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If you are interested in learning more about the availability of clinical trials please visit www.clinicaltrials.gov. This site is maintained by the National Institutes of Health with the most up to date list of clinical trials.  Not all trials will be actively looking for participant and those that are may have certain criteria they are seeking. Please see below for a list of frequently asked questions related to clinical trials.  Click here to visit the NIH NF Clinical Trials page.

Choosing to participate in a research call or clinical trial is an important personal decision. You may wish to talk to your health care provider before you enroll. After you have identified some trial options, the next step is to contact the study research staff and ask questions about specific trials.

Continued advances in our understanding of neurofibromatosis (NF) and how to develop treatments for NF rely on information that scientists and physicians get from doing and basic research and clinical trials . You can help by participating in these studies! The following addresses some of the questions you may have.

What is a clinical research study?

Many scientists are doing basic NF research that needs tissue donations from people with NF. For example, blood samples might be used to study genetics. Samples from a surgically removed neurofibroma might be used to study tumor biology. Some studies don't need tissue samples but are 'natural history' studies that e.g. monitor a person's neurofibromas over time to understand how they grow. In other studies, volunteers might be interviewed or asked to complete surveys about living with NF, or have a magnetic resonance imaging (MRI) scan. All of these studies and others will eventually help doctors better understand NF and how we might develop new treatments.

What is a clinical trial?

A clinical trial is a test in humans of a new treatment for some aspect of a disease or disorder, to see if the treatment is safe and effective. NF clinical trials test new drugs as potential treatments to halt neurofibroma growth, treat learning disabilities or treat some other aspects of NF. These may be drugs never tested in humans before, or they may be drugs already being tested or prescribed for disorders other than NF.

Clinical trials are conducted in four phases:

Phase I trials are the first step in testing a new treatment that has never been tested in humans. These studies are done on a small group (20-80 patients), to ensure the drug can be given safely (what dose?), how it can best be delivered (e.g. pill? injection?), and to look out for any potential side effects. Phase I studies are also limited number of patients who would not be helped by other known treatments.

Phase II trials include a larger number of patients (100-300) and focus on learning whether the new treatment is effective (e.g. does it stop growth of neurofibromas?) Phase II studies are also a limited number of patients who would not be helped by other known treatments.

Phase III trials test the drug in a larger population (1,000-3,000) and compare the results when half of the volunteers receive the test drug, while the remainder receive the current standard' treatment. Where there is no standard treatment forma disorder (as is the case with NF), test drug is compared to a placebo (a look-alike pill that contains no active drug). To avoid bias, in these trials both the volunteer and the doctors overseeing the trials are typically 'blinded' as to whether or not they are receiving the active test drug. If drugs are successful in Phase II they will be marketed.

Phase IV studies are done after the drug or treatment has been marketed. These studies continue testing the study drug or treatment to collect information about their effect in various populations and any side effects associated with long-term use.

Where do clinical trials take place?

Clinical trials can take place in a variety of locations, such as hospitals, universities, doctor offices, or community clinics. Phase III trials are often multi-site, with patients being recruited and treated in a number of sites across the US or even around the world.

Who can participate in a clinical trial?

Each clinical trial will have inclusion criteria and exclusion criteria, guidelines about who does and does not qualify to participate. These criteria are not intended to exclude anyone, but rather are used to identify appropriate participants and keep them safe. Criteria are principally based on age, medical history, and current medical condition.

As well as volunteers with the disorder being studied, some studies seek healthy volunteers to participate in Phase I trials, vaccine studies, or preventive care studies.

How can I be sure a clinical trial is safe and approved?

Every clinical trial in the U.S. must be approved and monitored by the Institutional Review Board (IRB), an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research. IRB approval ensures the potential risks and benefits have been fully evaluated.

What are the potential benefits associated with clinical trials?

These include the following:

• You can gain access to new treatments that are not available to the public.
• You can obtain expert medical care at leading health care facilities during the trial.
• You can help others by contributing to medical research.

What are the potential risks associated with clinical trials?

These include the following:

• There may be side effects or adverse reactions to medications or treatments. These might include headache, nausea, hair loss, skin irritation, or other physical problems. Side effects may appear in the short- or long-term.
• The treatment may not be effective for you
• You may be required to devote a lot of your time for trips to the study site, treatments, hospital stays, or complex dosage requirements

What do I do if I want to participate in a clinical trial?

You should contact the staff person listed. You will be asked for certain information and to meet with the clinic staff so you can be evaluated to see if you are an appropriate candidate for the trial. This will also be your opportunity to ask questions about the trial. The trial staff will fully apprise you of all the details of the trial to ensure you understand them. This is called informed consent .

What is informed consent?

Informed consent is the process of learning the key facts (including those listed below) about a clinical trial before you decide whether or not to participate.

The trial staff will give you informed consent documents that include study details in English or your native language. You may wish to take the consent documents home and discuss them with family members or friends. If you decide to join the clinical trial, be sure to ask for a copy of the informed consent documents so you can review them at any time.

It is important to remember that informed consent is more than signing a form. It is a process that continues through the study. You should feel free to ask the research team questions before, during, and after the study. Informed consent continues as long as you are in the study.

What questions should I be sure to ask before I join a clinical trial?

You should feel comfortable asking questions of the clinic staff and feel you are getting answers you understand so that you are as informed as possible.

Before enrolling in a trial, you may want to know:

• What is the purpose of the study?
• How long is the study?
• Why does the research team think the treatment will work?
• What kinds of procedures or tests will I have?
• Will they hurt? What other treatment options do I have?
• How do the possible risks and benefits of the study compare with approved treatments for me?
• Will the study researchers work with my doctor while I am in the study?
• Can I talk to other people in the study?
• Can anyone find out that I am participating in a study?
• What are the possible immediate and long-term side effects?
• Will I be able to find out the results of the trial?
• Who will provide my medical care after the study ends?

What questions should I ask about my responsibilities before I join a clinical trial?

It is important that you fully understand what will be expected of you before you enroll in a trial. Again, you should feel comfortable asking questions of the clinic staff.

Before enrolling in a trial, you may want to know:

• What are my responsibilities if I enroll?
• What medications, procedures, or treatments must I avoid?
• Will I be able to take my regular medications?
• Where is the study site and how often do I have to go there?
• Will I have to be in the hospital for any period?
• Will I have to pay anything to participate in the study?
• What are the charges likely to be?
• Will my insurance cover those expenses?

What happens during a clinical trial?

You will work with a clinical trial team that will include doctors, nurses, social workers and other health care professionals. They will check your health at the beginning of the trial, give you specific instructions for participating in the trial, monitor you carefully during the trial, and stay in touch with you after the study. Some trials involve more tests and doctor visits than you would normally have for your regular health care. Your participation will be most successful if you carefully follow any instructions given, and stay in contact with the research staff.

Can I leave a clinical trial after it has begun?

Yes. You can leave a clinical trial at any time. If you plan to stop participating, let the research team know why you are leaving the study.

Who pays for clinical trials?

Clinical trials are sponsored by a variety of sources. These include government agencies such as the National Institutes of Health (NIH); pharmaceutical companies; health maintenance organizations (HMOs); and organizations that develop medical devices.

Will I be paid for participating in a clinical trial?

Some clinical trials will pay you for joining the trial, while others will not. You may be reimbursed for transportation costs, childcare, meals, and accommodations.