DoD Neurofibromatosis Clinical Trial Award | HT9425 23 NFRP CTA

Frequently Asked Questions (FAQ) - DoD NFRP Clinical Trial Award (HT9425-23-NFRP-CTA)

1) What is the DoD Neurofibromatosis Research Program (NFRP) Clinical Trial Award?

The NFRP Clinical Trial Award is a Department of Defense funding opportunity intended to move neurofibromatosis (NF) research quickly into the clinic. It supports the rapid start-up and execution of clinical trials that could meaningfully change how NF is treated or managed, with an emphasis on practical, patient-relevant outcomes.

2) What is the main goal of this award for FY23?

The program frames its FY23 goal as decreasing the clinical impact of NF. In practice, that means focusing on outcomes that improve health, function, and quality of life for people living with NF1, NF2, and Schwannomatosis.

3) What kinds of clinical trials are supported (stage and size)?

This opportunity supports a wide range of trial stages and sizes, from early, smaller proof-of-concept studies (such as pilot trials, first-in-human efforts, or phase 0 work) to larger-scale trials designed to determine efficacy in the relevant patient population.

4) Are early-stage studies like pilot, first-in-human, or phase 0 trials eligible?

Yes. The announcement explicitly notes that applicants can propose early and smaller proof-of-concept studies, including pilot trials, first-in-human efforts, and phase 0 work. These may be aimed at demonstrating feasibility, refining dosing or procedures, or generating data needed to justify a larger trial.

5) Are larger efficacy trials eligible under this mechanism?

Yes. The program also allows proposals involving larger-scale trials designed to determine efficacy in the relevant patient population.

6) What types of interventions or approaches can be tested in supported trials?

The award is flexible regarding what is being evaluated. Trials may test pharmacologic agents (including drugs and biologics), devices, clinical guidance or care approaches, and other emerging methods or technologies that could improve outcomes for people with NF.

7) Which NF conditions are within scope?

The program is oriented toward outcomes that improve quality of life for people living with NF1, NF2, and Schwannomatosis, and it supports a balanced portfolio across these conditions rather than focusing narrowly on one area.

8) What does the program mean by "patient impact"?

The award is aimed at trials with clear potential to meaningfully change how NF is treated or managed. Examples described include testing a new therapy, improving symptom management, validating better ways to measure disease, or bringing emerging technology into real-world clinical use.

9) Does this award prioritize translational research?

Yes. A central priority described is translational and clinical work that moves promising ideas from bench to bedside, accelerating trials that can change care.

10) Is innovation emphasized, and what does that look like in this program?

Yes. The NFRP emphasizes high-impact research that opens new directions and addresses issues that have been underexplored or neglected in NF. Innovation is also reflected in the emphasis on improved endpoints/biomarkers, new assessment technologies, and data science approaches that strengthen clinical trials.

11) Are multidisciplinary and multi-institutional collaborations encouraged?

Yes. The program explicitly encourages multidisciplinary and multi-institutional collaborations, including proposals that bring together different specialties and multiple clinical sites.

12) What types of disciplines or specialties might be relevant for collaboration?

The announcement gives examples such as neurology, oncology, otology, imaging, genetics, pain medicine, rehabilitation, and data science, among others.

13) What are the FY23 Areas of Emphasis?

FY23 emphasis areas include: NF2 and Schwannomatosis-related problems (including hearing and balance issues, schwannomas, and associated tumor types such as ependymomas and meningiomas, as well as genetic drivers and subtypes involving LZTR1 and SMARCB1); endpoint validation and biomarker discovery plus technological innovation for assessments; application of data science; and non-tumor manifestations such as pain, cognitive manifestations, and sleep.

14) Does the opportunity specifically highlight NF2 and Schwannomatosis?

Yes. One highlighted area focuses on NF2 and Schwannomatosis-related challenges, including hearing and balance issues, schwannomas, related tumor types (including ependymomas and meningiomas), and genetic drivers/subtypes such as LZTR1 and SMARCB1.

15) Are trials focused on endpoints, biomarkers, or measurement tools considered a good fit?

Yes. The program prioritizes endpoint validation and biomarker discovery, as well as technological innovation for assessments, to improve how trials measure treatment response, disease change, and patient function.

16) How does data science fit into this funding opportunity?

The program encourages the application of data science, including advanced analytics, integration of clinical and real-world data, computational approaches to improve trial design, and methods that sharpen prediction and decision-making.

17) Are non-tumor symptoms like pain, cognition, and sleep within scope for clinical trials?

Yes. The opportunity explicitly calls attention to non-tumor manifestations, naming pain, cognitive manifestations, and sleep, reinforcing that symptom-focused trials can directly address patient needs.

18) Does the program expect attention to diversity in trial materials and data?

Yes. Across the emphasis areas, applications are encouraged to include materials and data from diverse populations, reflecting an expectation that evidence should be relevant and equitable across different patient groups.

19) Who is the sponsoring agency and administering office for this opportunity?

The opportunity is offered by the Department of Defense, Department of the Army, through the US Army Medical Research Acquisition Activity (USAMRAA).

20) What is the opportunity number and CFDA listing?

The opportunity number is HT9425-23-NFRP-CTA. It is listed as a discretionary funding opportunity under CFDA 12.420.

21) What funding mechanisms are used (grant vs cooperative agreement)?

The notice indicates it uses both grant and cooperative agreement mechanisms. This means some awards may involve more active involvement or coordination by the funder, depending on the specific agreement structure.

22) Who is eligible to apply?

Eligibility is described as unrestricted (open to any type of entity), subject to any additional limits that may be described in the full announcement.

23) What were the posting date and closing date listed in the notice?

The posting date was May 22, 2023, and the original closing date listed was August 25, 2023.

24) How many awards are expected?

The notice lists an expected number of awards of 1.

25) What is the maximum award amount (ceiling)?

The award ceiling is shown as 0 in the summary, which typically indicates the ceiling is not specified in the summary and would need to be confirmed in the full funding announcement or related program documents.

26) What is the overall takeaway: what kinds of projects are most aligned?

Based on the description, the best-aligned projects are clinical trials that can start and run quickly, have a clear line of sight to meaningful patient impact, and produce measurable, clinically relevant outcomes. Strong alignment is also suggested for projects that address FY23 emphasis areas (especially NF2/Schwannomatosis challenges, improved endpoints/biomarkers/assessment technology, data science-enabled trial improvements, and non-tumor symptoms like pain, cognition, and sleep), and that involve multidisciplinary, multi-site collaboration and diverse populations.