Cold Agglutinin Disease Clinical Trials

Emily Malcolm, PhD avatar

by Emily Malcolm, PhD |

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Patients with rare conditions like cold agglutinin disease (CAD) can help with the development of new treatments by participating in clinical trials. This is also a way for patients to get access to new medications in development that are not otherwise available.

What are clinical trials?

Clinical trials are research studies in which human volunteers try medications under controlled circumstances to determine whether the treatment is safe and effective. They are an important part of the process for getting medications approved for use. Clinical trials also help doctors and researchers determine what side effects a therapy may cause and whether any other medicines might interact with the new treatment.

The U.S. Food and Drug Administration (FDA) has to approve a clinical trial’s design before it can begin. To do this, the medication must first be tested in a model of the disease in question, usually an animal model. If these preclinical studies are successful, the FDA may approve a human clinical trial.

What are the advantages of participating?

Patients can get access to a medication for their condition that is not available on the market, at no cost.

The clinical trial generally covers the cost of medical care during the trial, the medication it’s testing, and any tests or aftercare you may need. Many clinical trials also reimburse patients’ travel costs to and from the trial site.

What are the disadvantages of participating?

Many clinical trials include a control group that does not receive the medication being tested, for comparison. If you sign up for a double-blind clinical trial, neither you nor the physicians running the trial will know whether you are receiving the medication or a placebo. In some cases, instead of a placebo, the control is standard care. In open-label trials, participants know which medication they are receiving.

The biggest potential disadvantage is that you are taking an experimental treatment that hasn’t been tested in humans before. There may be side effects that can be painful or dangerous. If severe side effects occur, you may have to withdraw from the trial. Until a clinical trial is done, researchers can’t be sure what side effects are likely.

How can I find out if I’m eligible for clinical trials?

Talk to your doctor. They can find out whether you are eligible for a clinical trial. They can also advise you on whether or not a potential treatment is likely to be helpful for you.

You can find a list of CAD clinical trials that are recruiting or preparing to recruit participants on the National Institute of Health Library website.


Last updated: May 21, 2020


Cold Agglutinin Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.