Clinical Trials in NMO

How Do Clinical Trials Work?

At present there is no definitive “cure” for neuromyelitis optica (NMO). Clinical research is seeking to find better ways to prevent, diagnose, treat, and eventually cure human disease. These advances come with improved understanding of the causes and effects of the disease.

Clinical trials are designed to determine which medicines or procedures best benefit patients, and which may not. These studies often involve expert teams from academic, governmental and pharmaceutical sectors. In some cases, clinical trials seek to test the efficacy of a new drug for a disease which has no proven effective therapy. In other trials, one treatment is compared with another to examine which may be best in patients of differing disease stage or condition.

Clinical Trials Information

Learn more about ongoing clinical trials. Download our clinical trials fact sheet, “Randomized Clinical Trials Actively Recruiting Patients to Evaluate Drug Treatments for NMO/SD”.

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Clinical Trials Webinars

The clinical trials webinar series focuses on clinical trials in NMO, with participation from Industry Council members, including Alexion, Chugai, and Medimmune. Watch the videos, download the PDF presentations, or read the transcripts online.

What Type of Questions Might I Ask?

If you are considering enrolling in a clinical trial, it is important that you ask any questions or bring up any issues concerning the trial at any time. The following suggestions may give you some ideas as you think about your own questions:

The Study
  • What is the purpose of the study?
  • Who will fund the study?
  • Who has reviewed and approved the study?
  • How are study results and safety of participants being monitored?
  • How long will the study last?
  • What will my responsibilities be if I participate?
  • What are the possible risks and benefits?
  • What are my possible short-term benefits?
  • What are my possible long-term benefits?
  • What are my short-term risks, such as side effects?
  • What are my possible long-term risks?
  • Who pays for my medical care in relation to the trial?
  • What happens if I experience an adverse event in the trial?
  • Can I exit the trial at any time for any reason if I wish?
  • What other options do people with my disease have?
  • How do the possible risks and benefits of this trial compare with those options?
Participation and Care
  • What kinds of therapies, procedures and /or tests will I have during the trial?
  • Will they hurt, and if so, for how long?
  • How do the tests in the study compare with those I would have outside of the trial?
  • Will I be able to take my regular medications while in the clinical trial?
  • Where will I have my medical care?
  • Who will be in charge of my care?
Personal issues
  • How could being in this study affect my daily life?
  • Can I talk to other people in the study?

A key step in enrolling in a clinical trial is called informed consent. In this step, each potential subject will be carefully and thoroughly informed of the details of the trial, and encouraged to ask any questions about any aspect of the trial design, purpose, clinical investigators, or industry sponsor. Only when all questions or concerns have been addressed, and qualification is determined based on inclusion and exclusion criteria, is a patient invited to sign the informed consent document to begin enrollment as a clinical trial subject.

For additional details, see the Further Information section listed below.

How Am I Protected?
Ethical Guidelines

The goal of clinical research is to develop knowledge that improves human health or increases understanding of human biology. People who participate in clinical research make it possible for these advances to occur. The path to finding out if a new drug or medical procedure is safe or effective is to test it on patient volunteers. Clinical trials are carefully designed by experts, and subjected to judicious review by institutional review boards and regulatory agencies to minimize the risks and maximize the information likely to be gained. However, because there are uncertainties in a clinical trial, there is an inherent potential for bias or adverse outcomes. The purpose of ethical guidelines is both to protect patient volunteers and to preserve the integrity of the science. Ethical guidelines in place today represent lessons learned from historical experiences to improve the safety and reliability of clinical trials in the best interests of patients and all concerned.

Informed Consent

As described above, informed consent is the process of volunteer patients learning the key facts about a clinical trial before deciding whether to participate as clinical trial subjects. The process of providing information to subjects and addressing any questions or concerns continues throughout the study. To help someone decide whether to participate, members of the research team explain details of the study. The research team provides an informed consent document, which includes such details about the study as its purpose, duration, required procedures, and who to contact for various purposes. The informed consent document also explains risks and potential benefits.

Once an individual decides to enroll in a clinical trial, they are required to sign an informed consent document. It is important to note that informed consent is not a contract, it is a process, and all clinical study volunteers are free to withdraw from the study at any time for any reason.

Institutional Review Board (IRB) or Ethics Committee (EC) Oversight

Each clinical trial must be approved and monitored by an Institutional Review Board (IRB) or Ethics Committee (EC) to ensure that the risks are minimal and are worth any potential benefits. The IRB or EC is an independent committee that consists of physicians, statisticians, and members of the community including ethicists and in some cases clergy, who ensure that clinical trials are ethical and that the rights of participants are protected. Governmental regulations require all institutions that conduct or support biomedical research to have an IRB or EC initially approve, regularly monitor, and periodically review the research.

For additional details, see the Further Information section listed below.

What Is Guthy-Jackson’s Role in Trials?

The Foundation endorses clinical trials as a best means to evaluate new or better ways to prevent, diagnose, treat, and potentially cure NMO. However, as a non-profit charitable organization, we strictly do not endorse any specific clinical trial, clinical trial design, agent or experimental drug or procedure, or pharmaceutical company. Additionally, the Foundation does not participate in subject recruitment or eligibility evaluation for any trial, nor does it act or participate in any way in performance, data assessment, interpretation, regulatory agency review, or recommendations that may be derived from any clinical trial or its outcomes.

The role of the Foundation is to act solely as a facilitator of public information to the NMO community.