The Children's Tumor Foundation
09/15/2026 | Press release | Distributed by Public on 09/15/2026 09:24
Bringing NF Clinical Trials Closer to Home
For people living with neurofibromatosis type 1 (NF1) and schwannomatosis, participating in a clinical trial can require far more than a willingness to help advance research. It may mean traveling long distances to a specialized medical center, missing work or school, arranging childcare, and returning repeatedly for assessments.
A newly published paper from the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration explores how incorporating decentralized elements into clinical trials could help reduce those burdens and make NF research more accessible.
Published in Cancers, "New Approaches to Clinical Trials for Rare Diseases: Decentralized Trial Design for Neurofibromatosis Type 1 and Schwannomatosis" identifies clinical trial assessments that may be possible to complete at home, online, or closer to where participants live.
Rethinking Where Clinical Trials Take Place
Traditional clinical trials are generally organized around research sites, with participants traveling to those locations for examinations, imaging, questionnaires, and other assessments. In a decentralized clinical trial, some of those activities take place outside the primary trial site.
For people with rare conditions such as NF1 and schwannomatosis, this approach could be especially valuable. Because patients and specialized NF centers are dispersed across wide geographic areas, travel can limit who is able to participate in research. It can also make it more difficult for trials to recruit and retain enough participants.
The authors examined several tools and assessments that show promise for use in decentralized NF trials, including:
Electronic patient-reported outcome measures
Computer-based visual acuity testing
Digital technologies that measure physical function
Remote photography to assess cutaneous neurofibromas
Virtual neurocognitive evaluations
Certain imaging completed at local facilities
Biomarkers collected using specialized blood tubes or self-collection devices
The paper does not establish that all of these measures are ready for immediate use in clinical trials. Additional research is needed to validate them and determine when decentralized approaches are both feasible and scientifically reliable.
Instead, the recommendations provide a framework for designing and evaluating future NF trials that include carefully selected remote or local assessments.
Building Trials Around the Needs of Patients
The recommendations grew from discussions held by REiNS members in December 2023. The group included NF researchers, clinicians, and people affected by NF, bringing together scientific expertise and lived experience to consider both the opportunities and the challenges of decentralized research.
This patient-centered approach is essential. A trial can only succeed if people are able to participate, and the burdens of participation are not distributed equally. Travel requirements can be especially difficult for people who live far from an NF specialty center, have mobility or health limitations, lack paid time off, or are balancing work, school, and caregiving responsibilities.
Reducing unnecessary travel could help more people consider trial participation and allow studies to include participants from a wider range of geographic and socioeconomic backgrounds. At the same time, the authors emphasize that decentralized designs must preserve participant safety, data quality, privacy, and equitable access to the necessary technology.
The Role of REiNS
The REiNS International Collaboration was established in 2011 at the Children's Tumor Foundation NF Conference. CTF has continued to support this international effort, which brings together researchers, clinicians, industry representatives, and people with NF to improve the way NF clinical trials are designed and evaluated.
REiNS develops standardized methods for measuring whether a treatment is working. These shared standards help researchers compare results across studies, identify meaningful changes in patients' health and quality of life, and generate evidence that can move promising treatments forward.
This latest publication shows how that work continues to evolve. As new technologies create opportunities to conduct portions of clinical trials remotely, NF-specific guidance will be important to ensure that these methods are scientifically sound and meaningful to patients.
Decentralized clinical trials will not eliminate the need for specialized research centers or in-person care. Many examinations and procedures will still require expert clinical teams. But thoughtfully combining in-person visits with remote and local assessments could make participation less burdensome while preserving the rigor required for high-quality research.
For a rare and geographically dispersed patient population, bringing parts of a clinical trial closer to home could help bring more people into research and move the entire NF field forward.
The Children's Tumor Foundation published this content on September 15, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on September 15, 2026 at 15:24 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]