Research Participation FAQs

Clinical trials and studies are an exciting and important opportunity for people with NF1 and NF2 to make a difference in their own lives as well as the lives of others affected with NF1 and NF2. When you participate in a clinical trial or study you are:

  • Receiving the most advanced care
  • Giving to future generations of people living with NF1 and NF2
  • Helping to change the way we practice medicine

Your dedication to participation, along with the tireless work of the physicians and researchers at the Washington University NF Center, makes the hope for a better future for people with NF1 and NF2 a reality. Together, we aim to:

  • Find the best possible treatments for people living with NF1 and NF2
  • Make life better for future generations of people living with NF1 and NF2
  • Change the way we practice medicine

Have questions about participating in a research study or trial? Review these research participation FAQs to learn more!

What is a clinical trial or clinical study?

Clinical trials and clinical studies are health-related research projects designed to identify the best ways of caring for people living with medical conditions like NF. While the terms “clinical study” and “clinical trial” are sometimes used interchangeably, there is a difference between these two types of projects.

Clinical research studies examine specific features of NF to learn more about how to predict who will develop certain NF-related problems and how these problems affect people’s lives.

In contrast, clinical trials aim to determine how drugs and other therapies can prevent or treat medical problems seen in individuals with NF1 and NF2. Examples of clinical trials include the use of the drug Lovastatin to treat learning deficits in children with NF1 or cancer therapies, like Gleevac and Rapamycin, to reduce plexiform neurofibroma growth.

Why should I participate in research?

You have the potential to improve the way your physicians treat people affected with NF1 and NF2. Without clinical trials and clinical studies, there will be little medical progress.

  1. You get the most advanced care – Clinical outcomes for patients involved in clinical trials are better than outcomes for patients who are not enrolled in clinical trials. This is because patients who are involved in clinical trials are receiving cutting edge therapies. Yes, it is true that the medications may still be in a trial phase; however, since the government has rules in place that make it so only the most promising new treatments ever become available to people, those individuals who participate in clinical trials are receiving a medication that may one day become a standard medication for the care of people with NF1 and NF2.
  2. You can give back – Participating in clinical trials and clinical studies allows individuals with NF1 and NF2 to create a better life for future generations of individuals with NF, including their own family members. What physicians and researchers learn today can only make the medical treatment options of tomorrow better.

What are the benefits to participating in clinical studies and trials?

When you participate in a clinical study or trial you are:

  • Receiving the most advanced care available
  • Giving to future generations of people living with NF
  • Helping change the way we care for individuals affected by NF

How can I become involved in clinical trials and clinical studies?

There are always exciting opportunities to get involved in clinical studies and clinical trials at the Washington University NF Center. Check out our listings of current clinical studies and current clinical trials to see if there is a study or trial that would benefit you. Additionally, you can explore the clinical studies regularly updated by the National Institutes of Health (NIH) at http://clinicaltrials.gov/.

Why are clinical trials and clinical studies the best way to improve medical treatment of NF?

Clinical trials and clinical studies provide physicians and researchers with quantifiable data—or information that has been specifically designed to result in unbiased conclusions. Quantifiable data follow strict rules and rely on numbers and factual observations.

When studies provide qualitative data—or information that comes from anecdotes or stories—there are no rules to define how that information is used or interpreted. This makes it easy for medical professionals to come to false conclusions.

Because clinical trials and clinical studies rely on quantifiable data, they are the most likely forms of research to produce unbiased results that will ultimately improve the medical treatment options available for people living with NF1 and NF2.

Does participation in a clinical study or trial make a person a “guinea pig”?

People who participate in clinical trials and clinical studies are NOT experimental “guinea pigs”. Here are the reasons why:

  1. All clinical trials and clinical studies must be approved by an Institutional Review Board (IRB). An IRB is a group of physicians, scientists and community advocates who are not involved in the specific clinical trial or study being reviewed. IRBs were developed to ensure that every study is ethical and preserves the individual rights of every participant. In addition, clinical trials that involve medications are regulated by government agencies, such as the Food and Drug Administration (FDA). All medications must undergo multiple phases before they are ever taken by a person during a clinical trial. These systems are put in place to ensure that people are treated as human beings, and that only the most promising new treatments and studies will ever become available to individuals.
  2. Before anyone participates in a clinical trial or study, the researcher is required to obtain informed consent. Informed consent requires the researcher to provide potential participants with a document that clearly explains what the study aims to accomplish and outlines any potential risks associated with the study, no matter how major or minor those risks might be. When participating in a clinical trial or study at the Washington University NF Center, all participants receive both written and verbal informed consent. Individuals interested in participating are encouraged to speak with any member of Team NF about any questions or concerns they might have.
  3. All clinical trials and clinical studies are strictly voluntary and participants are free to leave the study at any time. There are never any consequences to individuals who decide to leave a study for any reason.

Why does treatment for NF need to be improved?

While we always provide the best possible care for individuals with NF1 and NF2, it is important to understand that just because the treatment offered is currently the best one available, it does not mean that it is the best possible therapy. Unlike most common medical conditions that have a standard plan of care—or a known most effective therapy—there is no standard plan of care for the treatment of NF1 and NF2.

Because NF is a complex set of genetic conditions that affect no two individuals exactly the same way, physicians and researchers must work together to find the most effective way to treat each individual living with NF. The promise of personalized medicine – or treatments designed specifically for certain people and certain features seen in NF1 or NF2 – may emerge from the pioneering studies underway at the Washington University NF Center.

What are the types of clinical trials?

Clinical trials can be broken into subgroups based on their plan of action. Some examples include:

  • Treatment trials – Treatment trials test new treatments for NF and might include new medications, new physical or occupational therapies, or new approaches to surgery.
  • Prevention trials – Prevention trials look for ways to prevent NF-specific traits from developing or progressing. These approaches may include medicines, vitamins, or other lifestyle changes.
  • Diagnostic trials – Diagnostic trials are conducted to find tests or procedures for diagnosing NF.
  • Screening trials – Screening trials are designed to find the best ways to detect NF1 and NF2 as well as NF-related conditions.
  • Quality of life trials – Quality of life trials explore ways to evaluate and improve the quality of life for individuals with NF.

What are the phases of clinical trials?

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.

  • Phase I (1) trial – In a phase I trial, only a small group of participants are recruited. Phase I is the first time that a new drug or therapy is tested with human participants. Determining the safety of a drug is the main objective of phase I. Most children are NOT asked to participate in phase I trials. 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.
  • Phase II (2) trial – In a phase II trial, a larger group of participants is recruited. The purpose of a phase II trial is to determine the effectiveness of the new drug or therapy. Children ARE asked to participate in phase II trials.
  • Phase III (3) trial – In a phase III trial, the largest number of participants is recruited. The purpose of a phase III trial is to compare the new drug or therapy to a commonly used treatment.
  • Phase IV (4) trial- Phase IV trials are conducted after the drug or treatment has been marketed. The purpose of a phase IV trial is to monitor the effectiveness of the approved intervention in the general population. Phase IV trials are also used to collect information about any adverse effects associated with the drug or treatment’s widespread use.

Who can participate in clinical trials?

Not everyone with NF will qualify for a clinical trial. Every clinical trial will have guidelines regarding who does and who does not qualify to participate. These criteria are not intended to exclude anyone, but rather are used to identify appropriate participants and to preserve the overall safety of all participants. If you are interested in participating in a clinical trial or study, please speak with the members of Team NF to learn if you are eligible for participation.

I am interested in learning about other research at the Washington University NF Center.

In addition to clinical research, the Washington University NF Center conducts basic laboratory research to find better ways to manage NF1-associated optic pathway gliomas, malignant cancers, learning and attention deficits, as well as NF2-associated tumors. To learn more about these exciting projects please visit the Washington University NF Center Laboratory Research page. Additionally, you can read our recent publications and select review articles.