The DoD’s Neurofibromatosis Clinical Trial Grant for Advancing NF Treatments

The NFRP Clinical Trial Award supports the rapid implementation of clinical trials with the potential to have a significant impact on the treatment or management of NF. Clinical trials may be designed to evaluate promising new products, pharmacologic agents (drugs or biologics), devices, clinical guidance, and/or emerging approaches and technologies. Proposed projects may range from small proof-of-concept trials (e.g., pilot, first in human, phase 0) to demonstrate the feasibility or inform the design of more advanced trials through large-scale trials to determine efficacy in relevant patient populations.

The vision of the FY23 NFRP is to decrease the clinical impact of NF. Toward this end, the NFRP seeks to support innovative, high-impact research that will foster new directions for and address neglected issues in NF research; sponsor multidisciplinary and multi-institutional collaborations that will bring new perspectives to the field; promote translational and clinical studies to move promising ideas from bench to bedside; and develop a balanced portfolio of meritorious research related to all aspects of NF1, NF2, and Schwannomatosis.

Areas of Emphasis: The FY23 NFRP strongly encourages research applications that specifically address the critical needs of the NF community in one or more of the Areas of Emphasis listed below. All applications are also encouraged to include materials and data from diverse populations in their research.

• NF2 and Schwannomatosis-related areas (e.g., hearing, balance, schwannoma, ependymoma, meningioma, LZTR1, SMARCB1)

• Endpoint validation, biomarker discovery, and technological innovation for assessments

• Application of data science

• Non-tumor manifestations not limited to:

o Pain

o Cognitive manifestations

o Sleep

The NFRP Clinical Trial Award supports the rapid implementation of clinical trials with the potential to have a significant impact on the treatment or management of NF. Clinical trials may be designed to evaluate promising new products, pharmacologic agents (drugs or biologics), devices, clinical guidance, and/or emerging approaches and technologies. Proposed projects may range from small proof-of-concept trials (e.g., pilot, first in human, phase 0) to demonstrate the feasibility or inform the design of more advanced trials through large-scale trials to determine efficacy in relevant patient populations.

The vision of the FY23 NFRP is to decrease the clinical impact of NF. Toward this end, the NFRP seeks to support innovative, high-impact research that will foster new directions for and address neglected issues in NF research; sponsor multidisciplinary and multi-institutional collaborations that will bring new perspectives to the field; promote translational and clinical studies to move promising ideas from bench to bedside; and develop a balanced portfolio of meritorious research related to all aspects of NF1, NF2, and Schwannomatosis.

Areas of Emphasis: The FY23 NFRP strongly encourages research applications that specifically address the critical needs of the NF community in one or more of the Areas of Emphasis listed below. All applications are also encouraged to include materials and data from diverse populations in their research.

• NF2 and Schwannomatosis-related areas (e.g., hearing, balance, schwannoma, ependymoma, meningioma, LZTR1, SMARCB1)

• Endpoint validation, biomarker discovery, and technological innovation for assessments

• Application of data science

• Non-tumor manifestations not limited to:

o Pain

o Cognitive manifestations

o Sleep