Clinical Trials

Although guidelines for the treatment and management of anti-NMDA receptor encephalitis exist, currently, there is no specific treatment for patients with anti-NMDA receptor encephalitis, also referred to as autoimmune encephalitis (AE). Patients are treated with off-label medications such as cortisone and immunotherapies also used in oncology. These medications aim to reduce the number of circulating autoantibodies and to suppress the autoimmune process. Due to its rarity, there are few clinical trials available. Currently, there are only a limited number of clinicals trials recruiting patients.

Find out more about research and clinical trials specific to autoimmune encephalitis at:
www.clinicaltrials.gov

ClinicalTrials.gov is a registry of clinical trials. It is run by the United States National Library of Medicine at the National Institutes of Health and is the largest clinical trials database.

Clinical Trials Q and A

What are clinical trials?
Clinical trials are studies using human volunteers, looking at new ways to prevent, detect, or treat disease. The goal of clinical trials is to determine whether a new treatment is safe, and whether it works. No new drugs or treatments are approved for human use without a rigorous clinical trial to show safety and efficacy. Clinical research is conducted according to specific protocols, carefully planned to safeguard the participants’ health, and answer specific research questions. A protocol describes the following:

  • Who is eligible to participate in the trial;
  • The length of the study and what information will be gathered;
  • The objective, design, approach, statistical considerations, and organization of the trial.

A clinical study is led by a principal investigator (PI), who is often a doctor. The research team regularly monitors each participant’s health to determine the study’s safety and effectiveness. Clinical trials are conducted in “phases.” Each phase helps researchers answer different questions and is vital to provide sufficient evidence to receive approval from the U.S. Food and Drug Administration (FDA). For a treatment or therapy to become available to the public, it must pass through three phases. If the FDA approves, researchers will continue ongoing evaluation of safety and effectiveness.

How are trials supervised?
Clinical trials are closely supervised by regulatory authorities. Most clinical trials in the United States are approved and monitored by an Institutional Review Board (IRB). An IRB is an independent committee, often based in the treating hospital, that consists of physicians, statisticians, and members of the community who ensure that clinical trials are ethical, and the rights of participants are protected.

What is informed consent?
Informed consent is the process of providing people the facts they need to know about a clinical trial before they decide to participate. The informed consent document describes the study’s purpose, duration, required procedures, risks, and potential benefits. The participant decides whether to sign the document. Informed consent is not a contract. You are free to withdraw from the study at any time, or to refuse treatments or tests.

Why do people participate in clinical trials?
There are many reasons people decide to join a clinical trial. People participate in trials to receive the newest treatment and to access additional care from the clinical trial team. Others want to help accelerate science as many studies are delayed or fail to get off the ground due to lack of volunteers.