For Adolescent Patients and Parents/Caregivers of Pediatric Patients

The BRAVE Study is evaluating a potential treatment option for Friedreich Ataxia.

If your child has been diagnosed with Friedreich Ataxia (FA), they may be eligible for the BRAVE Study.

About the BRAVE Study.

The BRAVE Study is evaluating how safe a study drug is and how well it works for the treatment of Friedreich Ataxia (FA) for children aged 2–15. The study drug is the only approved treatment available for FA in adults and adolescents 16 years and older.
The study drug is an oral capsule and will be compared to a placebo. A placebo looks just like the study drug but does not contain any active ingredients. Your child will be randomly assigned to take either the study drug or placebo once daily.

Your child will have a higher chance of receiving the study drug than the placebo. Neither you, your child, nor the study team will know which treatment they have been assigned to, but in case of an emergency, the study team can quickly find out.

Study design.

The BRAVE Study may last up to three years and is made up of
two parts.

Part 1

• Screening (28 days) – The study team will determine if your child is able to join the study. There will be one study site visit.
• Treatment (about one year) – Your child will be randomly assigned to receive the study drug or placebo as an oral capsule. The capsule will need to be taken once daily with a parent or caregiver’s help. There will be six study site visits, one home (or study site) visit, and one phone call during the treatment period. There will also be a follow-up phone call about one month after the last study site visit for those not participating in Part 2. If needed, the study team may schedule an additional visit for your child.

If your child completes Part 1 of the study, they may be able to enter Part 2. All participants in Part 2 will receive the study drug, even if they received the placebo in Part 1.

Part 2

• Open-Label Extension (about two years) – Your child will be guaranteed to receive the study drug. During the first three months, there will be two study site visits, one home (or study site) visit, and one phone call. Then, there will be study visits about every six months until the study ends. There will also be a follow-up phone call about one month after the last dose of study drug. If needed, the study team may schedule an additional visit for your child.

Study visits.

Throughout the study periods, the study team will perform standard tests and assessments to ensure the health of your child and to find out if their assigned study treatment is working.

Some of these tests and assessments include:

• Modified Friedreich Ataxia Rating Scale (MFARS) assessmentsto measure your child’s FA progression
• Blood and urine sample collections
• Measurements of blood pressure, pulse rate, and body temperature
• Height and weight measurements
• Pregnancy tests (if applicable)
• Questionnaires about how your child is feelingEchocardiograms (ECHOs) and electrocardiograms (ECGs)
  see how well your child’s heart is working

These tests and assessments are common in clinical research studies and are done at no cost to you. The study team will monitor your child’s health over the entire length of the BRAVE Study. Your child will receive the same level of care regardless of whether they are receiving the study drug or placebo.

About the study drug.

The study drug is called omaveloxolone and has been approved for the treatment of FA in adults and adolescents 16 years and older. The BRAVE Study is evaluating the safety of omaveloxolone and how well it works for the treatment of FA for children aged 2–15.

The study drug or placebo may be given orally or, if your child is unable to swallow whole capsules, it may be opened. Once open, the contents can be sprinkled onto applesauce or another approved food item, which should be confirmed with your child’s doctor and stirred until it Is mixed.

Study cost.

Neither you nor your insurance provider will be charged for the costs of any tests or procedures performed as part of the clinical study. Travel support may be available if needed.

See if your child may qualify.If you are interested in your child joining the BRAVE Study,
they must:

• Be between 2 and 15 years old
• Have a diagnosis of FA
• Not have uncontrolled diabetes
• Not have a history of liver disease
• Mild or moderate heart damage

There are more requirements to join the study, which can
be explained by the study team.

What you should know aboutclinical research studies.

Clinical research studies aim to answer specific questions
about how medicines may work. These studies are the way new
treatments are approved to become accessible for patients
who need them in the future. You should feel fully informed about what to expect from participation in a clinical research study.

Researchers use clinical research studies to:

• Answer specific health questions
• Learn about the effects and safety of a study drug

Regulations and policies have been developed to help protect the rights, safety, and well-being of people who take part in clinical research studies and to help ensure that these studies are conducted according to strict scientific and ethical principles. Before a clinical research study can begin, an institutional review board (IRB) or ethics committee (EC) must review and approve the study.

Participation in any clinical research study is completely voluntary, and you or your child may withdraw from a clinical research study at any time for any reason. Before participating in a clinical research study, it is important to weigh the potential risks and benefits of participation. The study team will inform you of the potential risks and benefits of study participation, as well as possible side effects. To make an informed decision, gather as much information as possible and talk to your or your child’s healthcare providers about any questions you may have.

During the study, you will work with a study team that may include study doctors, study nurses, and other research staff.

The information on this page is based on a downloadable brochure.

Download Brochure

For further information: There are two bilingual research centers participating in the study.

Quebec City

CHU de Quebec – Laval University

Principal investigator:

Dr. Nicolas Chrestian

Coordinator: Davy Eng davy.eng@crchudequebec.qc.ca

418-525-4444 ext. 71801

Montreal

McGill University

Principal investigator:

Dr. Maryam Oskoui

Coordinator: Mbaye Ndiaye Mbaye.Ndiaye@MUHC.MCGILL.CA

514-934-1934, ext. 37299