The RESPOND Study is evaluating the safety and efficacy of Spinraza (nusinersen) in infants and children with SMA three years of age and under who have been previously treated with Zolgensma (onasemnogene abeparvovec).
To be eligible, your child must:
Be three years of
age or under
Other criteria will need to be met before your child can participate. The study doctor will be able to explain these in more detail with you.
If your child is eligible and you choose to participate, the study will last up to 115 weeks, approximately 29 months. All participants will receive the investigational drug.
Choosing to take part in a clinical research study is a big decision. It is important for you to understand what the study is about, what the potential risks are, and what your child's participation would involve before agreeing to have them take part.
If you are interested in the RESPOND Study, you will meet with the study doctor and be able to ask more questions about procedures and requirements. The study center will give you a document called an “Informed Consent Form” which explains the study in more detail.
If you decide to have your child participate, you must provide your consent by signing the form. This process is called the informed consent process, and it is standard procedure for participation in a clinical study.
Once you have completed this process, the study doctor and their staff will conduct medical tests and procedures to determine if your child is eligible to participate.
If your child is eligible and enrolled in the study, they will receive the following at no cost during the course of the study:
Your child will continue to see their regular doctors for routine office visits and checkups.
The RESPOND Study is being conducted by Biogen.
It’s natural to have questions about participation in a clinical research study, especially when it involves the well-being of your child. The study doctor can answer your questions so that you have all the information you need to make a decision.