Clinical Trials for Neurofibromatosis

Neurofibromatosis patient discussing clinical trials

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.

 

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Open Clinical Trials:

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CLINICAL TRIAL 18752

ACTION Trial- Adoptive Cellular Therapy following Dose-Intensified Temozolomide in Newly-diagnosed Pediatric High-grade Gliomas (Phase I)

Disease Site: Brain and Nervous System, Gliomas

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CLINICAL TRIAL 19327

A Phase 2 Trial of Pembrolizumab Plus Bevacizumab in Patients with Metastatic Melanoma or Non-small Cell Lung Cancer with Untreated Brain Metastases

Disease Site: Non small cell lung cancer, Brain and Nervous System, Lung, Melanoma, skin, Gliomas

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CLINICAL TRIAL 20145

A Pilot Study of Intratumorally and Intraparenchymally Administered OS2966 using Convection-enhanced Delivery in Patients with Recurrent/Progressive High-grade Glioma Undergoing a Clinically-indicated Surgical Resection

Disease Site: Brain and Nervous System, Gliomas

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CLINICAL TRIAL 20487

Phase I/II Study of Radiation Therapy Followed by Intrathecal Trastuzumab/Pertuzumab in the Management of HER2+ Breast Leptomeningeal Disease

Disease Site: Brain and Nervous System, Breast, Leptomeningeal (LMD)

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CLINICAL TRIAL 20637

Phase III Trial of Post-Surgical Single Fraction Stereotactic Radiosurgery (SRS) Compared with Fractionated SRS (FSRS) for Resected Metastatic Brain Disease

Disease Site: Brain and Nervous System, Brain metastasis

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CLINICAL TRIAL 20899

Phase I/II Study of Stereotactic Radiation and Abemaciclib in the Management of Hormone Receptor Positive HER2 Negative Breast Cancer Brain Metastases

Disease Site: Brain and Nervous System, Breast

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CLINICAL TRIAL 20956

A Two-Part, Phase 1a/B, Open-Label, Multicenter Trial Evaluating Pharmacokinetics, Safety and Efficacy Of PF-07284890 (ARRY-461) In Participants With BRAF V600-Mutant Solid Tumors With And Without Brain Involvement

Disease Site: Any Site, Brain and Nervous System, Larynx, Lip, Oral Cavity and Pharynx, Lung, Melanoma, skin, Other Skin, Brain metastasis, Gliomas

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CLINICAL TRIAL 20964

A Phase 2 Basket Study of Tucatinib in Combination with Trastuzumab in Subjects with Previously Treated, Locally-Advanced Unresectable or Metastatic Solid Tumors Driven by HER2 Alterations

Disease Site: Brain and Nervous System, Breast, Ill-Defined Sites, Lung, Other Digestive Organ, Urinary Bladder, Brain metastasis, Leptomeningeal (LMD)

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CLINICAL TRIAL 21022

A Phase 1b/2, Master Protocol Evaluating the Safety, Tolerability, Pharmacokinetics, and Efficacy of AMG 510 (pINN sotorasib) in Subjects With Advanced Solid Tumors With KRAS p.G12C Mutation

Disease Site: Any Site, Brain and Nervous System, Lung, Pancreas, Rectum, Brain metastasis, Leptomeningeal (LMD)

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CLINICAL TRIAL 21039

A Pivotal Randomized, Open-Label Study Of OPTUNE(R)(TTFIELDS, 200KHZ) Concomitant With Radiation Therapy And Temozolomide For The Treatment Of Newly Diagnosed Glioblastoma

Disease Site: Brain and Nervous System, Gliomas

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CLINICAL TRIAL 21084

A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of Enzastaurin Added to Temozolomide During and Following Radiation Therapy in Newly Diagnosed Glioblastoma Patients Who Possess the Novel Genomic Biomarker DGM1PI

Disease Site: Brain and Nervous System, Gliomas

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CLINICAL TRIAL 21193

Pre-operative Hypofractionated Stereotactic Radiosurgery for Resectable Brain Metastases

Disease Site: Brain and Nervous System, Brain metastasis

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CLINICAL TRIAL 21681

A Safety Run-In and Phase II Study Evaluating the Efficacy, Safety, and Impact on the Tumor Microenvironment of the Combination of Tocilizumab, Atezolizumab, and Fractionated Stereotactic Radiotherapy in Recurrent Glioblastoma

Disease Site: Brain and Nervous System

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CLINICAL TRIAL 19731

Electronic Social Network Assessment Program (eSNAP) + Caregiver Navigator Intervention for Neuro-Oncology Couples

Disease Site: Brain and Nervous System, Brain metastasis, Gliomas, Leptomeningeal (LMD), Meningiomas

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CLINICAL TRIAL 21737

A Pilot Study of Advanced Multi-parametric MRI to Evaluate De Novo Glioblastoma Tumors Prior to Surgical Resection

Disease Site: Brain and Nervous System, Gliomas

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