Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

To join this study, you must have successfully finished an earlier study involving apitegromab, either the TOPAZ or SAPPHIRE trial. You should also generally be in good health, with an expected lifespan of more than two years.

You'll need to be able to receive the study drug through an IV (a drip in your arm) and provide blood samples. It's also important to be able to attend all clinic visits and follow the study instructions carefully.

Some things might prevent you from joining. For example, if you stopped taking the study drug early in a previous trial, or if you have severe allergies to certain medicines, including apitegromab. If you're currently in another drug study, or if your nutrition isn't stable, you might not be able to participate.

If you join the ONYX study, you will continue to receive the study medicine, apitegromab, through an intravenous (IV) infusion, which is like a drip into your vein. You will have regular visits to the clinic so the study team can check your health, see how the medicine is affecting you, and monitor for any side effects. These visits will involve physical examinations, providing blood samples, and answering questions about your well-being and any changes you notice.

The study is designed to look at the long-term effects of apitegromab, so it will continue for an extended period. The exact total duration will be discussed by the study team, but it's important to be prepared for ongoing participation. You'll need to commit to attending all scheduled appointments and following the study guidelines. The study team will provide more details on the schedule and what each visit involves.