Frequently Asked Questions
The NF Clinical Trials Consortium was formed to make it easier to conduct clinical trials for patients with neurofibromatosis. The consortium brings together several clinical centers around the U.S. and one international site (Australia) with special expertise in management of neurofibromatosis and links them together with a central Operations Center. The Operations Center helps to organize the trials and ensure that all of the necessary regulations regarding clinical trials are followed. Having multiple centers participating around the country makes it easier to recruit a sufficient number of participants to obtain reliable conclusions.
A clinical trial is a research study designed to answer the question of whether a particular approach to treatment is or is not effective. A clinical trial follows a strict protocol that is required to ensure that a reliable conclusion can be reached.
Yes. The Consortium is now set up to conduct trials for patients with NF1, NF2, and Schwannomatosis.
Because of the importance of strict adherence to a clinical trial protocol, it is necessary to travel to a consortium patient recruitment site in order to participate in a clinical trial. Unfortunately, there are no funds available to cover the costs of travel to a patient recruitment site.
Why are there no patient recruitment sites in some parts of the country?
Patient recruitment sites were selected on the basis of ability to recruit participants and effectively conduct trials. This has resulted in some parts of the U.S. not having a nearby site. Although this makes it difficult for people in some regions to participate, the overall goal was to ensure that the Consortium would be able to recruit sufficient numbers of participants to ensure a successful trial. In the long run, this benefits the entire community, by ensuring that results can be obtained as quickly and reliably as possible.
In some cases, some tests, such as blood tests, can be arranged by a local physician. It is necessary, however, to travel to a Consortium patient recruitment site in order to obtain study medication and have routine follow-up examinations. Unfortunately, there is no funding available to cover the costs of travel.
There are no rules that prevent persons who live outside the U.S. to participate in a Consortium trial. The need to travel to a patient recruitment site, however, may make participation difficult for such people.
There are no charges for participation in a clinical trial or for access to study medications. Some trials may involve billing insurance for some routine tests while a patient is involved in a trial, and routine clinical visits or tests done during a trial would be billed in the usual manner.
Any experimental treatment has potential risks and benefits. These will be explained in detail by the study team as part of the informed consent process. You can ask questions about risks at any time and can withdraw from a trial at any time for any reason.
Each clinical trial has specific eligibilty rules, including the age range of participants. Some trials are open to participation by children.
The informed consent process involves education about the procedures involved in participation in a clinical trial as well as the risks and benefits. This education will be provided by the study team at a patient recruitment site. You will be given the opportunity to ask questions and take the information home to think about the clinical trial. When you are ready, you will then be asked to sign a form indicating that you understand these things and are willing to participate in the trial. If the participant is a child, the parent(s) will be asked to sign for the child; if the child is old enough, he or she will be asked to sign a form indicating willingness to participate (assent form). The study protocol and informed consent process are closely monitored by the Institutional Review Board (IRB), which is responsible to ensure that research involving human participants is conducted ethically.
Why are the rules for entry into a clinical trial so strict? Can I participate even if I do not exactly qualify for a trial?
It is critical that the study protocol be followed exactly in order to ensure that reliable results are obtained. Although it may seem unfair that persons who do not precisely fulfill eligibility criteria cannot participate, remember that the study medications have not been proved to be effective and therefore no one is being denied access to a treatment that is known to be effective. It is in the best interests of all people in the NF community to ensure that the trials are conducted carefully, so as to identify effective treatments and exclude ineffective treatments as quickly as possible.
Each trial has a predetermined set of goals that must be met before the trial is concluded. In some cases, this requires waiting from months to a few years to see if participants experience a benefit. The results of the trial will not be released until a definitive conclusion is reached. The trial progress is monitored by an external Data Safety and Monitoring Board to ensure that the trial is closed if the treatment is found to have unacceptable side effects, or to end the trial early if it becomes clear the the treatment either works or does not work. Eventually, the results of each trial will be published in medical journals.
It is the goal of the Consortium to obtain clear results of benefit or lack of benefit as quickly as possible, but the actual time required depends on the behavior of the aspect of NF being treated. This can mean waiting times as short as a few months or as long as several years. Once a trial is concluded, if positive results are achieved, it can still take time to satisfy all of the regulatory requirements to establish a routine treatment.
The U.S. Army Medical Research and Materiel Command has an active program in supporting NF research. The NF Consortium is one of many research projects supported by the program. For more information, please go to their website (http://cdmrp.army.mil/nfrp/default.htm).