Alport Syndrome Study – Children Ages 12-17

STUDY BASICS

Are you the parent of a child ages 12-17 who has been diagnosed with (or who is suspected of having) Alport syndrome? If so, your child may be able to participate in a research study to find out if a medication called bardoxolone methyl is safe and can help improve the symptoms of Alport Syndrome. Compensation provided.

STUDY PURPOSE

Alport syndrome is a rare genetic condition that leads to problems with the kidneys, eyes, and ears. Symptoms of Alport syndrome can range from mild to severe, and may include blood or protein in the urine, high blood pressure, swelling in the lower limbs, hearing loss, and vision problems. Over time, symptoms become worse and most people develop life-threatening kidney disease. While medications can help slow the progression and relieve some symptoms of Alport syndrome, there is currently no treatment for the underlying disorder. The purpose of this study is to help researchers find out if a medication called bardoxolone methyl is safe and can improve the symptoms of Alport syndrome. While bardoxolone methyl has been tested in over 2,600 patients with diseases including type 2 diabetes, chronic kidney disease, cancer, and pulmonary arterial hypertension, the drug is not currently FDA-approved and is considered to be investigational in this study.

COULD THIS STUDY BE RIGHT FOR YOUR CHILD?

• Ages 12-17
• Diagnosed with Alport syndrome, or suspected of having Alport syndrome
• Able to swallow capsules

WHAT PARTICIPANTS CAN EXPECT

Participation in this study involves an initial 2-visit screening period to make sure your child is eligible for the study. If eligible after screening, your child will be randomly assigned (like the flip of a coin) to take a once-daily dose of the study drug or a placebo for 100 weeks. The placebo looks and tastes like the study drug, but does not contain any active medication. During the first year, your child will have 10 clinic visits. You/your child will also be called by the study staff. During the second year of the study, your child will have 5 study visits. Tests and assessments during the study may include the following: questionnaires, physical exams, pregnancy test, electrocardiogram, vision and hearing tests, blood and urine collection, and genetic testing (if not previously documented).