Anakinra for Behcet s Disease
- Behcet s disease (BD) is an autoimmune disease where the immune system attacks the body. People with BD may develop oral or genital ulcers, skin problems, and eye disease. Most drugs used to treat BD suppress the immune system, but they are not always helpful and may have side effects. A new drug, anakinra, may be able to treat BD with fewer side effects. Because it has not been studied in people with BD, anakinra is considered an experimental treatment.
- To test whether anakinra can be a safe and effective treatment for Behcet s disease.
- People who have active Behcet's disease, with an oral or genital ulcer within the past month, or three or more flares of eye disease in the past 6 months.
- Participants will be screened with a physical exam and medical history. They will also have blood and urine tests. They will be divided into two groups: those with oral or genital ulcers and those with eye disease.
- All participants will keep a diary of symptoms for a month before starting the study drug.
- Participants with oral or genital ulcers will receive daily injections of anakinra for 3 to 6 months. Treatment will be monitored with frequent blood draws and daily diaries. Those who improve but do not have a full response to the drug may receive a higher dose. Those who improve after 6 months may have an extra 6 months on either anakinra or placebo to study the differences in response.
- Participants with eye disease will receive anakinra for up to 12 months. Treatment will be monitored with frequent blood draws, daily diaries, and regular eye exams.
- All participants will have a final study visit 1 month after stopping the study drug.
Autoimmune/Connective Tissue Diseases
Immune System Diseases
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Primary Purpose: Treatment
|Official Title:||A Pilot Study of Anakinra in Behcet's Disease (BD)|
- Assess the time to disease flare in patients randomized to continuation of anakinra vs. withdrawal in BD for six months
- Clinical and biochemical indicators of inflammation, total number of responders, long term clinical and biochemical response, and safety. [ Time Frame: through month 13 ] [ Designated as safety issue: Yes ]
|Study Start Date:||September 2011|
|Estimated Study Completion Date:||October 2014|
|Estimated Primary Completion Date:||October 2014 (Final data collection date for primary outcome measure)|
Active Comparator: Group A
Open label followed by double blinded active drug
100 mg subcutaneously daily with dose escalation up to 200 mg daily for three months. Responders at months 3-6 will be randomised to continuation of drug vs. placebo
Placebo Comparator: Group B
Open label followed by double blinded placebo
Autoinflammatory diseases are illnesses characterized by episodes of inflammation that, unlike autoimmune disorders, lack the production of high titer autoantibodies or antigen-specific T cells. There is growing genetic and clinical evidence that Interleukin-1 (IL-1) plays a pathogenic role in several of these diseases. This exploratory study aims to examine the utility of anakinra in the treatment of adult subjects with Behcet s Disease (BD), a disease which shows similarities to the known anakinra-responsive autoinflammatory disorders, familial cold autoinflammatory syndrome (FCAS) and Muckle-Wells Syndrome (MWS). Anakinra is a recombinant form of the human interleukin-1 receptor antagonist that has been studied in RA and the autoinflammatory disorders. It has a half life of 4 to 6 hours with a FDA approved recommended dose of 100 mg/day subcutaneously for the treatment of rheumatoid arthritis.
This pilot study is designed to address: 1) the utility of anakinra in the treatment of BD; 2) the effect of anakinra on laboratory biomarkers in BD; and 3) an exploratory assessment of the safety of anakinra in individuals with Behcet s Disease.
Subjects with oral or genital ulcers will receive anakinra for three to six months. If five of the initial seven patients have a positive response, up to 20 patients with oral or genital ulcers will then be randomized to withdrawal or continuation of drug for six months once placebo is available. Patients with eye disease will be treated with anakinra for a total of twelve months without randomization to withdrawal. Clinical and biochemical correlates of inflammation will be measured at appropriate intervals to assess response and to further understand disease mechanisms.
|Contact: Elaine M Novakovich||(301) firstname.lastname@example.org|
|Contact: Peter C Grayson, M.D.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL) 800-411-1222 ext TTY8664111010 firstname.lastname@example.org|
|Principal Investigator:||Peter C Grayson, M.D.||National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)|