 years old. Researchers believe that adding salanersen after ZOLGENSMA® may help improve outcomes for infants with SMA.
How it works
You're not alone.
Here's how to get started.
1
Share your contact details
- Fill out the contact form so we can connect you with a Patient Navigator.
- Participation is voluntary, and you may stop at any time.
- Your personal information is kept private.
2
Quick eligibility check
- Answer a few questions to see if you might be able to join the STELLAR-2 Study.
- We only ask for the basic medical information needed to check if you might qualify.
- If you seem to qualify for the trial, you can schedule a free phone call with a Patient Navigator on a day and time that works for you.
3
Meet Your Patient Navigator
- Your Patient Navigator will explain the basics of the trial, including what to expect and what is required.
- They will check if you meet requirements for the trial, guide you through the next steps, and answer any questions you may have.
- If you are interested and seem to qualify, your Patient Navigator will connect you with a trial site.
Frequently asked questions
How long is the study?
The total study duration is about 5 and a half years long. The study includes three parts: Screening, Treatment, and Follow-up. Screening lasts up to 6 months, during which the study team will confirm your child’s SMA diagnosis through genetic testing and clinical assessments and determine whether the study is a good match. This involves one or more visits to the study site. If your child is eligible, you can choose to join the study.
Treatment and Follow-Up will last up to 5 years. There will be about 20 clinic visits and 16 phone calls during this time to monitor your child’s health and progress.
The study team will walk you through the full timeline and answer any questions you may have. Your child’s participation is completely voluntary, and you can withdraw at any time.
Will there be any reimbursement and/or travel support?
Yes. Travel reimbursement is available to you and your family to support your participation in the STELLAR-2 Study. There is no cost for the study drug, study-related care, or monitoring.
What should we expect during the study?
In the STELLAR-2 Study, participants will receive an intrathecal lumbar puncture injection once every year. This means that a thin needle will be put into the lower part of the spine to deliver the study drug into the fluid surrounding the spine.
Participants will be assigned to one of two treatment groups. Depending on their assignment, they will receive either: five injections of the study drug or one sham procedure followed by four injections of the study drug
A sham procedure is used so researchers can compare two groups in a fair way. This helps them learn what changes are caused by the study drug. In the sham procedure, the doctor will do a small needle prick on the skin of the lower back. The needle will not go deeper into the back, and it will not reach the space around the spinal cord. No study drug will be given during the sham procedure. The sham procedure is made to look similar to the real procedure, without giving the medicine. You and most of the study team (including the doctors who check your child’s progress) will not know whether your child received the study drug or the sham procedure.
In the STELLAR-2 Study, your child will undergo several tests and assessments commonly performed in clinical research, such as physical and neurological exams, questionnaires, and blood and urine sample collections. Some visits may require a short stay for observation, especially after dosing.
What kind of care will my child receive during the study?
Your child can usually continue seeing their regular healthcare providers while participating in the study.
At the same time, the clinical study offers access to a dedicated study team who carefully monitors your child’s health and progress. This team focuses on the condition being studied and provides all study related medications and procedures at no cost. The study does not replace your child’s overall medical care but adds an extra layer of support.
What support or follow-up care is available after the study ends?
The study team will help you plan the next steps and share all study-related health information with your child’s regular doctor to help with continuity of care.
Find a study near you
STELLAR-2 is a global study running at hospitals and clinics in 16 countries around the world.
Your Patient Navigator will work with you to connect to the right site and answer any questions about travel or logistics.
See available study sites in the US on the map below:
Please read, your consent is required
If you are a parent or caregiver of a child with genetic diagnosis of Spinal Muscular Atrophy (SMA), you are invited to complete pre-screening questions to determine for participation in an SMA clinical study.
Biogen, the sponsor of this study, is responsible for the protection of your child’s personal information. Biogen has engaged myTomorrows (“myTomorrows”, “we” or “us”) to support study recruitment for the study by collecting pre-screening information for the study through this website.
With your consent, we will use this information to evaluate your child’s potential eligibility for the study.
The information that could identify your child will not be shared with Biogen. Only de-identified data will be shared with Biogen MA Inc.
You may withdraw your consent at any time and upon withdrawal of your consent we will promptly delete your personal data, except to the extent required to comply with our legal obligations. If you have a question, wish to withdraw consent, or exercise your other privacy rights, please contact myTomorrows at dataprotection@mytomorrows.com as Biogen MA Inc. will not receive any information that can directly identify you or your child. For more information about the Biogen Privacy Policy, please visit https://www.biogen.com/privacy-center.html or https://www.biogen.com/privacy-center/international-privacy.html.
After answering the pre-screener questions, you will have the opportunity to schedule a call with one of our Patient Navigators, who will collect additional information about your child’s health to assess the initial eligibility for this study. If potentially eligible, they will provide you with further information and answer questions about possible site locations, travel logistics and preferences, if applicable. If we find that your child may be eligible for this study, and you confirm your interest in participating, we will, with your consent, share the personal information collected with a clinical study site. The site will contact you to discuss next steps and answer any questions you may have. Your information may also be shared with other vendors of the Sponsor.
Even if your child does not qualify for this study, it is possible to schedule a call with one of our Patient Navigators to discuss other options that may be available.
We will retain your data for 2 years so we can get in touch with you if other options become available to your child during this time period.
Powered by
