Clinical Trials for Neurofibromatosis

Moffitt Cancer Center has a robust portfolio of clinical trials that offer unique treatment opportunities for patients who have been diagnosed with neurofibromatosis. This relatively uncommon genetic condition causes benign and sometimes malignant tumors to develop within the nervous system, including the brain, spinal cord and nerves. In some instances, even benign tumors can progress into malignant peripheral nerve sheath tumors.

Within the scientific community, all of the causes of neurofibromatosis are not yet fully understood. However, through extensive medical research studies, the team in Moffitt’s Neuro-Oncology Program is continually expanding the knowledge base about this complex condition, and our work is leading to better outcomes and quality of life for all current and future patients with neurofibromatosis.

What is a clinical trial and why should I consider participating?

A clinical trial is a research study that focuses on evaluating the effectiveness of a promising new treatment option as compared to the current standard of care. For instance, the aim of a neurofibromatosis clinical trial might be to assess the success of a new medication or a new combination of existing medications in stopping or slowing the growth of neurofibromas.

While many patients with neurofibromatosis are treated with surgery, chemotherapy, radiation therapy and/or targeted therapy, clinical trials present additional options that allow some patients to:

• Access cutting-edge therapies that are not yet widely available in other settings
• Take a more active role in their care
• Contribute to important medical research that will help other patients with neurofibromatosis

Clinical trials are safe and the rights of the participants are well protected. Each study must first be approved by—and continually monitored by—an independent committee of physicians, scientists, community advocates and others.

Medical Reviewed by Dr. Peter Forsyth, Chair, Department of Neuro-Oncology  

If you have questions or would like to find neurofibromatosis clinical trials that are currently accepting new patients, please call 1-888-663-3488 or complete our new patient registration form online.