About the ILLUMINATE Study


To join the study, participants must:
Potential participants will need to meet other requirements to join the ILLUMINATE Study. The study team will tell you more about the additional criteria.

The study is divided into 2 parts and will last a maximum of around 99 weeks (about 2 years), with up to 15 study visits.*
During the first part of the study, which will last 20 weeks, participants will be screened and then randomly selected (like flipping a fair coin, where there's a 50% chance of getting heads and a 50% chance of getting tails) to receive either the study drug, or placebo, which looks like the study drug but contains no active ingredients.
After completing part 1, participants may be eligible for part 2 of the ILLUMINATE Study. At that time, all participants will receive the study drug; no placebo will be given.
*Please note that those who do not have access to the study drug through an alternative source (e.g., commercially available product or a managed access program) after 52 weeks in the open-label period may continue receiving it with site visits for safety assessments every 6 months until alternative access is available.

During study visits, you or your loved one will have physical and neurological exams, have vital signs checked (blood pressure, pulse, temperature), give blood and urine samples for testing, and have electrocardiograms (ECGs) and echocardiograms (ECHOs) to check heart health. Caregivers will keep an electronic diary of medications and complete questionnaires.
What happens at the study visits?
Study visits may vary in length, lasting from half a day to a full day at the study site. Some visits will also take place by telephone, and a qualified nurse may conduct certain visits at your home. If you or your loved one is eligible to join the study and chose to participate, detailed information will be provided about the study and what to expect at each visit.
During study visits, participants will undergo various tests and assessments, which may include the following:
The ILLUMINATE Study team is committed to supporting you or your loved one throughout study participation. We want to make your participation as easy and stress-free as possible. Comprehensive travel, reimbursement support, and compensation may be provided for the participant and all caregivers.
Before you or your loved one decides to join the ILLUMINATE Study, the study team will explain everything related to the study in more detail. You will have the opportunity to ask any questions you may have.
You can answer the questions in the pre-screener questionnaire to see if you or your loved one might qualify for the ILLUMINATE Study. You will find a study center near you on the Find a study center page.
In addition to talking about the details of the ILLUMINATE Study, the study center team will also go through the Informed Consent Form (ICF) with you. An ICF is there to make sure you have all the information you need to make the best decision for yourself and your loved one about taking part in the clinical study.