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.
Medically reviewed by Dr. Peter Forsyth, Chair, Neuro-Oncology Program.
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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Clinical Trials
CLINICAL TRIAL 20110
GBM Agile: Global Adaptive Trial Master Protocol
Condition: Neurologic Oncology
Intervention: ADI-PEG 20 (); AZD1390 (); BAY 73-4506 (Regorafenib); Lomustine (CeeNU); Paxalisib (); Regorafenib (Stivarga); Temodal (Temozolomide); Temozolomide (); Troriluzole (); VAL-083 (); VT1021 ()
CLINICAL TRIAL 20487
Phase I/II Study of Radiation Therapy Followed by Intrathecal Trastuzumab/Pertuzumab in the Management of HER2+ Breast Leptomeningeal Disease
Condition: Breast
Intervention: Herceptin (Trastuzumab); Pertuzumab (); Radiotherapy (); Trastuzumab (); rhuMAb HER2 (Trastuzumab)
CLINICAL TRIAL 20899
Phase I/II Study of Stereotactic Radiation and Abemaciclib with or without Imlunestrant in the Management of Hormone Receptor Positive HER2 Negative Breast Cancer Brain Metastases (STRONG Trial)
Condition: Breast
Intervention: Abemaciclib (); Endocrine Therapy (); Imlunestrant (); LY2835219 (Abemaciclib); Radiotherapy ()
CLINICAL TRIAL 21262
A First in Human dose escalation of Dendritic Cell Vaccine (DCV) administered Intrathecally (IT) primed against HER2/HER3 in Patients with Leptomeningeal Disease (LMD) from Triple-Negative Breast Cancer (TNBC) or HER2+ Breast Cancer (HER2+BC)
Condition: Neurologic Oncology
Intervention: Dendritic Cell Vaccine ()
CLINICAL TRIAL 22563
A randomized, open-label, multicentric, two-arm pivotal trial of SonoCloud-9 combined with carboplatin (CBDCA) vs standard of care lomustine (CCNU) or temozolomide (TMZ) in patients undergoing planned resection for first recurrence glioblastoma
Condition: Neurologic Oncology
Intervention: Lomustine (CeeNU); Paraplatin (carboplatin); Temodal (Temozolomide); Temozolomide (); carboplatin ()
CLINICAL TRIAL 23439
A Phase 1 and Randomized Phase 2 Trial of Selinexor and Temozolomide in Recurrent Glioblastoma
Condition: Neurologic Oncology
Intervention: KPT-330 (Selinexor); Selinexor (); Temodal (Temozolomide); Temozolomide ()
CLINICAL TRIAL 23486
Phase III Trial of Single Fraction Stereotactic Radiosurgery (SRS) Versus Fractionated SRS (FSRS) for Intact Brain Metastases
Condition: Neurologic Oncology
Intervention: Radiotherapy (); fSRS ()
CLINICAL TRIAL 23552
Complications and Management of Neurocutaneous Disorders
Condition: Neurologic Oncology
Intervention:
CLINICAL TRIAL 23773
A Phase 1b/2 Study of AZD0120 (also known as GC012F), a Chimeric Antigen Receptor T Cell Therapy Targeting CD19 and B cell Maturation Antigen in Participants with Relapsed or Refractory AL Amyloidosis
Condition: Malignant Hematology
Intervention: AZD0120 ()
CLINICAL TRIAL 23834
Deciphering the Role of Immune-Related Gene Signature and Tumor-Immune Microenvironment in Glioblastoma
Condition: Neurologic Oncology
Intervention:
Treatment