DIRA clinical trials

Rilonacept for Deficiency of the Interleukin-1 Receptor Antagonist (DIRA)

Background: - Deficiency of the IL-1 receptor antagonist (DIRA) is a condition that causes repeated episodes of inflammation. People with DIRA can have rashes, fever, and joint pain. Most treatments for DIRA are intended to control the immune system to stop these inflammations. There are drugs that can treat DIRA, but they have to be given daily as injections. Researchers want to try another drug, rilonacept, as a treatment for DIRA. It needs to be given only once a week. Rilonacept will be given to individuals who are at least 3 months old and who have DIRA. Objectives: - To test the safety and effectiveness of rilonacept for children and adults with DIRA. Eligibility: - Individuals at least 3 months old who have DIRA. Design: - Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Other tests to study pain and movement will be given. Imaging studies such as bone density scans and x-rays may also be taken. - Participants will have a minimum of four to five study visits over 12 months. Those who are on different anti-inflammatory drugs (such as anakinra) will stop taking them before beginning the study visits. - Participants will have rilonacept injections weekly while on this study. The dose will be adjusted as needed to help treat the DIRA symptoms. Participants will keep a diary to monitor their symptoms and any side effects. - Treatment with rilonacept will be given for 1 year. Participants will have study visits to monitor the treatment. They will provide blood samples and have other tests at these study visits.