Clinical trials are essential for testing the effectiveness of promising new therapies to improve the lives of people with neurofibromatosis. Many people living with NF are interested in advancing research through participation in clinical trials.

Patients in the UAB Neurofibromatosis Program benefit from access to leading-edge treatments through participation in clinical trials for all forms of NF. As a national leader in clinical trials research, UAB serves as the coordinating center for the NF Clinical Trials Consortium (link). This collaborative group of 15 primary and 10 affiliate clinical centers across the country and in Australia is dedicated to conducting clinical trials of the most promising drug therapies for all forms of neurofibromatosis.

What is a Clinical Trial?

A clinical trial is a type of clinical research designed to evaluate and test new treatments using human volunteers who meet specific study criteria. Stringent rules govern clinical trials, which are monitored by the National Institutes of Health and the U.S. Food and Drug Administration. Before the FDA approves a clinical trial, scientists perform laboratory tests and studies in animals to determine a potential therapy’s safety and efficacy. If these studies show favorable results, the FDA gives permission for the medication or device to be tested in humans.

How Do I Find a Clinical Trial

The website clinicaltrials.gov, maintained by the NIH U.S. National Library of Medicine, is the primary resource to learn about current and upcoming clinical trials in the U.S. and internationally. Staring at the home page, users of the site can search for clinical trials by disease or condition, location, and key terms, such as a drug or investigatory name. The federal government requires trials to be registered and to include detailed information on the site about eligibility criteria, site location, primary outcome measures, and other information.

Who is Eligible to Participate in a Clinical Trial?

Clinical studies have specific requirements, called eligibility criteria. These criteria outline who can participate in the study. Inclusion criteria are the requirements that allow someone to participate, while exclusion criteria are the factors that disqualify an individual from participating.

Inclusion criteria for a trial might include factors such as: age; stage of disease; sex; genetic profile; and family history. Exclusion criteria might include factors such as specific health conditions or medications that could interfere with the treatment being tested.

These criteria are not arbitrary and are designed to avoid potential adverse effects of the treatment being tested by excluding individuals who may be at high risk of complications. If individuals are included in a trial who do not precisely fit the criteria stated in the protocol, there is a risk that the trial will fail to adequately test the effectiveness of the treatment.

What are the Phases of a Clinical Trial?

Clinical trials are divided into several phases, and each phase can be thought of as its own study. It may take many years for a treatment to pass through the various phases.

• Phase 0 – Study how the drug interacts with the disease tissue and affects the body in a small number of subjects.
• Phase I – Determine the safety of the treatment and the most appropriate dosage (of a medication) that can be safely given.
• Phase II – Determine the efficacy of the treatment. Specific criteria are established in advance to judge whether the treatment is working; side effects are also monitored.
• Phase III – Compare the efficacy of the treatment to other existing treatments for the disease. Following completion of Phase II, clinical data may be submitted to the FDA for approval, generally for indications that were tested in the clinical trial.
• Phase IV – Monitor the treatment after FDA approval for long-term outcomes and rare side effects.

What are the Risks and Benefits of Participating in a Clinical Trial?

There are many advantages of participating in a clinical trial, including access to new therapies for NF.  A few of the most important benefits include:

• Access to promising new treatments often not available outside the setting of a clinical trial
• Close monitoring, advice, care, and support by a research team of doctors and other healthcare professionals who understand NF
• The opportunity to be the first to benefit from a new medication or method under study.
• The chance to play a more active role in your won healthcare and gain a greater understanding of NF
• The opportunity to help society by contributing to medical research

It is also important to understand that some risks are also involved in clinical research. The specific risks associated with a clinical trial are described in detail in the consent document that participants are asked to sign before taking part in the research.