Clinical Studies

Clinical Studies at the NIH Clinical Center

Rheumatology fellows and advanced trainees participate in the numerous clinical trials ongoing at the NIH Clinical Center, the country's largest medical center exclusively devoted to clinical research. Patients are recruited into the NIH clinical trials not only from the local area, but nationally, and even internationally, depending on the rarity of the disease under study. Clinical studies at the NIH fall into two categories:

  1. natural history studies, in which patients come to the NIH for evaluation and follow up to learn more about the pathogenic mechanisms and genetic causes of their disease, and
  2. interventional studies, in which investigational agents are used to treat a cohort of patients with a specific disease

Our investigational clinical trials tend to be early stage phase II trials that also test hypotheses about disease mechanisms, rather than large, later stage company-sponsored multi-center trials.

A listing of current clinical trials at the NIAMS can be found here.

See patients enrolled in a NIAMS lupus trial and a vasculitis trial tell their stories:

Selected Achievements in Rheumatology Clinical Research the NIH

Clinical and translational researchers at the NIAMS and other Institutes at the NIH have made major contributions to the understanding and treatment of rheumatic disease over many years. The use of cyclophosphamide to treat vasculitis and lupus nephritis, which has become part of the standard of care around the world, was pioneered at the NIH in the 1970s and 1980s. Under the leadership of our faculty member Dr. Dan Kastner, the genetic causes of and recognition of the shared features of diseases now known as autoinflammatory diseases were discovered. Many of these discoveries were celebrated during the 25th anniversary of the founding of the NIAMS in 2011, and an interactive timeline of those discoveries can be found on the NIAMS 25th Anniversary Milestones. Noteworthy discoveries made at the NIH in rheumatic diseases include:

  • Identification of the gene which causes Familial Mediterranean Fever, published in Cell in 1997.
  • Identification of the JAK3 kinase as a key mediator of cytokine signal transduction, which led to the development of JAK kinase inhibitors, which are being developed for the treatment of rheumatoid arthritis and related conditions.
  • Identification of mutations in the TNF-receptor 1 gene underlying the periodic fever syndrome TRAPS, published in Cell in 1999.
  • Demonstration that blockade of the cytokine interleukin-1 is efficacious in diseases linked to mutations in the gene encoding NALP3, a key component of the inflammasome enzymatic complex that processes Interleukin 1-beta. Findings were published in the New England Journal of Medicine.
  • Discovery of the disease DIRA, in which patients suffer from inflammation as a result of deficiency of the interleukin-1 receptor antagonist gene.
  • Discovery that IgE autoantibodies and basophils may be important in the pathogenesis of SLE, as published in Nature Medicine.


New findings and discoveries in rheumatic diseases are highlighted in the NIAMS Intramural Research Program Facebook page Exit disclaimer icon..

This page last reviewed on July 17, 2020