Now Enrolling: The OPAL Study for Children Less Than 2 Years of Age With SMA

The compassion of clinical research. Find out more about a new SMA study.

At our center, we are always looking for ways to help families learn about clinical research. Today we want to share a new study that is now enrolling: the OPAL study. It is a clinical research study for children less than 2 years of age with Spinal Muscular Atrophy (SMA).

Below, you will find the main facts in simple words. If you have questions, we are here to help.

What is the OPAL study?

The purpose of the OPAL study is to learn more about the potential effectiveness and safety of an investigational (or study) drug called apitegromab in children less than 2 years of age with SMA. In this study, all participants will receive apitegromab.

Why is this study being done?

At this time, there are approved treatments available to help treat SMA. But there are no approved treatments that specifically focus on treating muscle atrophy in SMA. Muscle atrophy is a condition in which a person's muscles are smaller and weaker than those of their peers.

What is the study drug?

The study drug, apitegromab, is a protein that acts upon a muscle protein called myostatin, which is one of the factors that controls the function of muscles. It is being developed as a potential first muscle-targeted therapy for people with SMA.

What does "investigational" mean?

"Investigational" means that apitegromab is still being studied in clinical research studies. It has been reviewed by a United States (US) Institutional Review Board (IRB) or a European Ethics Committee (EC) for testing in people.

Apitegromab has not been approved by any health authorities, such as the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), or others.

Who can participate in the OPAL study?

The OPAL study is for children less than 2 years of age who have SMA and have either:

• previously taken onasemnogene abeparvovec-xioi (ZOLGENSMA®), or

• are currently taking nusinersen (SPINRAZA®) or risdiplam (Evrysdi®).*

*Other eligibility criteria will apply. Talk to your doctor to see if your child is able to participate in this clinical research study.

How long will the OPAL study last?

If your child qualifies and you decide to let them participate, participation will last about 1 to 4 years, depending on the following:

• Screening period: up to 4 weeks.

• Study drug treatment period: 48 weeks. Participants will be randomly assigned (by chance, like drawing straws) to receive 1 of 2 doses of apitegromab by intravenous (IV) infusion. If participants are currently taking nusinersen or risdiplam, they should continue to take their usual dose throughout the study.

• Open-label extension (OLE) period (optional): up to 3 years. Participants will continue to receive apitegromab.

• Safety follow-up period: about 20 weeks, after the completion of either the study drug treatment period or the optional OLE period.

What will happen during the OPAL study?

You or your child will be asked to:

• Answer questions about your child's health

• Complete activities and assessments

• Have a physical examination and heart tests

• Give blood and urine samples

• Get the study drug by IV infusion

• If applicable, continue using nusinersen or risdiplam as prescribed

You can always ask the OPAL study doctor or staff any questions about this clinical research study or the study drug at any time.

What are the costs to take part?

The study drug will be given at no cost to your child. You will not be charged for any study doctor visits, laboratory work, assessments, or procedures needed for the OPAL study.

Your child will receive no direct payment for taking part in the OPAL study. However, you may be reimbursed for transportation or parking payments related to visiting the study center.

What are clinical research studies?

Clinical research studies are conducted to test investigational drugs for potential treatment of disease and conditions. They help determine if investigational drugs are safe to use and if they work to improve people's health. Before any investigational drug can be approved and made available to the public, it must go through several phases of clinical research.

People take part in clinical research for many reasons. Some want to learn more about their disease. Others want to help researchers learn more about a disease to potentially help themselves and others in the future. Joining a study is always your choice.

How can I learn more?

• Visit the study website: www.myopalstudy.com/us

• Contact our study team: research@neuromdcenter.com

• Talk to your child's doctor to see if your child is able to participate.

Other eligibility criteria will apply.

The investigational drug (apitegromab) has not been approved by any health authorities, such as the United States (US) Food and Drug Administration (FDA), the European Medicines Agency (EMA), or others. This page is for information only and is not medical advice.

ZOLGENSMA is a registered trademark of Novartis Gene Therapies, Inc. SPINRAZA is a registered trademark of Biogen. Evrysdi is a registered trademark of Genentech, Inc.