What happens during the study?
The study lasts up to 72 weeks (up to 17 months) with 29 visits for tests and 2 hospital stays.
*At the end of the study, your child will have the option to enroll in a long-term follow-up study in which your child’s health will be monitored for an additional 15 years.
Screening
Before starting the study, you will need to give permission for your child to join by reading and signing an Informed Consent Form. Depending on their age, some children may also give permission by signing an Informed Assent Form. The study doctor will then collect information and run tests to make sure the study is right for your child.
Treatment and Follow-Up
The study has 2 treatment periods along with 2 follow-up periods:
How is OAV101 given?
OAV101 is given as a one-time injection into the lower part of the spine (intrathecal injection).
What kind of tests will my child have?
Some of the tests your child will have during the study include:
Blood Tests
Breathing test
Heart activity (electrocardiogram)
Heart imaging (echocardiogram)
Mental health check*
Motor function tests
Neurological exam
Physical exam
Spinal tap
Urine test
X-ray (chest, spine)
*Mental health check may not apply to children younger than 7 years.
These are only some of the tests that will take place during the study. Your child will not have all of the tests at each visit. Talk to the study doctor for more information.
Caregivers will also be asked to fill out a questionnaire about how their child’s SMA affects their everyday life.