Cold Agglutinin Disease (CAD) Clinical Trials and Treatments

Cold agglutinin disease (CAD) is a rare autoimmune hemolytic anemia caused by pathogenic IgM autoantibodies reactive at cold temperatures (typically <4°C but sometimes up to 18°C) that bind red blood cell surface antigens, usually I or i blood group antigens. These IgM-coated red blood cells activate the complement cascade (classical pathway via C1q binding), leading to membrane attack complex formation and intravascular hemolysis. Patients experience hemolytic anemia with symptoms and severity exacerbated by cold exposure; even minor cold contact triggers hemolytic episodes.

The most common symptoms of Cold Agglutinin Disease include Hemolytic anemia and fatigue, Symptoms worsened by cold exposure, Jaundice and dark urine, Acute hemolytic episodes in cold weather, Acrocyanosis and Raynaud phenomenon, among others. Estimated at 1 in 300,000 to 1,000,000 people. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Cold Agglutinin Disease from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Sutimlimab (Enjaymo), an antibody that blocks the complement protein C1s at the start of the classical pathway, was FDA approved in February 2022 and since January 2023 has been approved for the treatment of hemolysis (red blood cell destruction) in adults with CAD. Recordati Rare Diseases bought Enjaymo from Sanofi in November 2024.

Yes. As of September 2026, Trial Friend lists 1 FDA-approved drug used to treat Cold Agglutinin Disease: Enjaymo (sutimlimab-jome), first approved in 2022.

Each has its own page with what the label covers, side effects and how it is given, and the Treatments section of this page adds any further approved drugs found in FDA labeling data.

Cold Agglutinin Disease is an acquired condition, not an inherited one. Caused by IgM autoantibodies; secondary CAD follows infection or lymphoproliferative disease. No germline gene test. Estimated at 1 in 300,000 to 1,000,000 people.

Eligibility for Cold Agglutinin Disease clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Cold Agglutinin Disease clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.