Clinical Trials

Clinical trials are an exciting new opportunity for individuals who have neurofibromatosis (NF). A clinical trial is a research study that relies on human volunteers to answer specific health questions. While surgery and other treatments for persons with NF are standard, a clinical trial allows individuals to play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research.

Potential clinical trials in NF might involve the testing of new drugs to treat learning disabilities or the testing of new drugs to stop or slow the growth of neurofibromas or meningiomas. Sometimes the drugs have been used before with other disorders, but not specifically for problems associated with NF. Other times, the drugs being tested in the clinical trial may have never been tried in adults or children before.

Clinical trials involve the testing of new drugs in an orderly series of steps, or phases. The phases are designed to determine safety, effectiveness and appropriate dosing of the drug. This allows doctors to obtain reliable information about the drug and, at the same time, protects the patient. Clinical trials are classified into one of three phases:

  • Phase I trials evaluate how a new drug should be given, how often it should be administered, and what dose is safe in children or adults. These trials typically involve small numbers of patients.
  • Phase II trials continue to test the safety of a new drug, and evaluate how well the new treatment works.
  • Phase III trials test a new drug, new combination of drugs, or a new surgical procedure in comparison to current therapy. Patients will usually be assigned to either the current therapy group or the new treatment group at random. These trials often involve large numbers of patients and may involve physicians at many different medical centers.

Each clinical trial must be approved and monitored by an independent committee of physicians, scientists, community advocates and others to ensure that the study is ethical and the rights of the patients are protected. This committee is called an Institutional Review Board.

NF clinical trial participation

Not everyone with NF will qualify for a clinical trial. Every clinical trial will have guidelines about who does and who does not qualify to participate. These criteria are not intended to exclude anyone, but rather are used to identify appropriate patients and preserve the overall safety of patients.

You do not have to participate in a clinical trial. Before you can participate, the research study team will discuss the rationale for the study, what is being done, what is required of you to participate, the potential benefits and side effects, and costs of participation. At this point, you may choose to participate or choose not to participate in any particular clinical trial.

Over the next several years, there will be additional therapies available for children and adults with NF. Our ability to determine whether these new treatments are safe and effective requires patient participation. It is our hope that these new treatments will be more effective than our current therapies, and will lead to improvements in the lives of our patients with NF.

You can learn more about clinical trials for people with NF by checking this website. If you are interested, you should contact the physician in charge of the study.

For more information on clinical trials in general, an excellent resource from the National Institutes of Health can be found at