The DEVOTE Study
is working to evaluate potential
treatment options for SMA.
Doctors are conducting this Biogen-sponsored study to evaluate the safety and effectiveness of different doses of a drug known as nusinersen. Infants, children, and adults who have genetic documentation of SMA may be eligible for the study.
The DEVOTE Study
A diagnosis of spinal muscular atrophy (SMA) can be very difficult for you and your family. That’s why we are committed to evaluating potential treatment options for people with SMA. The DEVOTE study is evaluating the safety and effectiveness of different doses of a drug known as nusinersen.
If you or your child is eligible and you choose to join, participation will last up to 10-14 months. All participants will receive the study drug, but some individuals may receive an investigational higher dose.
The study has three parts, which have different eligibility criteria and visits:
Note: Not all countries will participate in all three parts of the study. Please click on the buttons below to see details on each part of the study.
Part A has been fully enrolled. Please check Parts B and C to see if you or your child may be eligible for these parts of the study.
Visits: 9-10
People who may be eligible:
- infants >1 week to ≤7 months (210 days) old who had symptoms appear when they were 6 months (180 days) old or younger (infantile-onset SMA) OR
- children 2 to <10 years old who had symptoms appear after they were 6 months (>180 days) of age (later-onset SMA)
- genetically* diagnosed with SMA
Study design:
blinded (meaning participants will be randomized to receive one of two different doses; they will not know which investigational dose they are receiving)**
Visits: 5-6
People who may be eligible:
- children and adults of any age (adults 18 or older must be ambulatory)
- genetically* diagnosed with SMA
- receiving nusinersen for at least one year before entering the study
Study design:
open-label (meaning participants will know what investigational dose they are receiving)**
*A genetic diagnosis is determined by a genetic test. This is a type of medical test that can determine whether a person carries genes for a specific inherited disease. To be eligible for this study, a genetic test must confirm that the potential participant has inherited the gene that causes SMA. A genetic test can be conducted using a small amount of blood.
**Eligibility and the appropriateness of participation should be discussed in full with the study team.
Starting the
Conversation
If you are interested in the DEVOTE study, you will be given an opportunity to ask the study doctor questions about the study procedures and requirements. You will be provided a document called an “Informed Consent Form” that explains the details of the study. If you decide you or your child will participate, you must provide your consent by signing the form. This is called the informed consent process, and it is a 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 you or your child is eligible to participate.
For You and Your Child: What Participation Means
If you or your child is eligible and enrolled in the study, you will receive the following at no cost:
- the study drug
- study-related care and monitoring
- appointments with the study doctor, a neurologist committed to advancing research for SMA
You or your child will continue to see your regular doctors for routine office visits and checkups.
At the end of the study, you may be eligible to participate in an extension study and continue to receive nusinersen at no cost.
Travel Support
Because you and other families may need to travel a long distance to participate in the study, travel support may be available in the form of coordination and coverage of expenses. (Depending on your distance to the study location, this may include air and / or ground transportation, and hotel.)
Neither you nor your insurance provider will be charged for the costs of any tests or procedures performed as part of the clinical study.
The DEVOTE study is being conducted by Biogen.
It’s natural to have questions about participation, especially when it’s for you or your child whose well-being and care you are committed to. The study doctor can answer your questions so that you have all the information you need to make a decision.
